CA3056638A1 - Adeno-associated virus vector delivery of muscle specific micro-dystrophin to treat muscular dystrophy - Google Patents

Adeno-associated virus vector delivery of muscle specific micro-dystrophin to treat muscular dystrophy Download PDF

Info

Publication number
CA3056638A1
CA3056638A1 CA3056638A CA3056638A CA3056638A1 CA 3056638 A1 CA3056638 A1 CA 3056638A1 CA 3056638 A CA3056638 A CA 3056638A CA 3056638 A CA3056638 A CA 3056638A CA 3056638 A1 CA3056638 A1 CA 3056638A1
Authority
CA
Canada
Prior art keywords
dystrophin
micro
aav vector
recombinant aav
muscular dystrophy
Prior art date
Legal status (The legal status 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 status listed.)
Pending
Application number
CA3056638A
Other languages
English (en)
French (fr)
Inventor
Louise RODINO-KLAPAC
Jerry R. MENDELL
Current Assignee (The listed assignees may be inaccurate. Google has not performed a legal analysis and makes no representation or warranty as to the accuracy of the list.)
Nationwide Childrens Hospital Inc
Original Assignee
Nationwide Childrens Hospital Inc
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.)
Filing date
Publication date
Application filed by Nationwide Childrens Hospital Inc filed Critical Nationwide Childrens Hospital Inc
Publication of CA3056638A1 publication Critical patent/CA3056638A1/en
Pending legal-status Critical Current

Links

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
    • 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
    • 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
    • C07K14/4701—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from animals; from humans from vertebrates from mammals not used
    • C07K14/4707—Muscular dystrophy
    • 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
    • C12N15/11—DNA or RNA fragments; Modified forms thereof; Non-coding nucleic acids having a biological activity
    • C12N15/113—Non-coding nucleic acids modulating the expression of genes, e.g. antisense oligonucleotides; Antisense DNA or RNA; Triplex- forming oligonucleotides; Catalytic nucleic acids, e.g. ribozymes; Nucleic acids used in co-suppression or gene silencing
    • 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
    • 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
    • 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
    • C12N2750/14151—Methods of production or purification of viral material
    • C12N2750/14152—Methods of production or purification of viral material relating to complementing cells and packaging systems for producing virus or viral particles
    • 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)
  • Engineering & Computer Science (AREA)
  • Genetics & Genomics (AREA)
  • Organic Chemistry (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • General Health & Medical Sciences (AREA)
  • Zoology (AREA)
  • Medicinal Chemistry (AREA)
  • Wood Science & Technology (AREA)
  • Biotechnology (AREA)
  • Biomedical Technology (AREA)
  • General Engineering & Computer Science (AREA)
  • Biochemistry (AREA)
  • Molecular Biology (AREA)
  • Veterinary Medicine (AREA)
  • Animal Behavior & Ethology (AREA)
  • Pharmacology & Pharmacy (AREA)
  • Public Health (AREA)
  • Biophysics (AREA)
  • Microbiology (AREA)
  • Virology (AREA)
  • Proteomics, Peptides & Aminoacids (AREA)
  • Gastroenterology & Hepatology (AREA)
  • Epidemiology (AREA)
  • Plant Pathology (AREA)
  • Physics & Mathematics (AREA)
  • Neurology (AREA)
  • Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
  • General Chemical & Material Sciences (AREA)
  • Chemical Kinetics & Catalysis (AREA)
  • Physical Education & Sports Medicine (AREA)
  • Orthopedic Medicine & Surgery (AREA)
  • Immunology (AREA)
  • Toxicology (AREA)
  • Marine Sciences & Fisheries (AREA)
  • Nutrition Science (AREA)
  • Dermatology (AREA)
  • Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
CA3056638A 2017-03-17 2018-03-16 Adeno-associated virus vector delivery of muscle specific micro-dystrophin to treat muscular dystrophy Pending CA3056638A1 (en)

Applications Claiming Priority (3)

Application Number Priority Date Filing Date Title
US201762473148P 2017-03-17 2017-03-17
US62/473,148 2017-03-17
PCT/US2018/022881 WO2018170408A1 (en) 2017-03-17 2018-03-16 Adeno-associated virus vector delivery of muscle specific micro-dystrophin to treat muscular dystrophy

Publications (1)

Publication Number Publication Date
CA3056638A1 true CA3056638A1 (en) 2018-09-20

Family

ID=63523321

Family Applications (1)

Application Number Title Priority Date Filing Date
CA3056638A Pending CA3056638A1 (en) 2017-03-17 2018-03-16 Adeno-associated virus vector delivery of muscle specific micro-dystrophin to treat muscular dystrophy

