SK95599A3 - Adenoviral-vector-mediated gene transfer into medullary motor neurons - Google Patents

Adenoviral-vector-mediated gene transfer into medullary motor neurons Download PDF

Info

Publication number: SK95599A3
Authority: SK; Slovakia
Prior art keywords: nucleic acid; motor neurons; adenovirus; virus; nucleic acids
Prior art date: 1997-01-17

Application number

SK955-99A

Other languages

English (en)

Slovak (sk)

Inventor

Francoise Finiels

Minerva Gimenez-Ribotta

Jacques Mallet

Alain Privat

Frederic Revah

Original Assignee

Rhone Poulenc Rorer Sa

Inst Nat Sante Rech Med

Priority date (The priority date is an assumption and is not a legal conclusion. Google has not performed a legal analysis and makes no representation as to the accuracy of the date listed.)

1997-01-17

Filing date

1998-01-16

Publication date

2000-05-16

1997-01-17 Priority claimed from US08/785,074 external-priority patent/US6632427B1/en

1998-01-16 Application filed by Rhone Poulenc Rorer Sa, Inst Nat Sante Rech Med filed Critical Rhone Poulenc Rorer Sa

2000-05-16 Publication of SK95599A3 publication Critical patent/SK95599A3/sk

Links

210000002161 motor neuron Anatomy 0.000 title claims abstract description 84
108090000623 proteins and genes Proteins 0.000 title claims description 79
239000013598 vector Substances 0.000 title claims description 43
238000012546 transfer Methods 0.000 title description 26
230000001404 mediated effect Effects 0.000 title description 3
150000007523 nucleic acids Chemical class 0.000 claims abstract description 81
210000003205 muscle Anatomy 0.000 claims abstract description 80
108020004707 nucleic acids Proteins 0.000 claims abstract description 71
102000039446 nucleic acids Human genes 0.000 claims abstract description 71
230000004770 neurodegeneration Effects 0.000 claims abstract description 12
239000000203 mixture Substances 0.000 claims abstract description 9
208000015122 neurodegenerative disease Diseases 0.000 claims abstract description 6
208000014674 injury Diseases 0.000 claims abstract description 5
210000000653 nervous system Anatomy 0.000 claims abstract description 5
241000701161 unidentified adenovirus Species 0.000 claims description 69
241001465754 Metazoa Species 0.000 claims description 47
238000002347 injection Methods 0.000 claims description 38
239000007924 injection Substances 0.000 claims description 38
108090000742 Neurotrophin 3 Proteins 0.000 claims description 30
230000014509 gene expression Effects 0.000 claims description 29
206010002026 amyotrophic lateral sclerosis Diseases 0.000 claims description 28
241000700605 Viruses Species 0.000 claims description 26
102000004169 proteins and genes Human genes 0.000 claims description 26
201000010099 disease Diseases 0.000 claims description 24
208000037265 diseases, disorders, signs and symptoms Diseases 0.000 claims description 24
210000000278 spinal cord Anatomy 0.000 claims description 24
241000282414 Homo sapiens Species 0.000 claims description 19
244000309466 calf Species 0.000 claims description 19
108010025020 Nerve Growth Factor Proteins 0.000 claims description 18
102000007072 Nerve Growth Factors Human genes 0.000 claims description 18
108010005939 Ciliary Neurotrophic Factor Proteins 0.000 claims description 17
102100031614 Ciliary neurotrophic factor Human genes 0.000 claims description 17
108091061960 Naked DNA Proteins 0.000 claims description 16
239000003900 neurotrophic factor Substances 0.000 claims description 16
241000702421 Dependoparvovirus Species 0.000 claims description 12
238000002360 preparation method Methods 0.000 claims description 11
239000003795 chemical substances by application Substances 0.000 claims description 10
230000002950 deficient Effects 0.000 claims description 10
239000003102 growth factor Substances 0.000 claims description 10
230000010076 replication Effects 0.000 claims description 9
102000004190 Enzymes Human genes 0.000 claims description 8
108090000790 Enzymes Proteins 0.000 claims description 8
241001430294 unidentified retrovirus Species 0.000 claims description 8
208000002320 spinal muscular atrophy Diseases 0.000 claims description 7
229940032018 neurotrophin 3 Drugs 0.000 claims description 6
230000006378 damage Effects 0.000 claims description 5
230000001939 inductive effect Effects 0.000 claims description 5
108090000695 Cytokines Proteins 0.000 claims description 4
102000004127 Cytokines Human genes 0.000 claims description 4
241000124008 Mammalia Species 0.000 claims description 4
208000027418 Wounds and injury Diseases 0.000 claims description 4
210000003141 lower extremity Anatomy 0.000 claims description 4
210000000715 neuromuscular junction Anatomy 0.000 claims description 4
210000002976 pectoralis muscle Anatomy 0.000 claims description 4
241001068295 Replication defective viruses Species 0.000 claims description 3
230000003376 axonal effect Effects 0.000 claims description 3
239000002858 neurotransmitter agent Substances 0.000 claims description 3
230000001242 postsynaptic effect Effects 0.000 claims description 3
208000028389 Nerve injury Diseases 0.000 claims description 2
230000008764 nerve damage Effects 0.000 claims description 2
210000001364 upper extremity Anatomy 0.000 claims description 2
102000004230 Neurotrophin 3 Human genes 0.000 claims 4
230000007433 nerve pathway Effects 0.000 claims 1
230000014616 translation Effects 0.000 claims 1
238000000034 method Methods 0.000 abstract description 41
230000007170 pathology Effects 0.000 abstract description 2
230000008733 trauma Effects 0.000 abstract description 2
241000699670 Mus sp. Species 0.000 description 82
210000004027 cell Anatomy 0.000 description 34
230000000694 effects Effects 0.000 description 31
210000003050 axon Anatomy 0.000 description 24
235000018102 proteins Nutrition 0.000 description 23
108010005774 beta-Galactosidase Proteins 0.000 description 21
102100029268 Neurotrophin-3 Human genes 0.000 description 20
230000001105 regulatory effect Effects 0.000 description 20
239000013612 plasmid Substances 0.000 description 19
238000010255 intramuscular injection Methods 0.000 description 18
239000007927 intramuscular injection Substances 0.000 description 17
241000699666 Mus <mouse, genus> Species 0.000 description 15
108090000765 processed proteins & peptides Proteins 0.000 description 15
102000004196 processed proteins & peptides Human genes 0.000 description 14
230000001225 therapeutic effect Effects 0.000 description 14
229920001184 polypeptide Polymers 0.000 description 13
102100026189 Beta-galactosidase Human genes 0.000 description 12
FTALBRSUTCGOEG-UHFFFAOYSA-N Riluzole Chemical compound C1=C(OC(F)(F)F)C=C2SC(N)=NC2=C1 FTALBRSUTCGOEG-UHFFFAOYSA-N 0.000 description 12
108700019146 Transgenes Proteins 0.000 description 12
230000007850 degeneration Effects 0.000 description 12
108020004414 DNA Proteins 0.000 description 11
108091028043 Nucleic acid sequence Proteins 0.000 description 11
210000005036 nerve Anatomy 0.000 description 11
238000010171 animal model Methods 0.000 description 10
238000012217 deletion Methods 0.000 description 10
230000037430 deletion Effects 0.000 description 10
238000001727 in vivo Methods 0.000 description 10
