AR061282A1 - Terapia genica para esclerosis lateral amiotrofica y otros trastornos del cordon espinal - Google Patents
Terapia genica para esclerosis lateral amiotrofica y otros trastornos del cordon espinalInfo
- Publication number
- AR061282A1 AR061282A1 ARP070102460A ARP070102460A AR061282A1 AR 061282 A1 AR061282 A1 AR 061282A1 AR P070102460 A ARP070102460 A AR P070102460A AR P070102460 A ARP070102460 A AR P070102460A AR 061282 A1 AR061282 A1 AR 061282A1
- Authority
- AR
- Argentina
- Prior art keywords
- transgene
- smn
- ventricle
- subject
- group
- Prior art date
Links
- 210000000278 spinal cord Anatomy 0.000 title abstract 2
- 206010002026 amyotrophic lateral sclerosis Diseases 0.000 title 1
- 208000037265 diseases, disorders, signs and symptoms Diseases 0.000 title 1
- 238000001415 gene therapy Methods 0.000 title 1
- 108700019146 Transgenes Proteins 0.000 abstract 5
- 102000003951 Erythropoietin Human genes 0.000 abstract 4
- 108090000394 Erythropoietin Proteins 0.000 abstract 4
- 229940105423 erythropoietin Drugs 0.000 abstract 4
- NOESYZHRGYRDHS-UHFFFAOYSA-N insulin Chemical compound N1C(=O)C(NC(=O)C(CCC(N)=O)NC(=O)C(CCC(O)=O)NC(=O)C(C(C)C)NC(=O)C(NC(=O)CN)C(C)CC)CSSCC(C(NC(CO)C(=O)NC(CC(C)C)C(=O)NC(CC=2C=CC(O)=CC=2)C(=O)NC(CCC(N)=O)C(=O)NC(CC(C)C)C(=O)NC(CCC(O)=O)C(=O)NC(CC(N)=O)C(=O)NC(CC=2C=CC(O)=CC=2)C(=O)NC(CSSCC(NC(=O)C(C(C)C)NC(=O)C(CC(C)C)NC(=O)C(CC=2C=CC(O)=CC=2)NC(=O)C(CC(C)C)NC(=O)C(C)NC(=O)C(CCC(O)=O)NC(=O)C(C(C)C)NC(=O)C(CC(C)C)NC(=O)C(CC=2NC=NC=2)NC(=O)C(CO)NC(=O)CNC2=O)C(=O)NCC(=O)NC(CCC(O)=O)C(=O)NC(CCCNC(N)=N)C(=O)NCC(=O)NC(CC=3C=CC=CC=3)C(=O)NC(CC=3C=CC=CC=3)C(=O)NC(CC=3C=CC(O)=CC=3)C(=O)NC(C(C)O)C(=O)N3C(CCC3)C(=O)NC(CCCCN)C(=O)NC(C)C(O)=O)C(=O)NC(CC(N)=O)C(O)=O)=O)NC(=O)C(C(C)CC)NC(=O)C(CO)NC(=O)C(C(C)O)NC(=O)C1CSSCC2NC(=O)C(CC(C)C)NC(=O)C(NC(=O)C(CCC(N)=O)NC(=O)C(CC(N)=O)NC(=O)C(NC(=O)C(N)CC=1C=CC=CC=1)C(C)C)CC1=CN=CN1 NOESYZHRGYRDHS-UHFFFAOYSA-N 0.000 abstract 4
- OXCMYAYHXIHQOA-UHFFFAOYSA-N potassium;[2-butyl-5-chloro-3-[[4-[2-(1,2,4-triaza-3-azanidacyclopenta-1,4-dien-5-yl)phenyl]phenyl]methyl]imidazol-4-yl]methanol Chemical compound [K+].CCCCC1=NC(Cl)=C(CO)N1CC1=CC=C(C=2C(=CC=CC=2)C2=N[N-]N=N2)C=C1 OXCMYAYHXIHQOA-UHFFFAOYSA-N 0.000 abstract 4
- 230000000508 neurotrophic effect Effects 0.000 abstract 3
- 102000004169 proteins and genes Human genes 0.000 abstract 3
- 108090000623 proteins and genes Proteins 0.000 abstract 3
- 230000009261 transgenic effect Effects 0.000 abstract 3
- 239000013603 viral vector Substances 0.000 abstract 3
- 102100021851 Calbindin Human genes 0.000 abstract 2
- 102000003693 Hedgehog Proteins Human genes 0.000 abstract 2
- 108090000031 Hedgehog Proteins Proteins 0.000 abstract 2
- 102100022875 Hypoxia-inducible factor 1-alpha Human genes 0.000 abstract 2
- 108050009527 Hypoxia-inducible factor-1 alpha Proteins 0.000 abstract 2
- 102000004877 Insulin Human genes 0.000 abstract 2
- 108090001061 Insulin Proteins 0.000 abstract 2
- 102000001675 Parvalbumin Human genes 0.000 abstract 2
- 108060005874 Parvalbumin Proteins 0.000 abstract 2
- 102000004669 Protein-Lysine 6-Oxidase Human genes 0.000 abstract 2
- 108010003894 Protein-Lysine 6-Oxidase Proteins 0.000 abstract 2
- -1 SIRT-2 Proteins 0.000 abstract 2
- 102000005789 Vascular Endothelial Growth Factors Human genes 0.000 abstract 2
- 108010019530 Vascular Endothelial Growth Factors Proteins 0.000 abstract 2
