AR111292A1 - Administración de vectores del virus adenoasociado de microdistrofina específica del músculo para tratar la distrofia muscular - Google Patents
Administración de vectores del virus adenoasociado de microdistrofina específica del músculo para tratar la distrofia muscularInfo
- Publication number
- AR111292A1 AR111292A1 ARP180100629A ARP180100629A AR111292A1 AR 111292 A1 AR111292 A1 AR 111292A1 AR P180100629 A ARP180100629 A AR P180100629A AR P180100629 A ARP180100629 A AR P180100629A AR 111292 A1 AR111292 A1 AR 111292A1
- Authority
- AR
- Argentina
- Prior art keywords
- vectors
- microdistrophin
- muscular dystrophy
- virus
- adeo
- Prior art date
Links
- 239000013598 vector Substances 0.000 title abstract 4
- 210000003205 muscle Anatomy 0.000 title abstract 2
- 201000006938 muscular dystrophy Diseases 0.000 title abstract 2
- 241000700605 Viruses Species 0.000 title 1
- HJORMJIFDVBMOB-UHFFFAOYSA-N rolipram Chemical compound COC1=CC=C(C2CC(=O)NC2)C=C1OC1CCCC1 HJORMJIFDVBMOB-UHFFFAOYSA-N 0.000 title 1
- 108090000623 proteins and genes Proteins 0.000 abstract 2
- 241000702421 Dependoparvovirus Species 0.000 abstract 1
- 206010016654 Fibrosis Diseases 0.000 abstract 1
- 208000027418 Wounds and injury Diseases 0.000 abstract 1
- 230000006378 damage Effects 0.000 abstract 1
- 230000004761 fibrosis Effects 0.000 abstract 1
- 208000014674 injury Diseases 0.000 abstract 1
- 210000004165 myocardium Anatomy 0.000 abstract 1
- 210000001087 myotubule Anatomy 0.000 abstract 1
- 210000002027 skeletal muscle Anatomy 0.000 abstract 1
Classifications
-
- 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
- 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
- A61K38/1703—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from animals; from humans from vertebrates
- A61K38/1709—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from animals; from humans from vertebrates from mammals
-
- 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
- A61K48/0058—Nucleic acids adapted for tissue specific expression, e.g. having tissue specific promoters as part of a contruct
-
- A—HUMAN NECESSITIES
- A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
- A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
- A61K9/00—Medicinal preparations characterised by special physical form
- A61K9/0012—Galenical forms characterised by the site of application
- A61K9/0019—Injectable compositions; Intramuscular, intravenous, arterial, subcutaneous administration; Compositions to be administered through the skin in an invasive manner
- A61K9/0029—Parenteral nutrition; Parenteral nutrition compositions as drug carriers
-
- C—CHEMISTRY; METALLURGY
- C07—ORGANIC CHEMISTRY
- C07K—PEPTIDES
- C07K14/00—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof
- C07K14/435—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from animals; from humans
- C07K14/46—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from animals; from humans from vertebrates
- C07K14/47—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from animals; from humans from vertebrates from mammals
-
- 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
-
- 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
- C12N7/00—Viruses; Bacteriophages; Compositions thereof; Preparation or purification thereof
-
- A—HUMAN NECESSITIES
