CN112040988A - 修饰的aav衣壳多肽用于治疗肌肉疾病 - Google Patents

修饰的aav衣壳多肽用于治疗肌肉疾病 Download PDF

Info

Publication number
CN112040988A
CN112040988A CN201980028398.0A CN201980028398A CN112040988A CN 112040988 A CN112040988 A CN 112040988A CN 201980028398 A CN201980028398 A CN 201980028398A CN 112040988 A CN112040988 A CN 112040988A
Authority
CN
China
Prior art keywords
muscle
aav capsid
aav
capsid polypeptide
amino acid
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
CN201980028398.0A
Other languages
English (en)
Chinese (zh)
Inventor
D·格里姆
J·温曼
J·埃尔安达里
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.)
Fraunhofer Gesellschaft zur Foerderung der Angewandten Forschung eV
Original Assignee
Fraunhofer Gesellschaft zur Foerderung der Angewandten Forschung eV
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 Fraunhofer Gesellschaft zur Foerderung der Angewandten Forschung eV filed Critical Fraunhofer Gesellschaft zur Foerderung der Angewandten Forschung eV
Publication of CN112040988A publication Critical patent/CN112040988A/zh
Pending legal-status Critical Current

Links

Images

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
    • C—CHEMISTRY; METALLURGY
    • C07—ORGANIC CHEMISTRY
    • C07K—PEPTIDES
    • C07K14/00—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof
    • C07K14/005—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from viruses
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K38/00—Medicinal preparations containing peptides
    • 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
    • A61K48/0008—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 'non-active' part of the composition delivered, e.g. wherein such 'non-active' part is not delivered simultaneously with the 'active' part of the composition
    • A61K48/0025—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 'non-active' part of the composition delivered, e.g. wherein such 'non-active' part is not delivered simultaneously with the 'active' part of the composition wherein the non-active part clearly interacts with the delivered nucleic acid
    • C—CHEMISTRY; METALLURGY
    • C07—ORGANIC CHEMISTRY
    • C07K—PEPTIDES
    • C07K2319/00—Fusion polypeptide
    • C07K2319/01—Fusion polypeptide containing a localisation/targetting motif
    • C07K2319/035—Fusion polypeptide containing a localisation/targetting motif containing a signal for targeting to the external surface of a cell, e.g. to the outer membrane of Gram negative bacteria, GPI- anchored eukaryote proteins
    • C—CHEMISTRY; METALLURGY
    • C07—ORGANIC CHEMISTRY
    • C07K—PEPTIDES
    • C07K2319/00—Fusion polypeptide
    • C07K2319/33—Fusion polypeptide fusions for targeting to specific cell types, e.g. tissue specific targeting, targeting of a bacterial subspecies
    • 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
    • C12N2750/14122—New viral proteins or individual genes, new structural or functional aspects of known viral proteins or genes
    • 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
    • C12N2750/14123—Virus like particles [VLP]
    • 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
    • C12N2750/14133—Use of viral protein as therapeutic agent other than vaccine, e.g. apoptosis inducing or anti-inflammatory
    • 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
    • C12N2750/14141—Use of virus, viral particle or viral elements as a vector
    • C12N2750/14145—Special targeting system for viral vectors

Landscapes

  • Chemical & Material Sciences (AREA)
  • Health & Medical Sciences (AREA)
  • Life Sciences & Earth Sciences (AREA)
  • Organic Chemistry (AREA)
  • Genetics & Genomics (AREA)
  • Molecular Biology (AREA)
  • Virology (AREA)
  • Biochemistry (AREA)
  • Biophysics (AREA)
  • General Health & Medical Sciences (AREA)
  • Engineering & Computer Science (AREA)
  • Gastroenterology & Hepatology (AREA)
  • Medicinal Chemistry (AREA)
  • Proteomics, Peptides & Aminoacids (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • Wood Science & Technology (AREA)
  • Biotechnology (AREA)
  • General Engineering & Computer Science (AREA)
  • Biomedical Technology (AREA)
  • Zoology (AREA)
  • Plant Pathology (AREA)
  • Microbiology (AREA)
  • Physics & Mathematics (AREA)
  • Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
  • Medicines Containing Material From Animals Or Micro-Organisms (AREA)
  • Micro-Organisms Or Cultivation Processes Thereof (AREA)
  • Peptides Or Proteins (AREA)
  • Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)
CN201980028398.0A 2018-04-27 2019-04-26 修饰的aav衣壳多肽用于治疗肌肉疾病 Pending CN112040988A (zh)