Country Status (33)

Country Link
US (2) US20200199621A1 (enExample)
EP (2) EP4245852A3 (enExample)
JP (3) JP7676111B2 (enExample)
KR (3) KR102887103B1 (enExample)
CN (2) CN118497275A (enExample)
AR (1) AR111292A1 (enExample)
AU (3) AU2018233732B2 (enExample)
BR (1) BR112019019248A2 (enExample)
CA (1) CA3056638A1 (enExample)
CO (1) CO2019011250A2 (enExample)
CY (1) CY1126080T1 (enExample)
DK (1) DK3596222T5 (enExample)
EA (1) EA201992201A1 (enExample)
ES (1) ES2948233T3 (enExample)
FI (1) FI3596222T3 (enExample)
HR (1) HRP20230522T1 (enExample)
HU (1) HUE062476T2 (enExample)
IL (3) IL311713B2 (enExample)
LT (1) LT3596222T (enExample)
MA (1) MA52112B1 (enExample)
MD (1) MD3596222T2 (enExample)
MX (3) MX2019011046A (enExample)
MY (1) MY203547A (enExample)
PL (1) PL3596222T3 (enExample)
PT (1) PT3596222T (enExample)
RS (1) RS64299B1 (enExample)
SA (1) SA519410146B1 (enExample)
SG (1) SG11201908575SA (enExample)
SI (1) SI3596222T1 (enExample)
SM (1) SMT202300201T1 (enExample)
TW (2) TW202525835A (enExample)
WO (1) WO2018170408A1 (enExample)
ZA (1) ZA201906251B (enExample)

Families Citing this family (27)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
HK1257955A1 (zh) 2015-11-16 2019-11-01 The Research Institute At Nationwide Children's Hospital 用於治疗肌联蛋白类肌病和其他肌联蛋白病变的材料和方法
US11338045B2 (en) 2017-03-17 2022-05-24 Newcastle University Adeno-associated virus vector delivery of a fragment of micro-dystrophin to treat muscular dystrophy
CN119193705A (zh) 2017-10-20 2024-12-27 全国儿童医院研究所 Nt-3基因疗法的方法和材料
KR102824021B1 (ko) 2018-01-31 2025-06-24 더 리서치 인스티튜트 앳 네이션와이드 칠드런스 하스피탈 2c형 지대근이영양증에 대한 유전자 치료
US12491265B2 (en) 2018-06-18 2025-12-09 Research Institute At Nationwide Children's Hospital Adeno-associated virus vector delivery of muscle specific micro-dystrophin to treat muscular dystrophy
MX2020014119A (es) 2018-06-29 2021-06-18 Res Inst Nationwide Childrens Hospital Productos y métodos de virus adenoasociado recombinante para el tratamiento de la distrofia muscular de cinturas 2a.
AU2019406214A1 (en) * 2018-12-21 2021-08-05 Northwestern University Use of annexins in preventing and treating muscle membrane injury
JP2022523038A (ja) * 2019-02-02 2022-04-21 ユニバーシティ オブ ピッツバーグ －オブ ザ コモンウェルス システム オブ ハイヤー エデュケイション 遺伝子置換及び抗炎症を介したデュシェンヌ型筋ジストロフィーに対する一段階遺伝子療法
MX2021010356A (es) 2019-02-26 2022-03-04 Res Inst Nationwide Childrens Hospital SUMINISTRO DE ß-SARCOGLICANO MEDIANTE VECTORES DE VIRUS ADENOASOCIADO Y EL TRATAMIENTO DE LA DISTROFIA MUSCULAR.
WO2020243706A1 (en) * 2019-05-30 2020-12-03 Solid Biosciences Inc. Recombinant herpesvirales vector
CN120400170A (zh) 2019-08-21 2025-08-01 全国儿童医院研究所 Α-肌聚糖的腺相关病毒载体递送和肌营养不良症的治疗
EP4022097A4 (en) * 2019-08-29 2022-12-14 Siemens Healthcare Diagnostics Inc. REAGENTS AND METHODS FOR DETECTING AAV EXCRETION
JP2023503637A (ja) * 2019-11-28 2023-01-31 リジェネックスバイオ インコーポレイテッド マイクロジストロフィン遺伝子治療コンストラクト及びその使用
EP4117783A4 (en) * 2019-12-16 2024-08-21 Research Institute at Nationwide Children's Hospital COMPOSITIONS AND METHODS FOR RESTORING AND MAINTAINING DYSTROPHIN-ASSOCIATED PROTEIN COMPLEX (DAPC)
BR112022021635A2 (pt) 2020-04-29 2022-12-06 Bristol Myers Squibb Co Distrofinas miniaturizadas tendo domínios de fusão de espectrina e usos das mesmas
WO2022029543A1 (en) * 2020-08-06 2022-02-10 Intas Pharmaceuticals Ltd. Adeno-associated virus vector delivery of micro-dystrophin to treat muscular dystrophy
IL317538A (en) * 2020-11-12 2025-02-01 Prec Biosciences Inc Engineered nucleases with specificity for recognition sequences in the dystrophin gene
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
CA3224482A1 (en) * 2021-08-05 2023-02-09 Brian Kaspar Adeno-associated virus particles and methods of use thereof
WO2023019168A1 (en) * 2021-08-11 2023-02-16 Ultragenyx Pharmaceutical Inc. Compositions and methods for treating a muscular dystrophy
EP4219726A1 (en) 2021-10-15 2023-08-02 Research Institute at Nationwide Children's Hospital Self-complementary adeno-associated virus vector and its use in treatment of 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
EP4509606A4 (en) * 2022-04-19 2025-12-10 Kanglin Biotech Hangzhou Co Ltd CONSTRUCTION OF NUCLEIC ACID FOR THE TREATMENT OF AN INHERITED COAGULATION FACTOR DEFICIENCY AND ITS USE
US20250346921A1 (en) * 2022-06-24 2025-11-13 Indian Institute Of Technology Kanpur An optimized aav vector for gene therapy of muscular dystrophy
CN115819546B (zh) * 2022-08-19 2025-12-05 成都金唯科生物科技有限公司 表达微缩抗肌营养不良蛋白基因的腺相关病毒载体及其应用