- A01—AGRICULTURE; FORESTRY; ANIMAL HUSBANDRY; HUNTING; TRAPPING; FISHING
- A01K—ANIMAL HUSBANDRY; AVICULTURE; APICULTURE; PISCICULTURE; FISHING; REARING OR BREEDING ANIMALS, NOT OTHERWISE PROVIDED FOR; NEW BREEDS OF ANIMALS
- A01K2227/00—Animals characterised by species
- A01K2227/10—Mammal
- A01K2227/105—Murine
-
- A—HUMAN NECESSITIES
- A01—AGRICULTURE; FORESTRY; ANIMAL HUSBANDRY; HUNTING; TRAPPING; FISHING
- A01K—ANIMAL HUSBANDRY; AVICULTURE; APICULTURE; PISCICULTURE; FISHING; REARING OR BREEDING ANIMALS, NOT OTHERWISE PROVIDED FOR; NEW BREEDS OF ANIMALS
- A01K2267/00—Animals characterised by purpose
- A01K2267/03—Animal model, e.g. for test or diseases
- A01K2267/0306—Animal model for genetic diseases
-
- 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
- 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
- C12N2830/00—Vector systems having a special element relevant for transcription
- C12N2830/008—Vector systems having a special element relevant for transcription cell type or tissue specific enhancer/promoter combination
Landscapes
- Health & Medical Sciences (AREA)
- Life Sciences & Earth Sciences (AREA)
- Chemical & Material Sciences (AREA)
- Genetics & Genomics (AREA)
- Engineering & Computer Science (AREA)
- Organic Chemistry (AREA)
- Bioinformatics & Cheminformatics (AREA)
- General Health & Medical Sciences (AREA)
- Zoology (AREA)
- Medicinal Chemistry (AREA)
- Biotechnology (AREA)
- Biomedical Technology (AREA)
- Wood Science & Technology (AREA)
- Biochemistry (AREA)
- General Engineering & Computer Science (AREA)
- Molecular Biology (AREA)
- Biophysics (AREA)
- Veterinary Medicine (AREA)
- Animal Behavior & Ethology (AREA)
- Pharmacology & Pharmacy (AREA)
- Public Health (AREA)
- Virology (AREA)
- Microbiology (AREA)
- Plant Pathology (AREA)
- Gastroenterology & Hepatology (AREA)
- Physics & Mathematics (AREA)
- Proteomics, Peptides & Aminoacids (AREA)
- Neurology (AREA)
- General Chemical & Material Sciences (AREA)
- Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
- Chemical Kinetics & Catalysis (AREA)
- Physical Education & Sports Medicine (AREA)
- Orthopedic Medicine & Surgery (AREA)
- Toxicology (AREA)
- Epidemiology (AREA)
- Immunology (AREA)
- Dermatology (AREA)
- Marine Sciences & Fisheries (AREA)
- Nutrition Science (AREA)
- Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
Abstract
La presente proporciona vectores de tratamiento génico, tales como vectores del virus adenoasociado (AAV), que expresan un gen de microdistrofina humana miniaturizado y un método para usar estos vectores en la expresión de microdistrofina en músculos esqueléticos, que incluyen el músculo de diafragma y cardiaco, y para proteger las fibras musculares de lesiones, aumentar la fuerza muscular y reducir y/o prevenir la fibrosis en sujetos que tienen distrofia muscular.
Applications Claiming Priority (1)
Application Number | Priority Date | Filing Date | Title |
---|---|---|---|
US201762473148P | 2017-03-17 | 2017-03-17 |
Publications (1)
Publication Number | Publication Date |
---|---|
AR111292A1 true AR111292A1 (es) | 2019-06-26 |
Family
ID=63523321
Family Applications (1)
Application Number | Title | Priority Date | Filing Date |
---|---|---|---|
ARP180100629A AR111292A1 (es) | 2017-03-17 | 2018-03-19 | Administración de vectores del virus adenoasociado de microdistrofina específica del músculo para tratar la distrofia muscular |