- 210000004556 brain Anatomy 0.000 abstract 2
- 108060001061 calbindin Proteins 0.000 abstract 2
- 239000003102 growth factor Substances 0.000 abstract 2
- 229940125396 insulin Drugs 0.000 abstract 2
- 239000003550 marker Substances 0.000 abstract 2
- 102100022987 Angiogenin Human genes 0.000 abstract 1
- 206010003591 Ataxia Diseases 0.000 abstract 1
- 208000029402 Bulbospinal muscular atrophy Diseases 0.000 abstract 1
- 102100021809 Chorionic somatomammotropin hormone 1 Human genes 0.000 abstract 1
- 108010005939 Ciliary Neurotrophic Factor Proteins 0.000 abstract 1
- 102100031614 Ciliary neurotrophic factor Human genes 0.000 abstract 1
- 102000012004 Ghrelin Human genes 0.000 abstract 1
- 101800001586 Ghrelin Proteins 0.000 abstract 1
- 208000027747 Kennedy disease Diseases 0.000 abstract 1
- 108010003044 Placental Lactogen Proteins 0.000 abstract 1
- 239000000381 Placental Lactogen Substances 0.000 abstract 1
- 102100024819 Prolactin Human genes 0.000 abstract 1
- 108010057464 Prolactin Proteins 0.000 abstract 1
- 208000034189 Sclerosis Diseases 0.000 abstract 1
- 108010072788 angiogenin Proteins 0.000 abstract 1
- 238000003556 assay Methods 0.000 abstract 1
- 210000003169 central nervous system Anatomy 0.000 abstract 1
- 230000002490 cerebral effect Effects 0.000 abstract 1
- 230000001886 ciliary effect Effects 0.000 abstract 1
- 210000004055 fourth ventricle Anatomy 0.000 abstract 1
- GNKDKYIHGQKHHM-RJKLHVOGSA-N ghrelin Chemical compound C([C@H](NC(=O)[C@@H](NC(=O)[C@H](CO)NC(=O)CN)COC(=O)CCCCCCC)C(=O)N[C@@H](CC(C)C)C(=O)N[C@@H](CO)C(=O)N1[C@@H](CCC1)C(=O)N[C@@H](CCC(O)=O)C(=O)N[C@@H](CC=1N=CNC=1)C(=O)N[C@@H](CCC(N)=O)C(=O)N[C@@H](CCCNC(N)=N)C(=O)N[C@@H](C(C)C)C(=O)N[C@@H](CCC(N)=O)C(=O)N[C@@H](CCC(N)=O)C(=O)N[C@@H](CCCNC(N)=N)C(=O)N[C@@H](CCCCN)C(=O)N[C@@H](CCC(O)=O)C(=O)N[C@@H](CO)C(=O)N[C@@H](CCCCN)C(=O)N[C@@H](CCCCN)C(=O)N1[C@@H](CCC1)C(=O)N1[C@@H](CCC1)C(=O)N[C@@H](C)C(=O)N[C@@H](CCCCN)C(=O)N[C@@H](CC(C)C)C(=O)N[C@@H](CCC(N)=O)C(=O)N1[C@@H](CCC1)C(=O)N[C@@H](CCCNC(N)=N)C(O)=O)C1=CC=CC=C1 GNKDKYIHGQKHHM-RJKLHVOGSA-N 0.000 abstract 1
- 210000003140 lateral ventricle Anatomy 0.000 abstract 1
- 229940097325 prolactin Drugs 0.000 abstract 1
- 208000020431 spinal cord injury Diseases 0.000 abstract 1
- 208000002320 spinal muscular atrophy Diseases 0.000 abstract 1
- 230000000472 traumatic effect Effects 0.000 abstract 1
- 239000013598 vector Substances 0.000 abstract 1
Classifications
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- 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
- A61K38/22—Hormones
- A61K38/30—Insulin-like growth factors, i.e. somatomedins, e.g. IGF-1, IGF-2
-
- 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
- A61K48/005—Medicinal preparations containing genetic material which is inserted into cells of the living body to treat genetic diseases; Gene therapy characterised by an aspect of the 'active' part of the composition delivered, i.e. the nucleic acid delivered
-
- 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
- A61K48/0075—Medicinal preparations containing genetic material which is inserted into cells of the living body to treat genetic diseases; Gene therapy characterised by an aspect of the delivery route, e.g. oral, subcutaneous
-
- 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
- A61P21/00—Drugs for disorders of the muscular or neuromuscular system