Applications Claiming Priority (3)

Application Number Priority Date Filing Date Title
EP18169822.6 2018-04-27
EP18169822 2018-04-27
PCT/EP2019/060790 WO2019207132A1 (en) 2018-04-27 2019-04-26 Modified aav capsid polypeptides for treatment of muscular diseases

Publications (1)

Publication Number Publication Date
CN112040988A true CN112040988A (zh) 2020-12-04

Family

ID=62167107

Family Applications (1)

Application Number Title Priority Date Filing Date
CN201980028398.0A Pending CN112040988A (zh) 2018-04-27 2019-04-26 修饰的aav衣壳多肽用于治疗肌肉疾病

Country Status (7)

Country Link
US (1) US20210363193A1 (https=)
EP (1) EP3784288A1 (https=)
JP (2) JP2021521833A (https=)
CN (1) CN112040988A (https=)
AU (2) AU2019258830B2 (https=)
CA (1) CA3097375A1 (https=)
WO (1) WO2019207132A1 (https=)

Cited By (4)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
CN113480615A (zh) * 2021-07-30 2021-10-08 上海信致医药科技有限公司 高视网膜亲和性的新型腺相关病毒衣壳蛋白及其应用
CN113563430A (zh) * 2021-07-30 2021-10-29 上海信致医药科技有限公司 用于治疗眼部疾病的基因递送系统及其应用
WO2023169115A1 (zh) * 2022-03-11 2023-09-14 上海勉亦生物科技有限公司 一种神经系统高亲和性的aav载体及其应用
CN118005749A (zh) * 2023-07-21 2024-05-10 广州派真生物技术有限公司 腺相关病毒突变体及其应用