Country Status (30)
Country | Link |
---|---|
US (2) | US20200199621A1 (es) |
EP (2) | EP4245852A3 (es) |
JP (2) | JP2020513811A (es) |
KR (2) | KR20240112964A (es) |
CN (2) | CN110997923B (es) |
AR (1) | AR111292A1 (es) |
AU (1) | AU2018233732A1 (es) |
BR (1) | BR112019019248A2 (es) |
CA (1) | CA3056638A1 (es) |
CO (1) | CO2019011250A2 (es) |
CY (1) | CY1126080T1 (es) |
DK (1) | DK3596222T5 (es) |
EA (1) | EA201992201A1 (es) |
ES (1) | ES2948233T3 (es) |
FI (1) | FI3596222T3 (es) |
HR (1) | HRP20230522T1 (es) |
HU (1) | HUE062476T2 (es) |
IL (2) | IL269391B2 (es) |
LT (1) | LT3596222T (es) |
MA (1) | MA52112B1 (es) |
MD (1) | MD3596222T2 (es) |
MX (1) | MX2019011046A (es) |
PL (1) | PL3596222T3 (es) |
PT (1) | PT3596222T (es) |
RS (1) | RS64299B1 (es) |
SG (1) | SG11201908575SA (es) |
SI (1) | SI3596222T1 (es) |
TW (1) | TW201840850A (es) |
WO (1) | WO2018170408A1 (es) |
ZA (1) | ZA201906251B (es) |
Families Citing this family (17)
Publication number | Priority date | Publication date | Assignee | Title |
---|---|---|---|---|
CA3079416A1 (en) | 2017-10-20 | 2019-04-25 | Research Institute At Nationwide Children's Hospital | Methods and materials for nt-3 gene therapy |
US20220143136A1 (en) * | 2018-12-21 | 2022-05-12 | Northwestern University | Use of annexins in preventing and treating muscle membrane injury |
SG11202113052YA (en) * | 2019-05-30 | 2021-12-30 | Solid Biosciences Inc | Recombinant herpesvirales vector |
US20220349017A1 (en) * | 2019-08-29 | 2022-11-03 | Siemens Healthcare Diagnostics Inc. | Reagents and methods for detecting aav shedding |
TW202134260A (zh) * | 2019-11-28 | 2021-09-16 | 美商銳進科斯生物股份有限公司 | 微小肌縮蛋白基因療法之構築體及其用途 |
JP2023506030A (ja) * | 2019-12-16 | 2023-02-14 | リサーチ インスティチュート アット ネイションワイド チルドレンズ ホスピタル | ジストロフィン関連タンパク質複合体(dapc)を回復および維持するための組成物および方法 |
CN115485291A (zh) | 2020-04-29 | 2022-12-16 | 百时美施贵宝公司 | 具血影蛋白融合结构域的微型化抗肌萎缩蛋白及其用途 |
WO2022029543A1 (en) * | 2020-08-06 | 2022-02-10 | Intas Pharmaceuticals Ltd. | Adeno-associated virus vector delivery of micro-dystrophin to treat muscular dystrophy |
CN116783293A (zh) * | 2020-11-12 | 2023-09-19 | 精密生物科学公司 | 对肌营养不良蛋白基因中的识别序列具有特异性的工程化大范围核酸酶 |
EP4086276A1 (en) | 2021-05-03 | 2022-11-09 | Université d'Aix-Marseille | Composition for treating dysferlinopathy |
EP4108263A3 (en) * | 2021-06-02 | 2023-03-22 | Research Institute at Nationwide Children's Hospital | Recombinant adeno-associated virus products and methods for treating limb girdle muscular dystrophy 2a |
MX2024001634A (es) * | 2021-08-05 | 2024-02-27 | Insmed Inc | Particulas de virus adenoasociados y metodos de uso de estas. |
EP4384196A1 (en) * | 2021-08-11 | 2024-06-19 | Ultragenyx Pharmaceutical Inc. | Compositions and methods for treating a muscular dystrophy |
CN114316070B (zh) * | 2021-12-29 | 2022-11-15 | 上海勉亦生物科技有限公司 | 用于治疗肌营养不良症的转基因表达盒 |
EP4215614A1 (en) | 2022-01-24 | 2023-07-26 | Dynacure | Combination therapy for dystrophin-related diseases |
CN118460618A (zh) * | 2022-04-19 | 2024-08-09 | 康霖生物科技(杭州)有限公司 | 一种用于遗传性凝血因子缺乏病治疗的核酸构建体 |
WO2023248251A1 (en) * | 2022-06-24 | 2023-12-28 | Indian Institute Of Technology Kanpur | An optimized aav vector for gene therapy of muscular dystrophy |
Family Cites Families (32)
Publication number | Priority date | Publication date | Assignee | Title |
