AU2018261769B2 - Compositions and methods for expressing Otoferlin - Google Patents

Compositions and methods for expressing Otoferlin Download PDF

Info

Publication number
AU2018261769B2
AU2018261769B2 AU2018261769A AU2018261769A AU2018261769B2 AU 2018261769 B2 AU2018261769 B2 AU 2018261769B2 AU 2018261769 A AU2018261769 A AU 2018261769A AU 2018261769 A AU2018261769 A AU 2018261769A AU 2018261769 B2 AU2018261769 B2 AU 2018261769B2
Authority
AU
Australia
Prior art keywords
leu
glu
asp
lys
val
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.)
Active
Application number
AU2018261769A
Other languages
English (en)
Other versions
AU2018261769A1 (en
Inventor
Omar AKIL
Sanford L. Boye
Frank DYKA
William W. Hauswirth
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.)
University of Florida Research Foundation Inc
University of California San Diego UCSD
Original Assignee
University of Florida Research Foundation Inc
University of California Berkeley
University of California San Diego UCSD
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
Family has litigation
First worldwide family litigation filed litigation Critical https://patents.darts-ip.com/?family=64016253&utm_source=google_patent&utm_medium=platform_link&utm_campaign=public_patent_search&patent=AU2018261769(B2) "Global patent litigation dataset” by Darts-ip is licensed under a Creative Commons Attribution 4.0 International License.
Application filed by University of Florida Research Foundation Inc, University of California Berkeley, University of California San Diego UCSD filed Critical University of Florida Research Foundation Inc
Publication of AU2018261769A1 publication Critical patent/AU2018261769A1/en
Application granted granted Critical
Publication of AU2018261769B2 publication Critical patent/AU2018261769B2/en
Priority to AU2024203084A priority Critical patent/AU2024203084B2/en
Active legal-status Critical Current
Anticipated expiration legal-status Critical

Links

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
    • 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
    • C12N15/87—Introduction of foreign genetic material using processes not otherwise provided for, e.g. co-transformation
    • C12N15/90—Stable introduction of foreign DNA into chromosome
    • C12N15/902—Stable introduction of foreign DNA into chromosome using homologous recombination
    • 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
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P27/00—Drugs for disorders of the senses
    • A61P27/02—Ophthalmic agents
    • 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
    • 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
    • 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
    • A01K2217/00—Genetically modified animals
    • A01K2217/07—Animals genetically altered by homologous recombination
    • A01K2217/075—Animals genetically altered by homologous recombination inducing loss of function, i.e. knock out
    • 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
    • C12N2800/00—Nucleic acids vectors
    • C12N2800/40—Systems of functionally co-operating 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
    • C12N2840/00—Vectors comprising a special translation-regulating system
    • C12N2840/44—Vectors comprising a special translation-regulating system being a specific part of the splice mechanism, e.g. donor, acceptor
    • C12N2840/445—Vectors comprising a special translation-regulating system being a specific part of the splice mechanism, e.g. donor, acceptor for trans-splicing, e.g. polypyrimidine tract, branch point splicing

Landscapes

  • Health & Medical Sciences (AREA)
  • Life Sciences & Earth Sciences (AREA)
  • Chemical & Material Sciences (AREA)
  • Genetics & Genomics (AREA)
  • Engineering & Computer Science (AREA)
  • Organic Chemistry (AREA)
  • Zoology (AREA)
  • General Health & Medical Sciences (AREA)
  • Molecular Biology (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • Biotechnology (AREA)
  • Biomedical Technology (AREA)
  • Biochemistry (AREA)
  • General Engineering & Computer Science (AREA)
  • Wood Science & Technology (AREA)
  • Biophysics (AREA)
  • Medicinal Chemistry (AREA)
  • Proteomics, Peptides & Aminoacids (AREA)
  • Gastroenterology & Hepatology (AREA)
  • Microbiology (AREA)
  • Plant Pathology (AREA)
  • Physics & Mathematics (AREA)
  • Toxicology (AREA)
  • Public Health (AREA)
  • Veterinary Medicine (AREA)
  • Animal Behavior & Ethology (AREA)
  • Pharmacology & Pharmacy (AREA)
  • Virology (AREA)
  • Epidemiology (AREA)
  • Mycology (AREA)
  • Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
  • Marine Sciences & Fisheries (AREA)
  • Chemical Kinetics & Catalysis (AREA)
  • General Chemical & Material Sciences (AREA)
  • Ophthalmology & Optometry (AREA)
  • Immunology (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)
  • Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)
AU2018261769A 2017-05-05 2018-05-04 Compositions and methods for expressing Otoferlin Active AU2018261769B2 (en)