238000007918 intramuscular administration Methods 0.000 description 10
229960004181 riluzole Drugs 0.000 description 10
239000002253 acid Substances 0.000 description 9
230000000692 anti-sense effect Effects 0.000 description 9
230000004083 survival effect Effects 0.000 description 9
241000700159 Rattus Species 0.000 description 8
235000001014 amino acid Nutrition 0.000 description 8
239000000835 fiber Substances 0.000 description 8
239000012634 fragment Substances 0.000 description 8
208000005264 motor neuron disease Diseases 0.000 description 8
230000035772 mutation Effects 0.000 description 8
230000002232 neuromuscular Effects 0.000 description 8
210000002569 neuron Anatomy 0.000 description 8
230000000750 progressive effect Effects 0.000 description 8
239000000126 substance Substances 0.000 description 8
230000001228 trophic effect Effects 0.000 description 8
230000003612 virological effect Effects 0.000 description 8
102000004219 Brain-derived neurotrophic factor Human genes 0.000 description 7
108090000715 Brain-derived neurotrophic factor Proteins 0.000 description 7
108060001084 Luciferase Proteins 0.000 description 7
150000001413 amino acids Chemical class 0.000 description 7
230000034994 death Effects 0.000 description 7
238000002474 experimental method Methods 0.000 description 7
238000001415 gene therapy Methods 0.000 description 7
230000006801 homologous recombination Effects 0.000 description 7
238000002744 homologous recombination Methods 0.000 description 7
238000002372 labelling Methods 0.000 description 7
230000000508 neurotrophic effect Effects 0.000 description 7
230000007441 retrograde transport Effects 0.000 description 7
230000001177 retroviral effect Effects 0.000 description 7
239000000243 solution Substances 0.000 description 7
239000005089 Luciferase Substances 0.000 description 6
238000004458 analytical method Methods 0.000 description 6
WQZGKKKJIJFFOK-FPRJBGLDSA-N beta-D-galactose Chemical compound OC[C@H]1O[C@@H](O)[C@H](O)[C@@H](O)[C@H]1O WQZGKKKJIJFFOK-FPRJBGLDSA-N 0.000 description 6
210000005056 cell body Anatomy 0.000 description 6
230000006870 function Effects 0.000 description 6
239000002773 nucleotide Substances 0.000 description 6
125000003729 nucleotide group Chemical group 0.000 description 6
210000000115 thoracic cavity Anatomy 0.000 description 6
206010056677 Nerve degeneration Diseases 0.000 description 5
206010033799 Paralysis Diseases 0.000 description 5
108010012715 Superoxide dismutase Proteins 0.000 description 5
230000000875 corresponding effect Effects 0.000 description 5
230000002638 denervation Effects 0.000 description 5
238000000338 in vitro Methods 0.000 description 5
210000004705 lumbosacral region Anatomy 0.000 description 5
230000007823 neuropathy Effects 0.000 description 5
201000001119 neuropathy Diseases 0.000 description 5
238000010186 staining Methods 0.000 description 5
238000013518 transcription Methods 0.000 description 5
230000035897 transcription Effects 0.000 description 5
108090000932 Calcitonin Gene-Related Peptide Proteins 0.000 description 4
108020004635 Complementary DNA Proteins 0.000 description 4
102100028188 Cystatin-F Human genes 0.000 description 4
101710169749 Cystatin-F Proteins 0.000 description 4
241001135569 Human adenovirus 5 Species 0.000 description 4
206010061218 Inflammation Diseases 0.000 description 4
FAPWRFPIFSIZLT-UHFFFAOYSA-M Sodium chloride Chemical compound [Na+].[Cl-] FAPWRFPIFSIZLT-UHFFFAOYSA-M 0.000 description 4
102000019197 Superoxide Dismutase Human genes 0.000 description 4
150000007513 acids Chemical class 0.000 description 4
VREFGVBLTWBCJP-UHFFFAOYSA-N alprazolam Chemical compound C12=CC(Cl)=CC=C2N2C(C)=NN=C2CN=C1C1=CC=CC=C1 VREFGVBLTWBCJP-UHFFFAOYSA-N 0.000 description 4
-1 amino amino Chemical class 0.000 description 4
230000009286 beneficial effect Effects 0.000 description 4
238000010804 cDNA synthesis Methods 0.000 description 4
239000002299 complementary DNA Substances 0.000 description 4
239000003814 drug Substances 0.000 description 4
239000003937 drug carrier Substances 0.000 description 4
208000015181 infectious disease Diseases 0.000 description 4
230000004054 inflammatory process Effects 0.000 description 4
230000030214 innervation Effects 0.000 description 4
230000003902 lesion Effects 0.000 description 4
238000004806 packaging method and process Methods 0.000 description 4
208000033808 peripheral neuropathy Diseases 0.000 description 4
239000011780 sodium chloride Substances 0.000 description 4
210000001519 tissue Anatomy 0.000 description 4
238000011830 transgenic mouse model Methods 0.000 description 4
239000013603 viral vector Substances 0.000 description 4
108010085238 Actins Proteins 0.000 description 3
102000007469 Actins Human genes 0.000 description 3
102100028892 Cardiotrophin-1 Human genes 0.000 description 3
LFQSCWFLJHTTHZ-UHFFFAOYSA-N Ethanol Chemical compound CCO LFQSCWFLJHTTHZ-UHFFFAOYSA-N 0.000 description 3
102000024452 GDNF Human genes 0.000 description 3
108091010837 Glial cell line-derived neurotrophic factor Proteins 0.000 description 3
PEDCQBHIVMGVHV-UHFFFAOYSA-N Glycerine Chemical compound OCC(O)CO PEDCQBHIVMGVHV-UHFFFAOYSA-N 0.000 description 3
DGAQECJNVWCQMB-PUAWFVPOSA-M Ilexoside XXIX Chemical compound C[C@@H]1CC[C@@]2(CC[C@@]3(C(=CC[C@H]4[C@]3(CC[C@@H]5[C@@]4(CC[C@@H](C5(C)C)OS(=O)(=O)[O-])C)C)[C@@H]2[C@]1(C)O)C)C(=O)O[C@H]6[C@@H]([C@H]([C@@H]([C@H](O6)CO)O)O)O.[Na+] DGAQECJNVWCQMB-PUAWFVPOSA-M 0.000 description 3
208000026072 Motor neurone disease Diseases 0.000 description 3
241001529936 Murinae Species 0.000 description 3
241000699660 Mus musculus Species 0.000 description 3
241000714474 Rous sarcoma virus Species 0.000 description 3
108700005077 Viral Genes Proteins 0.000 description 3
230000036982 action potential Effects 0.000 description 3
238000007792 addition Methods 0.000 description 3
210000004556 brain Anatomy 0.000 description 3
206010061592 cardiac fibrillation Diseases 0.000 description 3
108010041776 cardiotrophin 1 Proteins 0.000 description 3
230000030833 cell death Effects 0.000 description 3
210000003169 central nervous system Anatomy 0.000 description 3
230000001886 ciliary effect Effects 0.000 description 3
230000006467 collateral growth Effects 0.000 description 3
230000000763 evoking effect Effects 0.000 description 3
210000003414 extremity Anatomy 0.000 description 3
230000002600 fibrillogenic effect Effects 0.000 description 3
108700004026 gag Genes Proteins 0.000 description 3
230000028709 inflammatory response Effects 0.000 description 3
208000032839 leukemia Diseases 0.000 description 3
230000007774 longterm Effects 0.000 description 3
238000004519 manufacturing process Methods 0.000 description 3
108020004999 messenger RNA Proteins 0.000 description 3
230000004048 modification Effects 0.000 description 3
238000012986 modification Methods 0.000 description 3
230000036961 partial effect Effects 0.000 description 3
210000003105 phrenic nerve Anatomy 0.000 description 3
230000008569 process Effects 0.000 description 3
210000000499 ranvier's node Anatomy 0.000 description 3
230000009467 reduction Effects 0.000 description 3
230000002829 reductive effect Effects 0.000 description 3