---|---|---|---|---|
US5173414A (en) | 1990-10-30 | 1992-12-22 | Applied Immune Sciences, Inc. | Production of recombinant adeno-associated virus vectors |
PT733103E (pt) | 1993-11-09 | 2004-07-30 | Targeted Genetics Corp | Criacao de elevados titulos de vectores de aav recombinantes |
ES2220923T3 (es) | 1993-11-09 | 2004-12-16 | Medical College Of Ohio | Lineas celulares estables capaces de expresar el gen de replicacion del virus adeno-asociado. |
US5658785A (en) | 1994-06-06 | 1997-08-19 | Children's Hospital, Inc. | Adeno-associated virus materials and methods |
US5856152A (en) | 1994-10-28 | 1999-01-05 | The Trustees Of The University Of Pennsylvania | Hybrid adenovirus-AAV vector and methods of use therefor |
EP0796339A1 (en) | 1994-12-06 | 1997-09-24 | Targeted Genetics Corporation | Packaging cell lines for generation of high titers of recombinant aav vectors |
FR2737730B1 (fr) | 1995-08-10 | 1997-09-05 | Pasteur Merieux Serums Vacc | Procede de purification de virus par chromatographie |
CA2230655C (en) | 1995-08-30 | 2008-06-17 | Genzyme Corporation | Chromatographic purification of adenovirus and aav |
EP0850313B8 (en) | 1995-09-08 | 2009-07-29 | Genzyme Corporation | Improved aav vectors for gene therapy |
US5910434A (en) | 1995-12-15 | 1999-06-08 | Systemix, Inc. | Method for obtaining retroviral packaging cell lines producing high transducing efficiency retroviral supernatant |
DE69738351T2 (de) | 1996-09-06 | 2008-11-13 | The Trustees Of The University Of Pennsylvania | Verfaheren zur durch rekombinante adeno-assoziierte virus-gerichtete gentherapie |
US6566118B1 (en) | 1997-09-05 | 2003-05-20 | Targeted Genetics Corporation | Methods for generating high titer helper-free preparations of released recombinant AAV vectors |
CA2830694C (en) | 1997-09-05 | 2018-02-27 | Genzyme Corporation | Methods for generating high titer helper-free preparations of recombinant aav vectors |
US6258595B1 (en) | 1999-03-18 | 2001-07-10 | The Trustees Of The University Of Pennsylvania | Compositions and methods for helper-free production of recombinant adeno-associated viruses |
EP1285078A2 (en) | 2000-04-28 | 2003-02-26 | The Trustees of The University of Pennsylvania | Recombinant aav vectors with aav5 capsids and aav5 vectors pseudotyped in heterologous capsids |
WO2002053703A2 (en) | 2001-01-05 | 2002-07-11 | Children's Hospital, Inc. | Aav2 vectors and methods |
EP1453547B1 (en) | 2001-12-17 | 2016-09-21 | The Trustees Of The University Of Pennsylvania | Adeno-associated virus (aav) serotype 8 sequences, vectors containing same, and uses therefor |
US20080044393A1 (en) * | 2004-07-16 | 2008-02-21 | White Robert L | Retinal dystrophin transgene and methods of use thereof |
HUE028662T2 (en) * | 2007-10-26 | 2016-12-28 | Academisch Ziekenhuis Leiden | Preparations and methods for controlling muscle disorders |
CN102459595A (zh) * | 2009-04-24 | 2012-05-16 | 普罗森那技术公司 | 用于治疗dmd的包含肌苷的寡核苷酸 |
EP3290055B1 (en) | 2011-07-25 | 2024-08-28 | Nationwide Children's Hospital, Inc. | Recombinant virus products and methods for inhibition of expression of dux4 |
WO2013102904A1 (en) * | 2012-01-05 | 2013-07-11 | Hadasit Medical Research Services & Development Ltd. | Methods and compositions for gene delivery |