Priority Applications (1)

Application Number Priority Date Filing Date Title
AU2024203084A AU2024203084B2 (en) 2017-05-05 2024-05-09 Compositions and methods for expressing Otoferlin

Applications Claiming Priority (3)

Application Number Priority Date Filing Date Title
US201762502462P 2017-05-05 2017-05-05
US62/502,462 2017-05-05
PCT/US2018/031009 WO2018204734A1 (en) 2017-05-05 2018-05-04 Compositions and methods for expressing otoferlin

Related Child Applications (1)

Application Number Title Priority Date Filing Date
AU2024203084A Division AU2024203084B2 (en) 2017-05-05 2024-05-09 Compositions and methods for expressing Otoferlin

Publications (2)

Publication Number Publication Date
AU2018261769A1 AU2018261769A1 (en) 2019-11-21
AU2018261769B2 true AU2018261769B2 (en) 2024-03-14

Family

ID=64016253

Family Applications (2)

Application Number Title Priority Date Filing Date
AU2018261769A Active AU2018261769B2 (en) 2017-05-05 2018-05-04 Compositions and methods for expressing Otoferlin
AU2024203084A Active AU2024203084B2 (en) 2017-05-05 2024-05-09 Compositions and methods for expressing Otoferlin

Family Applications After (1)

Application Number Title Priority Date Filing Date
AU2024203084A Active AU2024203084B2 (en) 2017-05-05 2024-05-09 Compositions and methods for expressing Otoferlin

Country Status (9)

Country Link
US (2) US12359221B2 (https=)
EP (1) EP3635100A4 (https=)
JP (3) JP7240675B2 (https=)
KR (2) KR102606810B1 (https=)
CN (1) CN110892062A (https=)
AU (2) AU2018261769B2 (https=)
CA (1) CA3061955A1 (https=)
MX (2) MX2019013151A (https=)
WO (1) WO2018204734A1 (https=)

Families Citing this family (18)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
WO2013075008A1 (en) 2011-11-16 2013-05-23 University Of Florida Research Foundation Inc. Aav dual vector systems for gene therapy
EP3510161A4 (en) 2016-08-23 2020-04-22 Akouos, Inc. COMPOSITIONS AND METHODS FOR TREATING PERSONAL HEARING LOSS IN A PERSON
KR102606810B1 (ko) 2017-05-05 2023-11-27 유니버시티 오브 플로리다 리서치 파운데이션, 인코포레이티드 오토펄린을 발현시키기 위한 조성물 및 방법
DE102018103924A1 (de) * 2018-02-21 2019-08-22 Georg-August-Universität Göttingen Stiftung Öffentlichen Rechts, Universitätsmedizin Gentherapeutische Behandlung von Schwerhörigkeit
IL325122A (en) * 2018-02-22 2026-02-01 Akouos Inc Compositions and methods for treating non-age-related hearing impairment in humans
US11660353B2 (en) 2018-04-27 2023-05-30 Decibel Therapeutics, Inc. Compositions and methods for treating sensorineural hearing loss using otoferlin dual vector systems
CN112703016B (zh) 2018-04-27 2024-11-22 分贝治疗公司 肌球蛋白15启动子及其用途
WO2020093018A1 (en) * 2018-11-01 2020-05-07 University Of Florida Research Foundation, Incorporated A codon optimized otoferlin aav dual vector gene therapy
EP4295913A3 (en) * 2019-01-18 2024-03-06 Institut Pasteur Aav-mediated gene therapy restoring the otoferlin gene
EP3921032A4 (en) * 2019-02-08 2022-11-30 Decibel Therapeutics, Inc. COMPOSITIONS AND METHODS FOR TREATMENT OF SENSORINEURAL HEARING LOSS USING OTOFERLIN DOUBLE VECTOR SYSTEMS
CA3129422A1 (en) 2019-02-08 2020-08-13 Decibel Therapeutics, Inc. Myosin 15 promoters and uses thereof
KR20220130093A (ko) * 2019-10-30 2022-09-26 데시벨 테라퓨틱스, 인크. 오토펄린 듀얼 벡터 시스템을 사용한 감각신경성 난청을 치료하기 위한 조성물 및 방법
CN115066432B (zh) * 2019-10-30 2026-04-17 分贝治疗公司 使用耳畸蛋白双载体系统治疗感觉神经性听力损失的组合物和方法
KR20220157944A (ko) 2020-02-21 2022-11-29 아카우오스, 인크. 인간 대상체에서 비-연령-연관 청각 장애를 치료하기 위한 조성물 및 방법
CN115666658A (zh) 2020-04-01 2023-01-31 佛罗里达州大学研究基金会 具有提高的治疗ush1b的安全性的双aav-myo7a载体
CN112125969A (zh) * 2020-09-30 2020-12-25 东南大学 生物因子rimbp2在维持内耳毛细胞特性上的应用
CN116925239B (zh) * 2023-07-17 2024-10-18 苏州星奥拓维生物技术有限公司 双载体系统表达Otof基因的组合物和方法
WO2025201333A1 (zh) * 2024-03-28 2025-10-02 苏州星奥拓维生物技术有限公司 递送Otof基因的双载体系统和其用途