230000004044 response Effects 0.000 description 3
210000003497 sciatic nerve Anatomy 0.000 description 3
230000002269 spontaneous effect Effects 0.000 description 3
238000010561 standard procedure Methods 0.000 description 3
208000024891 symptom Diseases 0.000 description 3
230000002103 transcriptional effect Effects 0.000 description 3
230000009261 transgenic effect Effects 0.000 description 3
238000012250 transgenic expression Methods 0.000 description 3
230000029812 viral genome replication Effects 0.000 description 3
XLYOFNOQVPJJNP-UHFFFAOYSA-N water Chemical compound O XLYOFNOQVPJJNP-UHFFFAOYSA-N 0.000 description 3
PUPZLCDOIYMWBV-UHFFFAOYSA-N (+/-)-1,3-Butanediol Chemical compound CC(O)CCO PUPZLCDOIYMWBV-UHFFFAOYSA-N 0.000 description 2
108091032973 (ribonucleotides)n+m Proteins 0.000 description 2
241000271566 Aves Species 0.000 description 2
206010068597 Bulbospinal muscular atrophy congenital Diseases 0.000 description 2
OYPRJOBELJOOCE-UHFFFAOYSA-N Calcium Chemical compound [Ca] OYPRJOBELJOOCE-UHFFFAOYSA-N 0.000 description 2
108091026890 Coding region Proteins 0.000 description 2
206010061818 Disease progression Diseases 0.000 description 2
102100039289 Glial fibrillary acidic protein Human genes 0.000 description 2
101710193519 Glial fibrillary acidic protein Proteins 0.000 description 2
WQZGKKKJIJFFOK-GASJEMHNSA-N Glucose Natural products OC[C@H]1OC(O)[C@H](O)[C@@H](O)[C@@H]1O WQZGKKKJIJFFOK-GASJEMHNSA-N 0.000 description 2
101000693844 Homo sapiens Insulin-like growth factor-binding protein complex acid labile subunit Proteins 0.000 description 2
102000018251 Hypoxanthine Phosphoribosyltransferase Human genes 0.000 description 2
108010091358 Hypoxanthine Phosphoribosyltransferase Proteins 0.000 description 2
208000027747 Kennedy disease Diseases 0.000 description 2
206010024453 Ligament sprain Diseases 0.000 description 2
TWRXJAOTZQYOKJ-UHFFFAOYSA-L Magnesium chloride Chemical compound [Mg+2].[Cl-].[Cl-] TWRXJAOTZQYOKJ-UHFFFAOYSA-L 0.000 description 2
108090000099 Neurotrophin-4 Proteins 0.000 description 2
102100033857 Neurotrophin-4 Human genes 0.000 description 2
ZLMJMSJWJFRBEC-UHFFFAOYSA-N Potassium Chemical compound [K] ZLMJMSJWJFRBEC-UHFFFAOYSA-N 0.000 description 2
241000713896 Spleen necrosis virus Species 0.000 description 2
208000010040 Sprains and Strains Diseases 0.000 description 2
239000004098 Tetracycline Substances 0.000 description 2
208000006269 X-Linked Bulbo-Spinal Atrophy Diseases 0.000 description 2
210000003626 afferent pathway Anatomy 0.000 description 2
230000008901 benefit Effects 0.000 description 2
229940077737 brain-derived neurotrophic factor Drugs 0.000 description 2
239000007975 buffered saline Substances 0.000 description 2
230000010261 cell growth Effects 0.000 description 2
239000007795 chemical reaction product Substances 0.000 description 2
210000000349 chromosome Anatomy 0.000 description 2
238000012761 co-transfection Methods 0.000 description 2
150000001875 compounds Chemical class 0.000 description 2
238000010276 construction Methods 0.000 description 2
230000003247 decreasing effect Effects 0.000 description 2
238000001514 detection method Methods 0.000 description 2
238000011161 development Methods 0.000 description 2
230000018109 developmental process Effects 0.000 description 2
239000003085 diluting agent Substances 0.000 description 2
230000005750 disease progression Effects 0.000 description 2
229940079593 drug Drugs 0.000 description 2
239000003623 enhancer Substances 0.000 description 2
108700004025 env Genes Proteins 0.000 description 2
230000002255 enzymatic effect Effects 0.000 description 2
208000019995 familial amyotrophic lateral sclerosis Diseases 0.000 description 2
238000009472 formulation Methods 0.000 description 2
210000005046 glial fibrillary acidic protein Anatomy 0.000 description 2
230000012010 growth Effects 0.000 description 2
230000028993 immune response Effects 0.000 description 2
230000036039 immunity Effects 0.000 description 2
230000002055 immunohistochemical effect Effects 0.000 description 2
230000001976 improved effect Effects 0.000 description 2
230000006698 induction Effects 0.000 description 2
201000010901 lateral sclerosis Diseases 0.000 description 2
230000000670 limiting effect Effects 0.000 description 2
239000002502 liposome Substances 0.000 description 2
239000003550 marker Substances 0.000 description 2
108010093030 mouse neurotropin 3 Proteins 0.000 description 2
230000003387 muscular Effects 0.000 description 2
210000001087 myotubule Anatomy 0.000 description 2
210000002241 neurite Anatomy 0.000 description 2
239000002245 particle Substances 0.000 description 2
230000003950 pathogenic mechanism Effects 0.000 description 2
230000002093 peripheral effect Effects 0.000 description 2
239000000902 placebo Substances 0.000 description 2
229940068196 placebo Drugs 0.000 description 2
108700004029 pol Genes Proteins 0.000 description 2
229920000642 polymer Polymers 0.000 description 2
230000002035 prolonged effect Effects 0.000 description 2
238000011160 research Methods 0.000 description 2
238000012552 review Methods 0.000 description 2
238000012216 screening Methods 0.000 description 2
210000002027 skeletal muscle Anatomy 0.000 description 2
210000000329 smooth muscle myocyte Anatomy 0.000 description 2
239000011734 sodium Substances 0.000 description 2
229910052708 sodium Inorganic materials 0.000 description 2
239000002904 solvent Substances 0.000 description 2
239000008223 sterile water Substances 0.000 description 2
230000000638 stimulation Effects 0.000 description 2
238000006467 substitution reaction Methods 0.000 description 2
239000000758 substrate Substances 0.000 description 2
239000000725 suspension Substances 0.000 description 2
238000003786 synthesis reaction Methods 0.000 description 2
238000007910 systemic administration Methods 0.000 description 2
230000009885 systemic effect Effects 0.000 description 2
229960002180 tetracycline Drugs 0.000 description 2
229930101283 tetracycline Natural products 0.000 description 2
235000019364 tetracycline Nutrition 0.000 description 2
150000003522 tetracyclines Chemical class 0.000 description 2
230000005030 transcription termination Effects 0.000 description 2
230000007306 turnover Effects 0.000 description 2
241001529453 unidentified herpesvirus Species 0.000 description 2
210000005167 vascular cell Anatomy 0.000 description 2
WRIDQFICGBMAFQ-UHFFFAOYSA-N (E)-8-Octadecenoic acid Natural products CCCCCCCCCC=CCCCCCCC(O)=O WRIDQFICGBMAFQ-UHFFFAOYSA-N 0.000 description 1
LQJBNNIYVWPHFW-UHFFFAOYSA-N 20:1omega9c fatty acid Natural products CCCCCCCCCCC=CCCCCCCCC(O)=O LQJBNNIYVWPHFW-UHFFFAOYSA-N 0.000 description 1
OCKGFTQIICXDQW-ZEQRLZLVSA-N 5-[(1r)-1-hydroxy-2-[4-[(2r)-2-hydroxy-2-(4-methyl-1-oxo-3h-2-benzofuran-5-yl)ethyl]piperazin-1-yl]ethyl]-4-methyl-3h-2-benzofuran-1-one Chemical compound C1=C2C(=O)OCC2=C(C)C([C@@H](O)CN2CCN(CC2)C[C@H](O)C2=CC=C3C(=O)OCC3=C2C)=C1 OCKGFTQIICXDQW-ZEQRLZLVSA-N 0.000 description 1
OPIFSICVWOWJMJ-AEOCFKNESA-N 5-bromo-4-chloro-3-indolyl beta-D-galactoside Chemical compound O[C@@H]1[C@@H](O)[C@@H](O)[C@@H](CO)O[C@H]1OC1=CNC2=CC=C(Br)C(Cl)=C12 OPIFSICVWOWJMJ-AEOCFKNESA-N 0.000 description 1
