AR109171A2 - 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 - Google Patents
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 sujetoInfo
- Publication number
- AR109171A2 AR109171A2 ARP170102115A ARP170102115A AR109171A2 AR 109171 A2 AR109171 A2 AR 109171A2 AR P170102115 A ARP170102115 A AR P170102115A AR P170102115 A ARP170102115 A AR P170102115A AR 109171 A2 AR109171 A2 AR 109171A2
- Authority
- AR
- Argentina
- Prior art keywords
- igf
- protein
- smn
- gene
- growth factor
- Prior art date
Links
- 108090000623 proteins and genes Proteins 0.000 title abstract 10
- 102000004169 proteins and genes Human genes 0.000 title abstract 5
- 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 title abstract 4
- 102000004877 Insulin Human genes 0.000 title abstract 2
- 108090001061 Insulin Proteins 0.000 title abstract 2
- 210000004556 brain Anatomy 0.000 title abstract 2
- 239000003102 growth factor Substances 0.000 title abstract 2
- 229940125396 insulin Drugs 0.000 title abstract 2
- 102000003951 Erythropoietin Human genes 0.000 abstract 2
- 108090000394 Erythropoietin 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
- 229940105423 erythropoietin Drugs 0.000 abstract 2
- 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 2
- 102100022987 Angiogenin Human genes 0.000 abstract 1
- 102100021851 Calbindin Human genes 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
- 102000003693 Hedgehog Proteins Human genes 0.000 abstract 1
- 108090000031 Hedgehog Proteins Proteins 0.000 abstract 1
- 108090000723 Insulin-Like Growth Factor I Proteins 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
- 102000013275 Somatomedins Human genes 0.000 abstract 1
- 102000005789 Vascular Endothelial Growth Factors Human genes 0.000 abstract 1
- 108010019530 Vascular Endothelial Growth Factors Proteins 0.000 abstract 1
- 108010072788 angiogenin Proteins 0.000 abstract 1
- 108060001061 calbindin Proteins 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
- 210000002161 motor neuron Anatomy 0.000 abstract 1
- -1 neuroserpine Proteins 0.000 abstract 1
- 230000000508 neurotrophic effect Effects 0.000 abstract 1
- 229940097325 prolactin Drugs 0.000 abstract 1
- 210000000278 spinal cord Anatomy 0.000 abstract 1
- 230000004083 survival effect Effects 0.000 abstract 1
- 239000013603 viral 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
-
- 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)
- General Health & Medical Sciences (AREA)
- Veterinary Medicine (AREA)
- Public Health (AREA)
- Medicinal Chemistry (AREA)
- Animal Behavior & Ethology (AREA)
- Chemical & Material Sciences (AREA)
- Pharmacology & Pharmacy (AREA)
- Bioinformatics & Cheminformatics (AREA)
- Neurology (AREA)
- General Chemical & Material Sciences (AREA)
- Chemical Kinetics & Catalysis (AREA)
- Organic Chemistry (AREA)
- Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
- Molecular Biology (AREA)
- Epidemiology (AREA)
- Genetics & Genomics (AREA)
- Biotechnology (AREA)
- Biomedical Technology (AREA)
- Neurosurgery (AREA)
- Orthopedic Medicine & Surgery (AREA)
- Physical Education & Sports Medicine (AREA)
- Gastroenterology & Hepatology (AREA)
- Endocrinology (AREA)
- Zoology (AREA)
- Diabetes (AREA)
- Immunology (AREA)
- Proteomics, Peptides & Aminoacids (AREA)
- Psychiatry (AREA)
- Pain & Pain Management (AREA)
- Hospice & Palliative Care (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
Reivindicación 1: Una 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, caracterizada porque comprende: un vector viral neurotrófico recombinante que comprende dicho gen IGF-1 o dicho gen SMN-1 operativamente ligado a un promotor, en donde dicho gen IGF-1 o dicho gen SMN-1 se expresa y la proteína IGF-1 o SMN-1 expresada se suministra al cordón espinal. Reivindicación 8: La proteína de acuerdo con la reivindicación 1, caracterizada porque expresa dos proteínas seleccionadas del grupo que consiste en factor de crecimiento de insulina 1 (IGF-1), calbindina D28, parvalbumina, HIF1-a, SIRT-2, VEGF, SMN-1, SMN-2, FNTC (factor neurotrófico ciliar), erizo sónico (shh), eritropoyetina (EPO), lisil oxidasa (LOX), progranulina, prolactina, grelina, neuroserpina, angiogenina, y lactógena placentaria.