Families Citing this family (49)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
MX2020011386A (es) 2018-04-27 2021-01-29 Spacecraft Seven Llc Terapia genica para la degeneracion del snc.
CN113966399A (zh) 2018-09-26 2022-01-21 加州理工学院 用于靶向基因疗法的腺相关病毒组合物
WO2020101042A1 (en) 2018-11-16 2020-05-22 Astellas Pharma Inc. Method for treating muscular dystrophy by targeting utrophin gene
EP3947700A4 (en) 2019-04-01 2023-01-04 Tenaya Therapeutics, Inc. Adeno-associated virus with engineered capsid
EP4028064A1 (en) * 2019-09-12 2022-07-20 The Broad Institute, Inc. Engineered adeno-associated virus capsids
JP7651562B2 (ja) * 2019-09-19 2025-03-26 ジェネトン Ｆｋｒｐの心毒性を軽減する遺伝子治療発現系
TWI897882B (zh) * 2019-09-20 2025-09-21 英商Ｕｃｌ商業有限責任公司 右心室心律失常性心肌病之基因療法組合物及治療
EP4041755A4 (en) * 2019-10-10 2023-10-25 Solid Biosciences Inc. Modified aav capsids and uses thereof
AU2020368539A1 (en) 2019-10-16 2022-04-28 Massachusetts Institute Of Technology Engineered muscle targeting compositions
JP2023523573A (ja) 2020-04-14 2023-06-06 ジェネトン 酸性セラミダーゼ欠乏症を処置するためのベクター
US12552838B2 (en) 2020-04-20 2026-02-17 Tenaya Therapeutics, Inc. Adeno-associated virus with engineered capsid
US20230173102A1 (en) * 2020-04-28 2023-06-08 Genethon Use of a Synthetic AAV Capsid for Gene Therapy of Muscle and Central Nervous System Disorders
EP4126912A4 (en) * 2020-05-05 2024-04-24 Duke University COMPOSITIONS AND METHODS FOR FORMING AND SECRETING EXTRACELLULAR VESILES AND AAV PARTICLES
WO2021230385A1 (en) 2020-05-15 2021-11-18 Astellas Pharma Inc. Method for treating muscular dystrophy by targeting utrophin gene
US20230159949A1 (en) * 2020-07-22 2023-05-25 The Broad Institute, Inc. Engineered muscle targeting compositions
KR20230053591A (ko) * 2020-07-22 2023-04-21 더 브로드 인스티튜트, 인코퍼레이티드 조작된 근육 표적화 조성물
KR20230042468A (ko) * 2020-08-05 2023-03-28 스페이스크래프트 세븐, 엘엘씨 Csrp3 (시스테인 및 글리신 풍부 단백질 3) 유전자 요법
KR20230043869A (ko) 2020-08-07 2023-03-31 스페이스크래프트 세븐, 엘엘씨 Aav 벡터를 사용한 플라코필린-2(pkp2) 유전자 요법
EP4421179A3 (en) 2020-09-10 2024-12-18 Genethon Peptide-modified aav capsid
CN113754727B (zh) * 2020-09-30 2022-07-12 广州派真生物技术有限公司 腺相关病毒突变体及其应用
BR112023006305A2 (pt) * 2020-10-09 2023-05-09 Tenaya Therapeutics Inc Métodos e composições de terapia gênica com placofilina-2
US11781156B2 (en) 2020-10-09 2023-10-10 Tenaya Therapeutics, Inc. Plakophillin-2 gene therapy methods and compositions
AU2021378075B2 (en) 2020-11-11 2025-03-27 Borea Therapeutics S.R.L. Modified viral particles for gene therapy
WO2022114243A1 (en) * 2020-11-25 2022-06-02 Astellas Pharma Inc. Method for treating muscular dystrophy by targeting dmpk gene
US20240115734A1 (en) * 2021-02-09 2024-04-11 Affinia Therapeutics Inc. Recombinant aavs with improved tropism and specificity
WO2023060113A1 (en) 2021-10-05 2023-04-13 Regenxbio Inc. Compositions and methods for recombinant aav production
CN118202060A (zh) 2021-10-05 2024-06-14 再生生物股份有限公司 用于重组aav生产的组合物和方法
KR20240095539A (ko) 2021-10-08 2024-06-25 디노 테라퓨틱스, 인코포레이티드 캡시드 변이체 및 이의 사용 방법
AU2022420596A1 (en) * 2021-12-21 2024-07-11 Research Institute At Nationwide Children's Hospital Materials and methods for the treatment of limb girdle muscular dystrophy
EP4493702A4 (en) * 2022-03-18 2026-04-01 Univ Florida METHODS AND COMPOSITIONS FOR TREATING TNNT2-RELATED CARDIOMYOPATHY WITH A VIRAL VECTOR
WO2023196862A1 (en) 2022-04-06 2023-10-12 Genzyme Corporation Targeted gene therapy for dm-1 myotonic dystrophy
WO2023198828A1 (en) 2022-04-13 2023-10-19 Universitat Autònoma De Barcelona Treatment of neuromuscular diseases via gene therapy that expresses klotho protein
EP4269426A1 (en) * 2022-04-29 2023-11-01 Ospedale San Raffaele S.r.l. Gene therapy
ES3057171T3 (en) 2022-06-10 2026-02-26 Genethon Peptide-modified aav capsid with enhanced muscle transduction efficiency
WO2024191778A1 (en) 2023-03-10 2024-09-19 Dyno Therapeutics, Inc. Capsid polypeptides and methods of use thereof
EP4677073A1 (en) 2023-03-10 2026-01-14 Genethon Induction of immune tolerance by aav vector comprising the combination of a liver detargeted capsid and a tandem liver-muscle specific promoter
EP4680741A2 (en) 2023-03-15 2026-01-21 RegenxBio Inc. Exon skipping gene therapy constructs, vectors and uses thereof
US20240384271A1 (en) 2023-04-05 2024-11-21 Genzyme Corporation Targeted gene therapy for dm-1 myotonic dystrophy
WO2024238807A2 (en) 2023-05-16 2024-11-21 Affinia Therapeutics Inc. Recombinant aavs with improved tropism and specificity
CN121712809A (zh) 2023-07-03 2026-03-20 Ucb生物制药有限责任公司 工程化的aav衣壳蛋白
AU2024332181A1 (en) 2023-08-31 2026-02-12 Dyno Therapeutics, Inc. Capsid polypeptides and methods of use thereof
WO2025133247A1 (en) 2023-12-21 2025-06-26 Genethon Methods for aav vector re-administration
TW202545971A (zh) 2024-02-08 2025-12-01 美商戴諾治療公司 殼體多肽及其使用方法
WO2025232917A1 (en) * 2024-05-10 2025-11-13 Hangzhou Jiayin Biotech Ltd. Novel liver detargeted aav variants and uses thereof
WO2026006341A1 (en) 2024-06-24 2026-01-02 Regenxbio Inc. Microdystrophin gene therapy administration for treatment of dystrophinopathies
WO2026017727A1 (en) 2024-07-16 2026-01-22 Genethon Alternative barcoding method to assess transduction efficiency of aav vectors
WO2026064428A2 (en) 2024-09-18 2026-03-26 Dyno Therapeutics, Inc. Capsid polypeptides and methods of use thereof
WO2026064442A2 (en) 2024-09-18 2026-03-26 Dyno Therapeutics, Inc. Capsid polypeptides and methods of use thereof
WO2026064444A1 (en) 2024-09-18 2026-03-26 Dyno Therapeutics, Inc. Capsid polypeptides and methods of use thereof