QSBYPNXLFMSGKH-UHFFFAOYSA-N 9-Heptadecensaeure Natural products CCCCCCCC=CCCCCCCCC(O)=O QSBYPNXLFMSGKH-UHFFFAOYSA-N 0.000 description 1
206010048998 Acute phase reaction Diseases 0.000 description 1
206010001258 Adenoviral infections Diseases 0.000 description 1
208000031873 Animal Disease Models Diseases 0.000 description 1
108020000948 Antisense Oligonucleotides Proteins 0.000 description 1
108020005544 Antisense RNA Proteins 0.000 description 1
241000894006 Bacteria Species 0.000 description 1
241000283690 Bos taurus Species 0.000 description 1
101100328086 Caenorhabditis elegans cla-1 gene Proteins 0.000 description 1
102000004414 Calcitonin Gene-Related Peptide Human genes 0.000 description 1
241000282465 Canis Species 0.000 description 1
101150044789 Cap gene Proteins 0.000 description 1
108090000565 Capsid Proteins Proteins 0.000 description 1
241001070941 Castanea Species 0.000 description 1
235000014036 Castanea Nutrition 0.000 description 1
102100035882 Catalase Human genes 0.000 description 1
108010053835 Catalase Proteins 0.000 description 1
102100038909 Caveolin-2 Human genes 0.000 description 1
241000282693 Cercopithecidae Species 0.000 description 1
102100023321 Ceruloplasmin Human genes 0.000 description 1
102000007644 Colony-Stimulating Factors Human genes 0.000 description 1
108010071942 Colony-Stimulating Factors Proteins 0.000 description 1
206010011224 Cough Diseases 0.000 description 1
102100031620 Cysteine and glycine-rich protein 3 Human genes 0.000 description 1
241000701022 Cytomegalovirus Species 0.000 description 1
241000450599 DNA viruses Species 0.000 description 1
102100036912 Desmin Human genes 0.000 description 1
108010044052 Desmin Proteins 0.000 description 1
241000196324 Embryophyta Species 0.000 description 1
241000588724 Escherichia coli Species 0.000 description 1
101150081880 FGF1 gene Proteins 0.000 description 1
102000001690 Factor VIII Human genes 0.000 description 1
108010054218 Factor VIII Proteins 0.000 description 1
102000018233 Fibroblast Growth Factor Human genes 0.000 description 1
108050007372 Fibroblast Growth Factor Proteins 0.000 description 1
108700039691 Genetic Promoter Regions Proteins 0.000 description 1
WHUUTDBJXJRKMK-UHFFFAOYSA-N Glutamic acid Natural products OC(=O)C(N)CCC(O)=O WHUUTDBJXJRKMK-UHFFFAOYSA-N 0.000 description 1
102000006587 Glutathione peroxidase Human genes 0.000 description 1
108700016172 Glutathione peroxidases Proteins 0.000 description 1
DHMQDGOQFOQNFH-UHFFFAOYSA-N Glycine Natural products NCC(O)=O DHMQDGOQFOQNFH-UHFFFAOYSA-N 0.000 description 1
239000004471 Glycine Substances 0.000 description 1
101150118046 Gmfb gene Proteins 0.000 description 1
108010017080 Granulocyte Colony-Stimulating Factor Proteins 0.000 description 1
102000004269 Granulocyte Colony-Stimulating Factor Human genes 0.000 description 1
102000004457 Granulocyte-Macrophage Colony-Stimulating Factor Human genes 0.000 description 1
108010017213 Granulocyte-Macrophage Colony-Stimulating Factor Proteins 0.000 description 1
241000696272 Gull adenovirus Species 0.000 description 1
241000282412 Homo Species 0.000 description 1
101000740981 Homo sapiens Caveolin-2 Proteins 0.000 description 1
101000993364 Homo sapiens Ciliary neurotrophic factor Proteins 0.000 description 1
241000598171 Human adenovirus sp. Species 0.000 description 1
241000701044 Human gammaherpesvirus 4 Species 0.000 description 1
UFHFLCQGNIYNRP-UHFFFAOYSA-N Hydrogen Chemical compound [H][H] UFHFLCQGNIYNRP-UHFFFAOYSA-N 0.000 description 1
108090000723 Insulin-Like Growth Factor I Proteins 0.000 description 1
102000004218 Insulin-Like Growth Factor I Human genes 0.000 description 1
108010050904 Interferons Proteins 0.000 description 1
102000014150 Interferons Human genes 0.000 description 1
108010063738 Interleukins Proteins 0.000 description 1
102000015696 Interleukins Human genes 0.000 description 1
102000011782 Keratins Human genes 0.000 description 1
108010076876 Keratins Proteins 0.000 description 1
ONIBWKKTOPOVIA-BYPYZUCNSA-N L-Proline Chemical compound OC(=O)[C@@H]1CCCN1 ONIBWKKTOPOVIA-BYPYZUCNSA-N 0.000 description 1
QNAYBMKLOCPYGJ-REOHCLBHSA-N L-alanine Chemical compound C[C@H](N)C(O)=O QNAYBMKLOCPYGJ-REOHCLBHSA-N 0.000 description 1
WHUUTDBJXJRKMK-VKHMYHEASA-N L-glutamic acid Chemical compound OC(=O)[C@@H](N)CCC(O)=O WHUUTDBJXJRKMK-VKHMYHEASA-N 0.000 description 1
108010046938 Macrophage Colony-Stimulating Factor Proteins 0.000 description 1
102000007651 Macrophage Colony-Stimulating Factor Human genes 0.000 description 1
102000008109 Mixed Function Oxygenases Human genes 0.000 description 1
108010074633 Mixed Function Oxygenases Proteins 0.000 description 1
241000713862 Moloney murine sarcoma virus Species 0.000 description 1
206010028813 Nausea Diseases 0.000 description 1
206010028980 Neoplasm Diseases 0.000 description 1
208000001738 Nervous System Trauma Diseases 0.000 description 1
102000004108 Neurotransmitter Receptors Human genes 0.000 description 1
108090000590 Neurotransmitter Receptors Proteins 0.000 description 1
102000003683 Neurotrophin-4 Human genes 0.000 description 1
ZQPPMHVWECSIRJ-UHFFFAOYSA-N Oleic acid Natural products CCCCCCCCC=CCCCCCCCC(O)=O ZQPPMHVWECSIRJ-UHFFFAOYSA-N 0.000 description 1
239000005642 Oleic acid Substances 0.000 description 1
102000004316 Oxidoreductases Human genes 0.000 description 1
108090000854 Oxidoreductases Proteins 0.000 description 1
229910019142 PO4 Inorganic materials 0.000 description 1
229930040373 Paraformaldehyde Natural products 0.000 description 1
241001494479 Pecora Species 0.000 description 1
102000003992 Peroxidases Human genes 0.000 description 1
NBIIXXVUZAFLBC-UHFFFAOYSA-L Phosphate ion(2-) Chemical compound OP([O-])([O-])=O NBIIXXVUZAFLBC-UHFFFAOYSA-L 0.000 description 1
102000012288 Phosphopyruvate Hydratase Human genes 0.000 description 1
108010022181 Phosphopyruvate Hydratase Proteins 0.000 description 1
ONIBWKKTOPOVIA-UHFFFAOYSA-N Proline Natural products OC(=O)C1CCCN1 ONIBWKKTOPOVIA-UHFFFAOYSA-N 0.000 description 1
108010076504 Protein Sorting Signals Proteins 0.000 description 1
208000032225 Proximal spinal muscular atrophy type 1 Diseases 0.000 description 1
241001112090 Pseudovirus Species 0.000 description 1
108020005067 RNA Splice Sites Proteins 0.000 description 1
108020004511 Recombinant DNA Proteins 0.000 description 1
108020005091 Replication Origin Proteins 0.000 description 1
241000283984 Rodentia Species 0.000 description 1
206010039491 Sarcoma Diseases 0.000 description 1
108010071390 Serum Albumin Proteins 0.000 description 1
102000007562 Serum Albumin Human genes 0.000 description 1
241000700584 Simplexvirus Species 0.000 description 1
101150113275 Smn gene Proteins 0.000 description 1
102000008221 Superoxide Dismutase-1 Human genes 0.000 description 1
108010021188 Superoxide Dismutase-1 Proteins 0.000 description 1
102000004357 Transferases Human genes 0.000 description 1
108090000992 Transferases Proteins 0.000 description 1
241000700618 Vaccinia virus Species 0.000 description 1
241000700647 Variola virus Species 0.000 description 1
102000005789 Vascular Endothelial Growth Factors Human genes 0.000 description 1