Family Cites Families (32)

* Cited by examiner, † Cited by third party
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
WO1995013392A1 (en) 1993-11-09 1995-05-18 Medical College Of Ohio Stable cell lines capable of expressing the adeno-associated virus replication gene
JPH09509564A (ja) 1993-11-09 1997-09-30 ターゲテッド ジェネティックス コーポレイション 高力価組換えａａｖベクターの生成
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
WO1997008298A1 (en) 1995-08-30 1997-03-06 Genzyme Corporation Chromatographic purification of adenovirus and aav
JPH11514853A (ja) 1995-09-08 1999-12-21 ジエンザイム コーポレイション 遺伝子治療のための改良されたａａｖベクター
US5910434A (en) 1995-12-15 1999-06-08 Systemix, Inc. Method for obtaining retroviral packaging cell lines producing high transducing efficiency retroviral supernatant
WO1998009657A2 (en) 1996-09-06 1998-03-12 Trustees Of The University Of Pennsylvania Method for recombinant adeno-associated virus-directed gene therapy
CA2682108C (en) 1997-09-05 2013-12-24 Targeted Genetics Corporation Methods for generating high titer helper-free preparations of recombinant aav vectors
US6566118B1 (en) 1997-09-05 2003-05-20 Targeted Genetics Corporation Methods for generating high titer helper-free preparations of released 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
WO2001083692A2 (en) 2000-04-28 2001-11-08 The Trustees Of The University Of Pennsylvania Recombinant aav vectors with aav5 capsids and aav5 vectors pseudotyped in heterologous capsids
US6962815B2 (en) 2001-01-05 2005-11-08 Children's Hopital Inc. AAV2 vectors and methods
JP4810062B2 (ja) 2001-12-17 2011-11-09 ザ・トラステイーズ・オブ・ザ・ユニバーシテイ・オブ・ペンシルベニア アデノ随伴ウイルス（ａａｖ）血清型８の配列
US20080044393A1 (en) * 2004-07-16 2008-02-21 White Robert L Retinal dystrophin transgene and methods of use thereof
CN101896186A (zh) * 2007-10-26 2010-11-24 莱顿教学医院 对抗肌肉病症的方式和方法
CA2759899A1 (en) * 2009-04-24 2010-10-28 Prosensa Technologies B.V. Oligonucleotide comprising an inosine for treating dmd
CA3106285A1 (en) 2011-07-25 2013-01-31 Nationwide Children's Hospital, Inc. Recombinant virus products and methods for inhibition of expression of dux4
US20150045416A1 (en) * 2012-01-05 2015-02-12 Hadasit Medical Research Services & Development Ltd. Methods and Compositions for Gene Delivery
CN104520428B (zh) * 2012-02-17 2018-09-21 费城儿童医院 将基因转移到细胞、器官和组织的aav载体组合物和方法
DE102012007232B4 (de) 2012-04-07 2014-03-13 Susanne Weller Verfahren zur Herstellung von rotierenden elektrischen Maschinen
CN109652385A (zh) * 2013-04-20 2019-04-19 全国儿童医院研究所 外显子2靶向U7snRNA多核苷酸构建体的重组腺相关病毒递送
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
CA2936726C (en) * 2014-01-21 2024-04-30 Vrije Universiteit Brussel Muscle-specific nucleic acid regulatory elements and methods and use thereof
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