Family Cites Families (12)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US6833447B1 (en) * 2000-07-10 2004-12-21 Monsanto Technology, Llc Myxococcus xanthus genome sequences and uses thereof
EP1456383B1 (en) * 2001-12-21 2014-03-12 Medigene AG A library of modified structural genes or capsid modified particles useful for the identification of viral clones with desired cell tropism
US8927269B2 (en) * 2003-05-19 2015-01-06 The United States Of America, As Represented By The Secretary, Department Of Health And Human Services Avian adenoassociated virus and uses thereof
US7467102B2 (en) * 2003-09-11 2008-12-16 International Business Machines Corporation Request type grid computing
US20130305398A1 (en) * 2012-02-16 2013-11-14 Marie Coffin Genes and uses for plant enhacement
WO2013170078A1 (en) * 2012-05-09 2013-11-14 Oregon Health & Science University Adeno associated virus plasmids and vectors
BR112015023272A2 (pt) * 2013-03-14 2017-07-18 Pioneer Hi Bred Int célula vegetal, planta, explante vegetal, semente transgênica, método para produzir uma célula vegetal tendo um polinucleotídeo heterólogo que codifica um polipeptídeo tendo atividade de dicamba descarboxilase, método para controlar plantas daninhas em um campo contendo uma cultura e método para controlar plantas daninhas em um campo contendo uma cultura
EP3384034B1 (en) * 2015-12-02 2020-07-08 The Board of Trustees of the Leland Stanford Junior University Novel recombinant adeno-associated virus capsids with enhanced human skeletal muscle tropism
US20190224339A1 (en) * 2016-04-29 2019-07-25 Voyager Therapeutics, Inc. Compositions for the treatment of disease
WO2018129203A2 (en) * 2017-01-06 2018-07-12 The Regents Of The University Of California Method for temporal and tissue-specific drug delivery and induced nucleic acid recombination
EP3728568A4 (en) * 2017-12-19 2021-10-06 The University of North Carolina at Chapel Hill METHODS AND COMPOSITIONS FOR DELIVERING VIRAL VECTORS BY THE BLOOD-BRAIN BARRIER
MA51353B1 (fr) * 2018-04-05 2022-09-30 Univ Sorbonne Sérotype du virus adéno-associé recombinant hybride entre aav9 et aavrh74 possédant un tropisme hépatique réduit