108010019530 Vascular Endothelial Growth Factors Proteins 0.000 description 1
108020005202 Viral DNA Proteins 0.000 description 1
SXEHKFHPFVVDIR-UHFFFAOYSA-N [4-(4-hydrazinylphenyl)phenyl]hydrazine Chemical compound C1=CC(NN)=CC=C1C1=CC=C(NN)C=C1 SXEHKFHPFVVDIR-UHFFFAOYSA-N 0.000 description 1
230000009471 action Effects 0.000 description 1
230000003213 activating effect Effects 0.000 description 1
239000004480 active ingredient Substances 0.000 description 1
230000001154 acute effect Effects 0.000 description 1
230000004658 acute-phase response Effects 0.000 description 1
235000004279 alanine Nutrition 0.000 description 1
125000001931 aliphatic group Chemical group 0.000 description 1
150000001408 amides Chemical class 0.000 description 1
125000000539 amino acid group Chemical group 0.000 description 1
102000001307 androgen receptors Human genes 0.000 description 1
108010080146 androgen receptors Proteins 0.000 description 1
238000011558 animal model by disease Methods 0.000 description 1
239000000074 antisense oligonucleotide Substances 0.000 description 1
238000012230 antisense oligonucleotides Methods 0.000 description 1
238000013459 approach Methods 0.000 description 1
125000003118 aryl group Chemical group 0.000 description 1
238000003556 assay Methods 0.000 description 1
206010003549 asthenia Diseases 0.000 description 1
208000025341 autosomal recessive disease Diseases 0.000 description 1
IOJUPLGTWVMSFF-UHFFFAOYSA-N benzothiazole Chemical class C1=CC=C2SC=NC2=C1 IOJUPLGTWVMSFF-UHFFFAOYSA-N 0.000 description 1
WQZGKKKJIJFFOK-VFUOTHLCSA-N beta-D-glucose Chemical compound OC[C@H]1O[C@@H](O)[C@H](O)[C@@H](O)[C@@H]1O WQZGKKKJIJFFOK-VFUOTHLCSA-N 0.000 description 1
230000004071 biological effect Effects 0.000 description 1
230000005540 biological transmission Effects 0.000 description 1
229920001222 biopolymer Polymers 0.000 description 1
230000015572 biosynthetic process Effects 0.000 description 1
230000000903 blocking effect Effects 0.000 description 1
230000008499 blood brain barrier function Effects 0.000 description 1
210000001218 blood-brain barrier Anatomy 0.000 description 1
210000005013 brain tissue Anatomy 0.000 description 1
AIYUHDOJVYHVIT-UHFFFAOYSA-M caesium chloride Chemical compound [Cl-].[Cs+] AIYUHDOJVYHVIT-UHFFFAOYSA-M 0.000 description 1
229960004015 calcitonin Drugs 0.000 description 1
239000011575 calcium Substances 0.000 description 1
229910052791 calcium Inorganic materials 0.000 description 1
239000001110 calcium chloride Substances 0.000 description 1
229910001628 calcium chloride Inorganic materials 0.000 description 1
239000001506 calcium phosphate Substances 0.000 description 1
229910000389 calcium phosphate Inorganic materials 0.000 description 1
235000011010 calcium phosphates Nutrition 0.000 description 1
201000011510 cancer Diseases 0.000 description 1
238000004113 cell culture Methods 0.000 description 1
230000024245 cell differentiation Effects 0.000 description 1
230000011748 cell maturation Effects 0.000 description 1
238000005119 centrifugation Methods 0.000 description 1
230000008859 change Effects 0.000 description 1
238000012512 characterization method Methods 0.000 description 1
208000027746 childhood spinal muscular atrophy Diseases 0.000 description 1
238000010367 cloning Methods 0.000 description 1
238000011260 co-administration Methods 0.000 description 1
230000001447 compensatory effect Effects 0.000 description 1
230000000295 complement effect Effects 0.000 description 1
239000003184 complementary RNA Substances 0.000 description 1
229910052802 copper Inorganic materials 0.000 description 1
230000002596 correlated effect Effects 0.000 description 1
239000012228 culture supernatant Substances 0.000 description 1
230000007547 defect Effects 0.000 description 1
230000007812 deficiency Effects 0.000 description 1
230000006735 deficit Effects 0.000 description 1
230000003412 degenerative effect Effects 0.000 description 1
210000005045 desmin Anatomy 0.000 description 1
239000008121 dextrose Substances 0.000 description 1
235000014113 dietary fatty acids Nutrition 0.000 description 1
238000012137 double-staining Methods 0.000 description 1
239000003651 drinking water Substances 0.000 description 1
235000020188 drinking water Nutrition 0.000 description 1
230000013020 embryo development Effects 0.000 description 1
230000002708 enhancing effect Effects 0.000 description 1
230000009144 enzymatic modification Effects 0.000 description 1
238000011156 evaluation Methods 0.000 description 1
229960000301 factor viii Drugs 0.000 description 1
239000000194 fatty acid Substances 0.000 description 1
229930195729 fatty acid Natural products 0.000 description 1
150000004665 fatty acids Chemical class 0.000 description 1
229940126864 fibroblast growth factor Drugs 0.000 description 1
239000000945 filler Substances 0.000 description 1
101150098622 gag gene Proteins 0.000 description 1
238000007429 general method Methods 0.000 description 1
230000002068 genetic effect Effects 0.000 description 1
230000002518 glial effect Effects 0.000 description 1
239000008103 glucose Substances 0.000 description 1
235000013922 glutamic acid Nutrition 0.000 description 1
239000004220 glutamic acid Substances 0.000 description 1
125000003630 glycyl group Chemical group [H]N([H])C([H])([H])C(*)=O 0.000 description 1
239000008187 granular material Substances 0.000 description 1
108091008039 hormone receptors Proteins 0.000 description 1
125000002887 hydroxy group Chemical group [H]O* 0.000 description 1
210000000987 immune system Anatomy 0.000 description 1
238000002649 immunization Methods 0.000 description 1
230000003053 immunization Effects 0.000 description 1
238000003364 immunohistochemistry Methods 0.000 description 1
230000006872 improvement Effects 0.000 description 1
238000007901 in situ hybridization Methods 0.000 description 1
238000011065 in-situ storage Methods 0.000 description 1
238000010348 incorporation Methods 0.000 description 1
230000002757 inflammatory effect Effects 0.000 description 1
239000003112 inhibitor Substances 0.000 description 1
230000002401 inhibitory effect Effects 0.000 description 1
230000005764 inhibitory process Effects 0.000 description 1
229940102223 injectable solution Drugs 0.000 description 1
230000002608 insulinlike Effects 0.000 description 1
229940047124 interferons Drugs 0.000 description 1
229940047122 interleukins Drugs 0.000 description 1
210000003963 intermediate filament Anatomy 0.000 description 1
238000007913 intrathecal administration Methods 0.000 description 1
238000002955 isolation Methods 0.000 description 1
QXJSBBXBKPUZAA-UHFFFAOYSA-N isooleic acid Natural products CCCCCCCC=CCCCCCCCCC(O)=O QXJSBBXBKPUZAA-UHFFFAOYSA-N 0.000 description 1
239000000644 isotonic solution Substances 0.000 description 1
210000003292 kidney cell Anatomy 0.000 description 1
231100000225 lethality Toxicity 0.000 description 1
150000002632 lipids Chemical class 0.000 description 1
210000004185 liver Anatomy 0.000 description 1
230000004807 localization Effects 0.000 description 1
230000033001 locomotion Effects 0.000 description 1
210000004072 lung Anatomy 0.000 description 1
229910001629 magnesium chloride Inorganic materials 0.000 description 1