- A61P21/02—Muscle relaxants, e.g. for tetanus or cramps
-
- 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
-
- 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
-
- 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
- A61P25/28—Drugs for disorders of the nervous system for treating neurodegenerative disorders of the central nervous system, e.g. nootropic agents, cognition enhancers, drugs for treating Alzheimer's disease or other forms of dementia
-
- A—HUMAN NECESSITIES
- A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
- A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
- A61P43/00—Drugs for specific purposes, not provided for in groups A61P1/00-A61P41/00
-
- 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
-
- 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
- C12N15/864—Parvoviral vectors, e.g. parvovirus, densovirus
- C12N15/8645—Adeno-associated virus
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- 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
-
- 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
- C12N2799/00—Uses of viruses
- C12N2799/02—Uses of viruses as vector
- C12N2799/021—Uses of viruses as vector for the expression of a heterologous nucleic acid
- C12N2799/025—Uses of viruses as vector for the expression of a heterologous nucleic acid where the vector is derived from a parvovirus
Landscapes
- Health & Medical Sciences (AREA)
- Life Sciences & Earth Sciences (AREA)
- Engineering & Computer Science (AREA)
- Animal Behavior & Ethology (AREA)
- Chemical & Material Sciences (AREA)
- Veterinary Medicine (AREA)
- Medicinal Chemistry (AREA)
- Public Health (AREA)
- General Health & Medical Sciences (AREA)
- Pharmacology & Pharmacy (AREA)
- Bioinformatics & Cheminformatics (AREA)
- General Chemical & Material Sciences (AREA)
- Organic Chemistry (AREA)
- Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
- Neurology (AREA)
- Chemical Kinetics & Catalysis (AREA)
- Epidemiology (AREA)
- Molecular Biology (AREA)
- Biotechnology (AREA)
- Genetics & Genomics (AREA)
- Neurosurgery (AREA)
- Biomedical Technology (AREA)
- Physical Education & Sports Medicine (AREA)
- Orthopedic Medicine & Surgery (AREA)
- Gastroenterology & Hepatology (AREA)
- Proteomics, Peptides & Aminoacids (AREA)
- Immunology (AREA)
- Diabetes (AREA)
- Endocrinology (AREA)
- Zoology (AREA)
- Hospice & Palliative Care (AREA)
- Psychiatry (AREA)
- Pain & Pain Management (AREA)
- Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
- Medicines Containing Material From Animals Or Micro-Organisms (AREA)
- Medicines Containing Antibodies Or Antigens For Use As Internal Diagnostic Agents (AREA)
- Measuring Or Testing Involving Enzymes Or Micro-Organisms (AREA)
- Peptides Or Proteins (AREA)
- Preparation Of Compounds By Using Micro-Organisms (AREA)
Abstract
Un producto transgénico para administrar a un ventrículo del cerebro de un sujeto, que comprende: un vector viral neurotrofico recombinante que comprende dicho transgen, donde en la administracion al ventrículo del cerebro, que preferentemente se selecciona del grupo que consiste de un ventrículo lateral y el cuarto ventrículo, dicho transgen está expresado y el producto proteico expresado se suministra al cordon espinal. Preferentemente, el vector viral es un vector VAA o VAA4, y transgen es IGF-1. Dicho producto transgénico es para ser administrado a un