EP2814958B1 (en) * | 2012-02-17 | 2019-08-07 | The Children's Hospital of Philadelphia | Aav vector compositions and methods for gene transfer to cells, organs and tissues |
DE102012007232B4 (de) | 2012-04-07 | 2014-03-13 | Susanne Weller | Verfahren zur Herstellung von rotierenden elektrischen Maschinen |
EA032706B1 (ru) * | 2013-04-20 | 2019-07-31 | Рисёрч Инститъют Эт Нэйшнвайд Чилдрен'С Хоспитал | ДОСТАВКА НАЦЕЛЕННЫХ НА ЭКЗОН 2 ПОЛИНУКЛЕОТИДНЫХ КОНСТРУКЦИЙ U7snRNA ПРИ ПОМОЩИ РЕКОМБИНАНТНОГО АДЕНОАССОЦИИРОВАННОГО ВИРУСА |
JP2015092462A (ja) | 2013-09-30 | 2015-05-14 | Tdk株式会社 | 正極及びそれを用いたリチウムイオン二次電池 |
PL3097197T3 (pl) * | 2014-01-21 | 2021-06-28 | Vrije Universiteit Brussel | Mięśniowo-specyficzne elementy regulatorowe kwasów nukleinowych oraz sposoby i ich zastosowanie |
JP6202701B2 (ja) | 2014-03-21 | 2017-09-27 | 株式会社日立国際電気 | 基板処理装置、半導体装置の製造方法及びプログラム |
EP2960336A1 (en) * | 2014-06-27 | 2015-12-30 | Genethon | Efficient systemic treatment of dystrophic muscle pathologies |
JP6197169B2 (ja) | 2014-09-29 | 2017-09-20 | 東芝メモリ株式会社 | 半導体装置の製造方法 |
GB201507842D0 (en) * | 2015-05-07 | 2015-06-17 | New Royal Holloway & Bedford | Production of large-sized microdystrophins in an AAV-based vector configuration |
MA45477A (fr) * | 2016-04-15 | 2019-02-20 | Res Inst Nationwide Childrens Hospital | Administration à vecteurs de virus adéno-associé de microarn-29 et micro-dystrophine pour traiter la dystrophie musculaire |
-
2018
- 2018-03-16 MA MA52112A patent/MA52112B1/fr unknown
- 2018-03-16 HU HUE18768395A patent/HUE062476T2/hu unknown
- 2018-03-16 SI SI201830926T patent/SI3596222T1/sl unknown
- 2018-03-16 JP JP2019550767A patent/JP2020513811A/ja active Pending
- 2018-03-16 SG SG11201908575S patent/SG11201908575SA/en unknown
- 2018-03-16 MX MX2019011046A patent/MX2019011046A/es unknown
- 2018-03-16 EP EP23167357.5A patent/EP4245852A3/en active Pending
- 2018-03-16 FI FIEP18768395.8T patent/FI3596222T3/fi active
- 2018-03-16 KR KR1020247022488A patent/KR20240112964A/ko active Search and Examination
- 2018-03-16 EP EP18768395.8A patent/EP3596222B1/en active Active
- 2018-03-16 DK DK18768395.8T patent/DK3596222T5/da active
- 2018-03-16 KR KR1020197029367A patent/KR102683682B1/ko active IP Right Grant
- 2018-03-16 CA CA3056638A patent/CA3056638A1/en active Pending
- 2018-03-16 AU AU2018233732A patent/AU2018233732A1/en active Pending
- 2018-03-16 IL IL269391A patent/IL269391B2/en unknown
- 2018-03-16 CN CN201880018893.9A patent/CN110997923B/zh active Active
- 2018-03-16 WO PCT/US2018/022881 patent/WO2018170408A1/en active Application Filing
- 2018-03-16 US US16/494,614 patent/US20200199621A1/en not_active Abandoned
- 2018-03-16 BR BR112019019248A patent/BR112019019248A2/pt unknown
- 2018-03-16 LT LTEPPCT/US2018/022881T patent/LT3596222T/lt unknown
- 2018-03-16 EA EA201992201A patent/EA201992201A1/ru unknown
- 2018-03-16 CN CN202311798281.5A patent/CN118497275A/zh active Pending
- 2018-03-16 RS RS20230434A patent/RS64299B1/sr unknown
- 2018-03-16 HR HRP20230522TT patent/HRP20230522T1/hr unknown
- 2018-03-16 ES ES18768395T patent/ES2948233T3/es active Active
- 2018-03-16 IL IL311713A patent/IL311713A/en unknown
- 2018-03-16 PT PT187683958T patent/PT3596222T/pt unknown
- 2018-03-16 MD MDE20200102T patent/MD3596222T2/ro unknown
- 2018-03-16 PL PL18768395.8T patent/PL3596222T3/pl unknown
- 2018-03-19 AR ARP180100629A patent/AR111292A1/es unknown