159000000003 magnesium salts Chemical class 0.000 description 1
210000001161 mammalian embryo Anatomy 0.000 description 1
210000000274 microglia Anatomy 0.000 description 1
230000002438 mitochondrial effect Effects 0.000 description 1
238000010369 molecular cloning Methods 0.000 description 1
XONPDZSGENTBNJ-UHFFFAOYSA-N molecular hydrogen;sodium Chemical compound [Na].[H][H] XONPDZSGENTBNJ-UHFFFAOYSA-N 0.000 description 1
238000012544 monitoring process Methods 0.000 description 1
125000000896 monocarboxylic acid group Chemical group 0.000 description 1
230000007659 motor function Effects 0.000 description 1
210000000663 muscle cell Anatomy 0.000 description 1
210000004699 muscle spindle Anatomy 0.000 description 1
108091008709 muscle spindles Proteins 0.000 description 1
201000000585 muscular atrophy Diseases 0.000 description 1
239000003471 mutagenic agent Substances 0.000 description 1
230000008693 nausea Effects 0.000 description 1
230000032405 negative regulation of neuron apoptotic process Effects 0.000 description 1
230000007383 nerve stimulation Effects 0.000 description 1
208000028412 nervous system injury Diseases 0.000 description 1
230000001537 neural effect Effects 0.000 description 1
210000005044 neurofilament Anatomy 0.000 description 1
230000001272 neurogenic effect Effects 0.000 description 1
210000004498 neuroglial cell Anatomy 0.000 description 1
230000000926 neurological effect Effects 0.000 description 1
230000004693 neuron damage Effects 0.000 description 1
230000000324 neuroprotective effect Effects 0.000 description 1
231100000252 nontoxic Toxicity 0.000 description 1
230000003000 nontoxic effect Effects 0.000 description 1
239000000346 nonvolatile oil Substances 0.000 description 1
239000003921 oil Substances 0.000 description 1
ZQPPMHVWECSIRJ-KTKRTIGZSA-N oleic acid Chemical compound CCCCCCCC\C=C/CCCCCCCC(O)=O ZQPPMHVWECSIRJ-KTKRTIGZSA-N 0.000 description 1
229920002866 paraformaldehyde Polymers 0.000 description 1
230000001991 pathophysiological effect Effects 0.000 description 1
230000037361 pathway Effects 0.000 description 1
230000035515 penetration Effects 0.000 description 1
230000010412 perfusion Effects 0.000 description 1
230000000737 periodic effect Effects 0.000 description 1
210000000578 peripheral nerve Anatomy 0.000 description 1
229940021222 peritoneal dialysis isotonic solution Drugs 0.000 description 1
108040007629 peroxidase activity proteins Proteins 0.000 description 1
239000008194 pharmaceutical composition Substances 0.000 description 1
239000000825 pharmaceutical preparation Substances 0.000 description 1
239000010452 phosphate Substances 0.000 description 1
NBIIXXVUZAFLBC-UHFFFAOYSA-K phosphate Chemical compound [O-]P([O-])([O-])=O NBIIXXVUZAFLBC-UHFFFAOYSA-K 0.000 description 1
230000001766 physiological effect Effects 0.000 description 1
230000008288 physiological mechanism Effects 0.000 description 1
101150088856 pix gene Proteins 0.000 description 1
238000013492 plasmid preparation Methods 0.000 description 1
230000008488 polyadenylation Effects 0.000 description 1
102000040430 polynucleotide Human genes 0.000 description 1
108091033319 polynucleotide Proteins 0.000 description 1
239000002157 polynucleotide Substances 0.000 description 1
239000011591 potassium Substances 0.000 description 1
229910052700 potassium Inorganic materials 0.000 description 1
239000001103 potassium chloride Substances 0.000 description 1
WCUXLLCKKVVCTQ-UHFFFAOYSA-M potassium chloride Inorganic materials [Cl-].[K+] WCUXLLCKKVVCTQ-UHFFFAOYSA-M 0.000 description 1
230000003389 potentiating effect Effects 0.000 description 1
238000004321 preservation Methods 0.000 description 1
238000004393 prognosis Methods 0.000 description 1
230000001681 protective effect Effects 0.000 description 1
208000022074 proximal spinal muscular atrophy Diseases 0.000 description 1
238000000746 purification Methods 0.000 description 1
238000011552 rat model Methods 0.000 description 1
108020003175 receptors Proteins 0.000 description 1
102000005962 receptors Human genes 0.000 description 1
230000006798 recombination Effects 0.000 description 1
238000005215 recombination Methods 0.000 description 1
238000011084 recovery Methods 0.000 description 1
230000007832 reinnervation Effects 0.000 description 1
101150066583 rep gene Proteins 0.000 description 1
230000003362 replicative effect Effects 0.000 description 1
230000000241 respiratory effect Effects 0.000 description 1
230000004202 respiratory function Effects 0.000 description 1
210000003019 respiratory muscle Anatomy 0.000 description 1
108090000064 retinoic acid receptors Proteins 0.000 description 1
102000003702 retinoic acid receptors Human genes 0.000 description 1
230000002441 reversible effect Effects 0.000 description 1
229940072169 rilutek Drugs 0.000 description 1
210000000954 sacrococcygeal region Anatomy 0.000 description 1
150000003839 salts Chemical class 0.000 description 1
210000004116 schwann cell Anatomy 0.000 description 1
230000003248 secreting effect Effects 0.000 description 1
230000001953 sensory effect Effects 0.000 description 1
210000002966 serum Anatomy 0.000 description 1
230000009528 severe injury Effects 0.000 description 1
229910052709 silver Inorganic materials 0.000 description 1
239000004332 silver Substances 0.000 description 1
241000894007 species Species 0.000 description 1
210000005070 sphincter Anatomy 0.000 description 1
208000020431 spinal cord injury Diseases 0.000 description 1
210000000952 spleen Anatomy 0.000 description 1
239000007921 spray Substances 0.000 description 1
108020003113 steroid hormone receptors Proteins 0.000 description 1
102000005969 steroid hormone receptors Human genes 0.000 description 1
230000004936 stimulating effect Effects 0.000 description 1
238000007920 subcutaneous administration Methods 0.000 description 1
125000004434 sulfur atom Chemical group 0.000 description 1
230000001629 suppression Effects 0.000 description 1
239000000375 suspending agent Substances 0.000 description 1
210000000225 synapse Anatomy 0.000 description 1
230000000946 synaptic effect Effects 0.000 description 1
101150065190 term gene Proteins 0.000 description 1
229940124597 therapeutic agent Drugs 0.000 description 1
231100000419 toxicity Toxicity 0.000 description 1
230000001988 toxicity Effects 0.000 description 1
238000001890 transfection Methods 0.000 description 1
238000012301 transgenic model Methods 0.000 description 1
230000001052 transient effect Effects 0.000 description 1
238000013519 translation Methods 0.000 description 1
QORWJWZARLRLPR-UHFFFAOYSA-H tricalcium bis(phosphate) Chemical compound [Ca+2].[Ca+2].[Ca+2].[O-]P([O-])([O-])=O.[O-]P([O-])([O-])=O QORWJWZARLRLPR-UHFFFAOYSA-H 0.000 description 1
208000032471 type 1 spinal muscular atrophy Diseases 0.000 description 1
241000701447 unidentified baculovirus Species 0.000 description 1
210000003934 vacuole Anatomy 0.000 description 1
230000004580 weight loss Effects 0.000 description 1
208000016261 weight loss Diseases 0.000 description 1
238000009736 wetting Methods 0.000 description 1
239000000080 wetting agent Substances 0.000 description 1
229910052725 zinc Inorganic materials 0.000 description 1