sujeto que tiene esclorosis amiotrofica lateral. El transgen del producto transgénico puede seleccionarse del grupo que consiste en factor de crecimiento de insulina 1 (IGF-1), calbindina D28, parvalbumina, HIF1-alfa, SIRT-2, VEGF, SMN-1, SMN-2, FNTC (factor neurotrofico ciliar), erizo sonico (shh), eritropoyetina (EPO), lisil oxidasa (LOX), progranulina, prolactina, grelina, neuroserpina, angiogenina, y lactogena placentaria. El sujeto a tratar puede tener una condicion seleccionada del grupo que consiste en esclerosis amitrofica lateral (ALS), atrofia muscular bulbar espinal, ataxia cerebelar espinal, atrofia muscular espinal, y lesion traumática del cordon espinal; y subexpresa una cantidad efectiva de una proteína seleccionada del grupo que consiste en factor de crecimiento de insulina 1 (IGF-1), calbindina D28, parvalbumina, HIF1-alfa, erizo sonico (shh), eritropoyetina (EPO), SIRT-2, VEGF, SMN-1, SMN-2, y FNTC (factor neurotrofico ciliar). Un sistema de ensayo para detectar la perdida de la funcion del sistema nervioso central de un sujeto, que comprende: una cantidad diagnosticamente efectiva de un vector viral neurotrofico que contiene un transgen que codifica un marcador detectable, donde el marcador detectable es proteína verde fluorescente.
Applications Claiming Priority (1)
| Application Number | Priority Date | Filing Date | Title |
|---|---|---|---|
| US81141906P | 2006-06-07 | 2006-06-07 |
Publications (1)
| Publication Number | Publication Date |
|---|---|
| AR061282A1 true AR061282A1 (es) | 2008-08-20 |
Family
ID=38832369
Family Applications (2)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| ARP070102460A AR061282A1 (es) | 2006-06-07 | 2007-06-07 | Terapia genica para esclerosis lateral amiotrofica y otros trastornos del cordon espinal |
| ARP170102115A AR109171A2 (es) | 2006-06-07 | 2017-07-27 | Proteína del factor de crecimiento tipo insulina (igf-1) o del gen 1 de la neurona motora de supervivencia (smn1) para administrar a un ventrículo del cerebro de un sujeto |
Family Applications After (1)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| ARP170102115A AR109171A2 (es) | 2006-06-07 | 2017-07-27 | Proteína del factor de crecimiento tipo insulina (igf-1) o del gen 1 de la neurona motora de supervivencia (smn1) para administrar a un ventrículo del cerebro de un sujeto |
Country Status (19)
| Country | Link |
|---|---|
| US (4) | US9034836B2 (es) |
| EP (3) | EP2489733B1 (es) |
| JP (6) | JP2009539847A (es) |
| CN (1) | CN101460621B (es) |
| AR (2) | AR061282A1 (es) |
| BR (1) | BRPI0711965A2 (es) |
| CA (2) | CA2654292C (es) |
| CY (1) | CY1118555T1 (es) |
| ES (2) | ES2596885T3 (es) |
| HU (2) | HUE044182T2 (es) |
| IL (2) | IL195647A (es) |
| LT (2) | LT2029742T (es) |
| MX (4) | MX364444B (es) |
| PL (2) | PL2489733T3 (es) |
| PT (2) | PT2489733T (es) |
| RU (1) | RU2008152821A (es) |
| SI (2) | SI2029742T1 (es) |
| TR (1) | TR201906106T4 (es) |
| WO (1) | WO2007146046A2 (es) |
Families Citing this family (35)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| SI2029742T1 (sl) | 2006-06-07 | 2016-11-30 | Genzyme Corporation | Genska terapija amiotrofične lateralne skleroze in drugih motenj hrbtenjače |
| EP2249861B1 (en) * | 2008-01-16 | 2016-07-06 | Neurodyn Life Sciences Inc. | Progranulin for use in treating parkinson's disease or alzheimer's disease |