- 2018-03-19 TW TW107109334A patent/TW201840850A/zh unknown
-
2019
- 2019-09-20 ZA ZA2019/06251A patent/ZA201906251B/en unknown
- 2019-10-10 CO CONC2019/0011250A patent/CO2019011250A2/es unknown
-
2022
- 2022-06-03 US US17/832,325 patent/US20220364117A1/en active Pending
-
2023
- 2023-02-13 JP JP2023019888A patent/JP2023053254A/ja active Pending
- 2023-06-21 CY CY20231100291T patent/CY1126080T1/el unknown
Also Published As
Similar Documents
Publication | Publication Date | Title |
---|---|---|
AR111292A1 (es) | Administración de vectores del virus adenoasociado de microdistrofina específica del músculo para tratar la distrofia muscular | |
CO2021000227A2 (es) | Suministro mediante vectores de virus adenoasociado de microdistrofina específica del músculo para tratar la distrofia muscular | |
CO2018012084A2 (es) | Administración de vectores del virus adenoasociado de microdistrofina para tratar la distrofia muscular | |
CO2018012082A2 (es) | Administración de vectores del virus adenoasociado de β-sarcoglicano y microarn-29 y el tratamiento de la distrofia muscular | |
WO2019012336A3 (en) | ADENO-ASSOCIATED VIRAL VECTOR DELIVERY OF A MICRO-DYSTROPHIN FRAGMENT FOR TREATING MUSCLE DYSTROPHY | |
CL2018001522A1 (es) | Focalización de péptidos para dirigir virus adenoasociados (aavs) | |
AR114350A1 (es) | Terapia genética para la distrofia muscular de cinturas del tipo 2c | |
MA50836A (fr) | Administration par vecteur à virus adéno-associé de micro-dystrophine spécifique de muscles pour traiter la dystrophie musculaire | |
CO2023000156A2 (es) | Administración de vectores de virus adenoasociado para distrofias musculares | |
CL2021002236A1 (es) | Inserción de vectores de virus adeno-asociados de b-sarcoglicano y el tratamiento de distrofia muscular | |
BR112018017247A2 (pt) | ?polinucleotídeo, vetor de ácido nucleico, preparação farmacêutica, kit, e, métodos para fabricar um vetor viral adenoassociado recombinante e para tratar uma doença? | |
BR112018009657A2 (pt) | métodos de tratamento de distrofia muscular | |
MA50914A (fr) | Vecteurs viraux comprenant des séquences codantes de rdh12 et méthodes de traitement de dystrophies rétiniennes | |
BR112017022621A2 (pt) | liberação do gene de smad7 como uma substância terapêutica | |
AR110947A1 (es) | Tratamiento de degeneración de la retina mediante el uso de células progenitoras | |
CO2020014399A2 (es) | Composiciones farmacéuticas para el tratamiento de la deficiencia de esfingomielinasa ácida | |
ES2978666T3 (es) | Suministro de alfa-sarcoglicano por vector de virus adenoasociado y tratamiento de la distrofia muscular | |
EA202190054A1 (ru) | Доставка специфичного для мышц микродистрофина с помощью вектора на основе аденоассоциированного вируса для лечения мышечной дистрофии | |
EA201892338A1 (ru) | Доставка микродистрофина вектором на основе аденоассоциированного вируса для лечения мышечной дистрофии | |
MX2020006451A (es) | Tratamiento de la fibrosis con inositol. | |
MA42736A1 (fr) | Méthodes de traitement de la dystrophie musculaire | |
MX2019010599A (es) | Usos medicos para tratamiento de retinitis pigmentosa. | |
MX2015007650A (es) | Tratamiento de retinitis pigmentosa. | |
AR083826A1 (es) | Terapia genica para trastornos neurodegenerativos mediante el empleo de un vector del virus adeno-asociado que expresa la proteina de la neurona motora de la supervivencia |