Classifications

- C—CHEMISTRY; METALLURGY
- C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
- C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
- C12N15/00—Mutation or genetic engineering; DNA or RNA concerning genetic engineering, vectors, e.g. plasmids, or their isolation, preparation or purification; Use of hosts therefor
- C12N15/09—Recombinant DNA-technology
- C12N15/63—Introduction of foreign genetic material using vectors; Vectors; Use of hosts therefor; Regulation of expression
- C12N15/79—Vectors or expression systems specially adapted for eukaryotic hosts
- C12N15/85—Vectors or expression systems specially adapted for eukaryotic hosts for animal cells
- C12N15/86—Viral vectors
- A—HUMAN NECESSITIES
- A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
- A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
- A61K38/00—Medicinal preparations containing peptides
- A61K38/16—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof
- A61K38/17—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from animals; from humans
- A61K38/18—Growth factors; Growth regulators
- A61K38/185—Nerve growth factor [NGF]; Brain derived neurotrophic factor [BDNF]; Ciliary neurotrophic factor [CNTF]; Glial derived neurotrophic factor [GDNF]; Neurotrophins, e.g. NT-3
- A—HUMAN NECESSITIES
- A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
- A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
- A61K48/00—Medicinal preparations containing genetic material which is inserted into cells of the living body to treat genetic diseases; Gene therapy
- A—HUMAN NECESSITIES
- A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
- A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
- A61P17/00—Drugs for dermatological disorders
- A61P17/02—Drugs for dermatological disorders for treating wounds, ulcers, burns, scars, keloids, or the like
- A—HUMAN NECESSITIES
- A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
- A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
- A61P21/00—Drugs for disorders of the muscular or neuromuscular system
- A—HUMAN NECESSITIES
- A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
- A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
- A61P25/00—Drugs for disorders of the nervous system
- A61P25/02—Drugs for disorders of the nervous system for peripheral neuropathies
- C—CHEMISTRY; METALLURGY
- C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
- C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
- C12N2750/00—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA ssDNA viruses
- C12N2750/00011—Details
- C12N2750/14011—Parvoviridae
- C12N2750/14111—Dependovirus, e.g. adenoassociated viruses
- C12N2750/14141—Use of virus, viral particle or viral elements as a vector
- C12N2750/14143—Use of virus, viral particle or viral elements as a vector viral genome or elements thereof as genetic vector

Landscapes

Health & Medical Sciences (AREA)
Life Sciences & Earth Sciences (AREA)
Engineering & Computer Science (AREA)
Chemical & Material Sciences (AREA)
Genetics & Genomics (AREA)
General Health & Medical Sciences (AREA)
Bioinformatics & Cheminformatics (AREA)
Medicinal Chemistry (AREA)
Animal Behavior & Ethology (AREA)
Veterinary Medicine (AREA)
Public Health (AREA)
Organic Chemistry (AREA)
Pharmacology & Pharmacy (AREA)
Biomedical Technology (AREA)
Zoology (AREA)
Biotechnology (AREA)
Wood Science & Technology (AREA)
Neurology (AREA)
Epidemiology (AREA)
Molecular Biology (AREA)
General Engineering & Computer Science (AREA)
Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
General Chemical & Material Sciences (AREA)
Neurosurgery (AREA)
Chemical Kinetics & Catalysis (AREA)
Gastroenterology & Hepatology (AREA)
Biochemistry (AREA)
Physics & Mathematics (AREA)
Plant Pathology (AREA)
Microbiology (AREA)
Proteomics, Peptides & Aminoacids (AREA)
Biophysics (AREA)
Immunology (AREA)
Psychology (AREA)
Virology (AREA)
Physical Education & Sports Medicine (AREA)
Dermatology (AREA)
Orthopedic Medicine & Surgery (AREA)
Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
Medicines Containing Material From Animals Or Micro-Organisms (AREA)