| US9573972B2 (en) | 2008-10-31 | 2017-02-21 | Neurodyn, Inc. | Nurotoxic sterol glycosides |
| MX356669B (es) | 2009-05-02 | 2018-06-08 | Genzyme Corp | Terapia genica para trastornos neurodegenerativos. |
| WO2012065248A1 (en) * | 2010-11-16 | 2012-05-24 | Kay Denis G | Method for increasing neprilysin expression and activity |
| CN103083667A (zh) * | 2011-11-01 | 2013-05-08 | 北京清美联创干细胞科技有限公司 | 嗅鞘细胞增殖试剂的连续给药方案 |
| US9512194B2 (en) | 2012-01-27 | 2016-12-06 | The Board Of Trustees Of The Leland Stanford Junior University | Modified IL-13 polypeptides |
| US20140065110A1 (en) | 2012-08-31 | 2014-03-06 | The Regents Of The University Of California | Genetically modified msc and therapeutic methods |
| US9663564B2 (en) | 2013-03-15 | 2017-05-30 | The Regents Of The University Of California | Vectors and methods to treat ischemia |
| CA2889499C (en) | 2012-10-24 | 2019-09-10 | Daiichi Sankyo Company, Limited | Growth hormone secretagogue receptor agonists for treating amyotrophic lateral sclerosis |
| WO2014084085A1 (ja) * | 2012-11-28 | 2014-06-05 | 国立大学法人名古屋大学 | 聴覚障害又は小脳性運動失調症の予防・治療剤 |
| BR112015027336A2 (pt) * | 2013-05-01 | 2017-09-26 | Genzyme Corp | composições e processos para tratamento de atrofia muscular espinhal |
| RU2018128780A (ru) | 2013-07-26 | 2018-12-05 | Юниверсити Оф Айова Рисерч Фаундейшн | Способы и композиции для лечения болезней мозга |
| KR101548989B1 (ko) | 2013-07-30 | 2015-09-01 | 광주과학기술원 | ＳｉｒＴ１을 이용한 관절 질환의 예방 또는 치료용 물질의 스크리닝 방법 |
| US9119832B2 (en) | 2014-02-05 | 2015-09-01 | The Regents Of The University Of California | Methods of treating mild brain injury |
| DE102014207498A1 (de) * | 2014-04-17 | 2015-10-22 | Universitätsklinikum Hamburg-Eppendorf | Viraler Vektor für den zielgerichteten Gentransfer in Gehirn und Rückenmark |
| MX2017005834A (es) | 2014-11-05 | 2017-11-17 | Voyager Therapeutics Inc | Polinucleotidos aad para el tratamiento de la enfermedad de parkinson. |
| US10214731B2 (en) * | 2015-05-28 | 2019-02-26 | Cornell University | Adeno-associated virus mediated delivery of C1E1 as a therapy for angioedema |
| CL2015003024A1 (es) | 2015-10-09 | 2016-05-13 | Univ Chile | Método de tratamiento genético utilizando el virus aav-xbp1s/gfp, y su uso en la prevención y tratamiento de la esclerosis lateral amiotrofica. |
| US10357543B2 (en) | 2015-11-16 | 2019-07-23 | Ohio State Innovation Foundation | Methods and compositions for treating disorders and diseases using Survival Motor Neuron (SMN) protein |
| EP3423109A4 (en) * | 2016-03-02 | 2019-08-14 | The Children's Hospital of Philadelphia | THERAPY FOR FRONTOTEMPORAL DEMENTIA |
| MD3589730T2 (ro) | 2017-02-28 | 2024-04-30 | Univ Pennsylvania | Vector de virus adeno-asociat (AAV) de cladă F și utilizările acestuia |
| JOP20190200A1 (ar) | 2017-02-28 | 2019-08-27 | Univ Pennsylvania | تركيبات نافعة في معالجة ضمور العضل النخاعي |
| JOP20190269A1 (ar) | 2017-06-15 | 2019-11-20 | Voyager Therapeutics Inc | بولي نوكليوتيدات aadc لعلاج مرض باركنسون |
| KR20200074132A (ko) | 2017-10-03 | 2020-06-24 | 프리베일 테라퓨틱스, 인크. | 리소좀 장애를 위한 유전자 요법 |
| WO2019070893A1 (en) | 2017-10-03 | 2019-04-11 | Prevail Therapeutics, Inc. | GENE THERAPIES FOR LYSOSOMAL DISORDERS |
| US20200390861A1 (en) * | 2017-10-10 | 2020-12-17 | Medicenna Therapeutics, Inc. | Il-4-fusion formulations for treatment of central nervous system (cns) tumors |
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