Cited By (7)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
CN113480615A (zh) * 2021-07-30 2021-10-08 上海信致医药科技有限公司 高视网膜亲和性的新型腺相关病毒衣壳蛋白及其应用
CN113563430A (zh) * 2021-07-30 2021-10-29 上海信致医药科技有限公司 用于治疗眼部疾病的基因递送系统及其应用
CN113563430B (zh) * 2021-07-30 2022-05-31 上海信致医药科技有限公司 用于治疗眼部疾病的基因递送系统及其应用
CN113480615B (zh) * 2021-07-30 2022-05-31 上海信致医药科技有限公司 高视网膜亲和性的新型腺相关病毒衣壳蛋白及其应用
WO2023169115A1 (zh) * 2022-03-11 2023-09-14 上海勉亦生物科技有限公司 一种神经系统高亲和性的aav载体及其应用
CN118005749A (zh) * 2023-07-21 2024-05-10 广州派真生物技术有限公司 腺相关病毒突变体及其应用
CN118005749B (zh) * 2023-07-21 2024-07-16 广州派真生物技术有限公司 腺相关病毒突变体及其应用

Also Published As

Publication number Publication date
AU2026202070A1 (en) 2026-04-09
JP7707261B2 (ja) 2025-07-14
US20210363193A1 (en) 2021-11-25
JP2024020346A (ja) 2024-02-14
AU2019258830A1 (en) 2020-12-03
CA3097375A1 (en) 2019-10-31
WO2019207132A1 (en) 2019-10-31
EP3784288A1 (en) 2021-03-03
JP2021521833A (ja) 2021-08-30
AU2019258830B2 (en) 2025-12-18

Similar Documents

Publication Publication Date Title
JP7707261B2 (ja) 筋疾患の治療のための改変ａａｖカプシドポリペプチド
US11661596B2 (en) Targeted RNA editing by leveraging endogenous ADAR using engineered RNAs
CN111235158B (zh) 用于表达重组人β-珠蛋白的病毒载体及其应用
CN113710693B (zh) Dna结合结构域反式激活因子及其用途
WO2020187268A1 (zh) 一种增强基因编辑的融合蛋白及其应用
WO2022235933A2 (en) Compositions and methods for treating sensorineural hearing loss using stereocilin dual vector systems
WO2023178280A2 (en) Compositions and methods for modulating alpha-synuclein expression
WO2022232442A2 (en) Multiplex crispr/cas9-mediated target gene activation system
CN111718420A (zh) 一种用于基因治疗的融合蛋白及其应用
TW202221119A (zh) Dna結合域轉活化子及其用途
JP2022553824A (ja) 前庭支持細胞プロモーター及びその使用
KR20230003557A (ko) 스펙트린 융합 도메인을 갖는 소형화 디스트로핀 및 그의 용도
US20230078498A1 (en) Targeted Translation of RNA with CRISPR-Cas13 to Enhance Protein Synthesis
WO2024097521A2 (en) Compositions for treatment of osteogenesis imperfecta
WO2020187272A1 (zh) 一种用于基因治疗的融合蛋白及其应用
HK40119094A (zh) 腺相关病毒变异衣壳和其使用方法
WO2025122739A1 (en) Compositions and methods for treating a heart disease
KR20240027748A (ko) Rbm20 돌연변이의 게놈 편집
CN118339290A (zh) 包含基于小分子药物的可变剪接调节元件的核酸分子
HK40109651A (zh) 重组tert编码病毒基因组和运载体
CN118416257A (zh) Mif基因在制备治疗和/或预防心肌缺血损伤的药物中的应用、载体和药物
BR112022000291B1 (pt) Métodos in vitro para editar um ácido ribonucleico (rna) alvo, e, usos de um construto e de um rna de recrutamento de deaminase (drna)

Legal Events

Date Code Title Description
PB01 Publication
PB01 Publication
SE01 Entry into force of request for substantive examination
SE01 Entry into force of request for substantive examination