SK955-99A 1997-01-17 1998-01-16 Adenoviral-vector-mediated gene transfer into medullary motor neurons SK95599A3 (en)

Applications Claiming Priority (3)

Application Number	Priority Date	Filing Date	Title
US08/785,074 US6632427B1 (en)	1994-12-13	1997-01-17	Adenoviral-vector-mediated gene transfer into medullary motor neurons
US4224797P	1997-03-31	1997-03-31
PCT/EP1998/000220 WO1998031395A1 (fr)	1997-01-17	1998-01-16	Adenoviral-vector-mediated gene transfer into medullary motor neurons

Publications (1)

Publication Number	Publication Date
SK95599A3 true SK95599A3 (en)	2000-05-16

Family

ID=26719029

Family Applications (1)

Application Number	Title	Priority Date	Filing Date
SK955-99A SK95599A3 (en)	1997-01-17	1998-01-16	Adenoviral-vector-mediated gene transfer into medullary motor neurons

Country Status (11)

Country	Link
US (1)	US6552003B2 (fr)
EP (1)	EP0969875B1 (fr)
JP (1)	JP2002516607A (fr)
AU (1)	AU6614398A (fr)
BR (1)	BR9806912A (fr)
CA (1)	CA2277869A1 (fr)
CZ (1)	CZ250499A3 (fr)
IL (1)	IL130852A0 (fr)
NO (1)	NO993465L (fr)
SK (1)	SK95599A3 (fr)
WO (1)	WO1998031395A1 (fr)

Families Citing this family (7)

* Cited by examiner, † Cited by third party
Publication number	Priority date	Publication date	Assignee	Title
DK2295070T3 (en)	2000-04-12	2015-11-16	Vib Vzw	Use of VEGF and homologs for the treatment of neuronal diseases
US20050032219A1 (en) *	2001-07-03	2005-02-10	Patrick Aubourg	Methods of administering vectors to synaptically connected neurons
CA2469658A1 (fr) *	2001-12-19	2003-07-03	Lijun Wang	Administration de gdnf induite par le virus adeno-associe dans des muscles du squelette
US20050234000A1 (en) *	2003-12-12	2005-10-20	Mitchell Gordon S	SiRNA delivery into mammalian nerve cells
PL1755647T3 (pl)	2004-05-27	2011-01-31	Vib Vzw	Leczenie stwardnienia zanikowego bocznego
US9078808B2 (en) *	2009-03-26	2015-07-14	Warsaw Orthopedic, Inc.	Device to deliver magnesium in PEG formulation
WO2021163274A1 (fr) *	2020-02-11	2021-08-19	The General Hospital Corporation	Méthodes de traitement de la sclérose latérale amyotrophique

Family Cites Families (5)

* Cited by examiner, † Cited by third party
Publication number	Priority date	Publication date	Assignee	Title
US5703055A (en) *	1989-03-21	1997-12-30	Wisconsin Alumni Research Foundation	Generation of antibodies through lipid mediated DNA delivery
GR1000980B (el) *	1989-08-30	1993-03-31	Max Planck Gesellschaft	Νευροτροφινη-3,ενας νεος neυροτροφικος παραγοντας που σχετιζεται με τον παραγοντα αναπτυξης νευρων και τον νευροτροφικο παραγοντα που προερχεται απο τον εγκεφαλο.
US5849571A (en) *	1990-10-10	1998-12-15	University Of Pittsburgh Of The Commonwealth System Of Higher Education	Latency active herpes virus promoters and their use
FR2727867B1 (fr) *	1994-12-13	1997-01-31	Rhone Poulenc Rorer Sa	Transfert de genes dans les motoneurones medullaires au moyen de vecteurs adenoviraux
WO1997039629A1 (fr) *	1996-04-25	1997-10-30	Genetic Therapy, Inc.	Vecteurs viraux incluant des polynucleotides codant des facteurs neurotrophiques ainsi que leur utilisation

1998
- 1998-01-16 EP EP98907949A patent/EP0969875B1/fr not_active Expired - Lifetime
- 1998-01-16 IL IL13085298A patent/IL130852A0/xx unknown
- 1998-01-16 WO PCT/EP1998/000220 patent/WO1998031395A1/fr not_active Application Discontinuation
- 1998-01-16 JP JP53230898A patent/JP2002516607A/ja active Pending
- 1998-01-16 SK SK955-99A patent/SK95599A3/sk unknown
- 1998-01-16 BR BR9806912-8A patent/BR9806912A/pt not_active Application Discontinuation
- 1998-01-16 AU AU66143/98A patent/AU6614398A/en not_active Abandoned
- 1998-01-16 CZ CZ992504A patent/CZ250499A3/cs unknown
- 1998-01-16 CA CA002277869A patent/CA2277869A1/fr not_active Abandoned
1999
- 1999-07-14 NO NO993465A patent/NO993465L/no not_active Application Discontinuation
- 1999-07-16 US US09/356,032 patent/US6552003B2/en not_active Expired - Lifetime

Also Published As

Publication number	Publication date
BR9806912A (pt)	2000-05-16
EP0969875A1 (fr)	2000-01-12
US20020164303A1 (en)	2002-11-07
NO993465D0 (no)	1999-07-14
EP0969875B1 (fr)	2008-11-26
WO1998031395A1 (fr)	1998-07-23
IL130852A0 (en)	2001-01-28
JP2002516607A (ja)	2002-06-04
CZ250499A3 (cs)	1999-11-17
NO993465L (no)	1999-09-09
CA2277869A1 (fr)	1998-07-23
AU6614398A (en)	1998-08-07
US6552003B2 (en)	2003-04-22

Publication	Publication Date	Title
US6632427B1 (en)	2003-10-14	Adenoviral-vector-mediated gene transfer into medullary motor neurons
US8486385B2 (en)	2013-07-16	Methods for therapy of neurodegenerative disease of the brain
Haase et al.	1998	Adenovirus-mediated transfer of the neurotrophin-3 gene into skeletal muscle of pmn mice: therapeutic effects and mechanisms of action
US6683058B1 (en)	2004-01-27	Methods for therapy of neurodegenerative disease of the brain
US7244423B2 (en)	2007-07-17	Methods for therapy of neurodegenerative disease of the brain
US20070037768A1 (en)	2007-02-15	Methods for therapy of neurodegenerative disease of the brain
AU2001261758A1 (en)	2002-05-02	Methods for therapy of neurodegenerative disease of the brain
SK95599A3 (en)	2000-05-16	Adenoviral-vector-mediated gene transfer into medullary motor neurons
US6167888B1 (en)	2001-01-02	Method for inducing partial recovery of lost voluntary motor function after spinal cord injury in a mammal
AU2011235890B2 (en)	2014-07-31	Methods for treating Parkinson's disease and other disorders of dopaminergic neurons of the brain
CN1155706C (zh)	2004-06-30	肌萎缩性侧索硬化的治疗方法
AU2928202A (en)	2002-05-23	Adenoviral-vector-mediated gene transfer into medullary motor neurons
KR20000070272A (ko)	2000-11-25	수질 운동 뉴런 중으로의 아데노바이러스 벡터 매개-유전자전달방법
US20010043920A1 (en)	2001-11-22	Methods for modulation of the effects of aging on the primate brain
MXPA99000533A (en)	1999-10-14	Device for mixing an audio sequence with a video sequence