Applications Claiming Priority (1)
| Application Number | Priority Date | Filing Date | Title |
|---|---|---|---|
| US81141906P | 2006-06-07 | 2006-06-07 |
Publications (1)
| Publication Number | Publication Date |
|---|---|
| AR109171A2 true AR109171A2 (es) | 2018-11-07 |
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 Before (1)
| 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 |
Country Status (19)
| Country | Link |
|---|---|
| US (4) | US9034836B2 (es) |
| EP (3) | EP3540054A3 (es) |
| JP (6) | JP2009539847A (es) |
| CN (1) | CN101460621B (es) |
| AR (2) | AR061282A1 (es) |
| BR (1) | BRPI0711965A2 (es) |
| CA (1) | CA2654292C (es) |
| CY (1) | CY1118555T1 (es) |
| ES (2) | ES2596885T3 (es) |
| HU (2) | HUE044182T2 (es) |
| IL (2) | IL195647A (es) |
| LT (2) | LT2029742T (es) |
| MX (4) | MX2008015530A (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 (43)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| BRPI0711965A2 (pt) | 2006-06-07 | 2012-01-24 | Genzyme Corp | terapia de gene para esclerose lateral amiotrófica e outras enfermidades da medula espinal |
| EP3009143B1 (en) * | 2008-01-16 | 2018-10-03 | Neurodyn Life Sciences Inc. | Progranulin for use in treating parkinson's disease or alzheimer's disease |
| CN102272321A (zh) | 2008-10-31 | 2011-12-07 | 神经动力公司 | 神经毒性甾醇糖苷 |
| CN102459611B (zh) | 2009-05-02 | 2016-11-09 | 建新公司 | 神经退行性疾病的基因治疗 |
| CN103491974A (zh) * | 2010-11-16 | 2014-01-01 | 神经机能生命科学公司 | 用于提高中性溶酶的表达与活性的方法 |
| 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 |
| EP2850093B1 (en) | 2012-01-27 | 2019-01-09 | The Board of Trustees of the Leland Stanford Junior University | Therapeutic il-13 polypeptides |
| US9738696B2 (en) | 2012-08-09 | 2017-08-22 | The Board Of Trustees Of The Leland Stanford Junior University | Superkines and synthekines: repurposed cytokines with new and enhanced signaling activities |
| US9663564B2 (en) | 2013-03-15 | 2017-05-30 | The Regents Of The University Of California | Vectors and methods to treat ischemia |
| US20140065110A1 (en) | 2012-08-31 | 2014-03-06 | The Regents Of The University Of California | Genetically modified msc and therapeutic methods |
| JP6262661B2 (ja) | 2012-10-24 | 2018-01-17 | 第一三共株式会社 | 筋萎縮性側索硬化症治療剤 |
| JP6232547B2 (ja) * | 2012-11-28 | 2017-11-22 | 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 |
| CA2911105A1 (en) * | 2013-05-01 | 2014-11-06 | Genzyme Corporation | Compositions and methods for treating spinal muscular atrophy |
| EP3024497B1 (en) | 2013-07-26 | 2021-01-13 | University of Iowa Research Foundation | Methods and compositions for treating brain diseases |
| KR101548989B1 (ko) | 2013-07-30 | 2015-09-01 | 광주과학기술원 | ＳｉｒＴ１을 이용한 관절 질환의 예방 또는 치료용 물질의 스크리닝 방법 |
| JP6936934B2 (ja) | 2013-09-24 | 2021-09-22 | メディシナ セラピューティクス インコーポレイテッド | インターロイキン−４受容体結合融合タンパク質及びその使用 |
| 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 |
| CN107106689A (zh) | 2014-11-05 | 2017-08-29 | 沃雅戈治疗公司 | 用于治疗帕金森病的aadc多核苷酸 |
| MX2017014966A (es) * | 2015-05-28 | 2018-08-15 | Univ Cornell | Liberación de c1ei mediada por virus adenoasociado como terapia para el 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. |
| CA3005434C (en) * | 2015-11-16 | 2024-09-10 | Ohio State Innovation Foundation | METHODS AND COMPOSITIONS FOR TREATING DISORDERS AND DISEASES USING MOTOR NERVE SURVIVAL PROTEIN (MNS) |
| CA3016314A1 (en) * | 2016-03-02 | 2017-09-08 | Julianne REIDERS | Therapy for frontotemporal dementia |
| JOP20190200A1 (ar) | 2017-02-28 | 2019-08-27 | Univ Pennsylvania | تركيبات نافعة في معالجة ضمور العضل النخاعي |
| AU2018227440B2 (en) | 2017-02-28 | 2024-06-06 | The Trustees Of The University Of Pennsylvania | Adeno-associated virus (AAV) clade f vector and uses therefor |
| JOP20190269A1 (ar) | 2017-06-15 | 2019-11-20 | Voyager Therapeutics Inc | بولي نوكليوتيدات aadc لعلاج مرض باركنسون |
| BR112020006671A2 (pt) | 2017-10-03 | 2020-12-01 | Prevail Therapeutics, Inc. | terapias genéticas para distúrbios lisossômicos |
| US11993790B2 (en) | 2017-10-03 | 2024-05-28 | Prevail Therapeutics, Inc. | Gene therapies for lysosomal disorders |
| WO2019070893A1 (en) | 2017-10-03 | 2019-04-11 | Prevail Therapeutics, Inc. | GENE THERAPIES FOR LYSOSOMAL DISORDERS |
| EP3694558B1 (en) | 2017-10-10 | 2024-04-10 | Medicenna Therapeutics Inc. | Il-4-fusion formulations for treatment of central nervous system (cns) tumors |
| JP2021523914A (ja) | 2018-05-15 | 2021-09-09 | ボイジャー セラピューティクス インコーポレイテッドＶｏｙａｇｅｒ Ｔｈｅｒａｐｅｕｔｉｃｓ，Ｉｎｃ． | パーキンソン病を治療するための組成物および方法 |
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| JP7528066B2 (ja) | 2018-09-28 | 2024-08-05 | ボイジャー セラピューティクス インコーポレイテッド | 操作されたプロモータを有するフラタキシン発現コンストラクトおよびその使用方法 |
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