Also Published As

Publication number Publication date
IL269391B2 (en) 2024-09-01
MD3596222T2 (ro) 2023-08-31
CN110997923A (zh) 2020-04-10
PL3596222T3 (pl) 2023-10-09
WO2018170408A1 (en) 2018-09-20
EP4245852A3 (en) 2023-11-22
SMT202300201T1 (it) 2023-09-06
MY203547A (en) 2024-07-02
KR20250163423A (ko) 2025-11-20
JP2020513811A (ja) 2020-05-21
IL269391B1 (en) 2024-05-01
MX2025007401A (es) 2025-08-01
KR102683682B1 (ko) 2024-07-09
IL311713B1 (en) 2025-06-01
IL269391A (en) 2019-11-28
CN110997923B (zh) 2024-01-02
DK3596222T3 (da) 2023-06-19
MA52112A (fr) 2020-01-22
KR20190130591A (ko) 2019-11-22
CN118497275A (zh) 2024-08-16
EP3596222B1 (en) 2023-04-12
AU2024266707B2 (en) 2025-11-27
HUE062476T2 (hu) 2023-11-28
BR112019019248A2 (pt) 2020-04-28
KR102887103B1 (ko) 2025-11-18
TW202525835A (zh) 2025-07-01
KR20240112964A (ko) 2024-07-19
EP3596222A4 (en) 2021-01-06
AU2018233732A1 (en) 2019-10-03
TWI878204B (zh) 2025-04-01
TW201840850A (zh) 2018-11-16
LT3596222T (lt) 2023-07-25
MA52112B1 (fr) 2023-08-31
EP4245852A2 (en) 2023-09-20
EA201992201A1 (ru) 2020-03-19
FI3596222T3 (fi) 2023-06-08
PT3596222T (pt) 2023-06-20
DK3596222T5 (da) 2024-09-02
SI3596222T1 (sl) 2024-05-31
HRP20230522T1 (hr) 2023-08-04
MX2025007402A (es) 2025-07-01
IL311713A (en) 2024-05-01
AU2018233732B2 (en) 2024-10-24
IL311713B2 (en) 2025-10-01
EP3596222A1 (en) 2020-01-22
US20220364117A1 (en) 2022-11-17
JP7676111B2 (ja) 2025-05-14
ES2948233T3 (es) 2023-09-06
CY1126080T1 (el) 2023-11-15
CO2019011250A2 (es) 2020-02-28
JP2023053254A (ja) 2023-04-12
AR111292A1 (es) 2019-06-26
RS64299B1 (sr) 2023-07-31
IL320791A (en) 2025-07-01
MX2019011046A (es) 2019-10-17
JP2025061676A (ja) 2025-04-11
US20200199621A1 (en) 2020-06-25
SA519410146B1 (ar) 2024-11-10
AU2024266707A1 (en) 2025-01-30
AU2025200437A1 (en) 2025-02-13
ZA201906251B (en) 2024-01-31
SG11201908575SA (en) 2019-10-30

Similar Documents

Publication Publication Date Title
AU2024266707B2 (en) Adeno-Associated Virus Vector Delivery Of Muscle Specific Micro-Dystrophin To Treat Muscular Dystrophy
AU2023201093B2 (en) Adeno-Associated Virus Vector Delivery Of Micro-Dystrophin To Treat Muscular Dystrophy
US11338045B2 (en) Adeno-associated virus vector delivery of a fragment of micro-dystrophin to treat muscular dystrophy
EP3807413A1 (en) Adeno-associated virus vector delivery of muscle specific micro-dystrophin to treat muscular dystrophy
CA3187353A1 (en) Adeno-associated virus vector delivery for muscular dystrophies
US20240366789A1 (en) Aav-ids vectors for treatment of mucopolysaccharidosis ii
HK40099903A (en) Adeno-associated virus vector delivery of muscle specific micro-dystrophin to treat muscular dystrophy
HK40021330B (en) Adeno-associated virus vector delivery of muscle specific micro-dystrophin to treat muscular dystrophy
HK40021330A (en) Adeno-associated virus vector delivery of muscle specific micro-dystrophin to treat muscular dystrophy

Legal Events

Date Code Title Description
EEER Examination request

Effective date: 20220915

EEER Examination request

Effective date: 20220915

EEER Examination request

Effective date: 20220915

EEER Examination request

Effective date: 20220915

EEER Examination request

Effective date: 20220915