Citations (3)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US20100003218A1 (en) * 2008-05-28 2010-01-07 Dongsheng Duan Hybrid-aav vectors to deliver large gene expression cassette
WO2016139321A1 (en) * 2015-03-03 2016-09-09 Fondazione Telethon Multiple vector system and uses thereof
WO2018039375A1 (en) * 2016-08-23 2018-03-01 Akouos, Inc. Compositions and methods for treating non-age-associated hearing impairment in a human subject

Family Cites Families (28)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
AU4445701A (en) * 2000-03-24 2001-10-03 Centre National De La Recherche Scientifique-Cnrs Multiple human and mouse otoferlin isoforms
CN1934260B (zh) 2004-01-22 2013-03-13 株式会社载体研究所 利用含有巨细胞病毒增强子和鸡β-肌动蛋白启动子的杂合启动子制造负链RNA病毒载体的方法
WO2007127428A2 (en) 2006-04-28 2007-11-08 University Of Florida Research Foundation, Inc. Double-stranded/self-complementary vectors with a truncated cba promoter and methods of gene delivery
AU2007284651B2 (en) 2006-08-09 2014-03-20 Institute For Systems Biology Organ-specific proteins and methods of their use
CN101711164B (zh) 2007-01-18 2014-06-04 密苏里-哥伦比亚大学 可修复肌纤维膜中的神经元型一氧化氮合酶的合成小/微-抗肌萎缩蛋白基因
FR2919305B1 (fr) 2007-07-26 2009-09-18 Genethon Ass Loi De 1901 Vecteurs viraux adeno-associes pour l'expression de la dysferline.
WO2010062905A2 (en) * 2008-11-26 2010-06-03 The Regents Of The University Of California Lateral ventricle cell compositions and use for treating neural degenerative diseases
SG10201800541SA (en) * 2010-04-23 2018-03-28 Univ Florida Raav-guanylate cyclase compositions and methods for treating leber's congenital amaurosis-1 (lca1)
WO2013075008A1 (en) 2011-11-16 2013-05-23 University Of Florida Research Foundation Inc. Aav dual vector systems for gene therapy
WO2013151665A2 (en) 2012-04-02 2013-10-10 modeRNA Therapeutics Modified polynucleotides for the production of proteins associated with human disease
EP2970972B1 (en) 2013-03-11 2018-08-15 Fondazione Telethon Mir-204 and mir-211 and uses thereof
AU2014255665B2 (en) 2013-04-18 2018-08-02 Fondazione Telethon Effective delivery of large genes by dual AAV vectors
EP3492597A3 (en) 2013-05-21 2019-08-28 University of Florida Research Foundation, Inc. Capsid-modified, raav3 vector compositions and methods of use in gene therapy of human liver cancer
US11000597B2 (en) 2015-01-21 2021-05-11 University Of Florida Research Foundation, Incorporated Engineered receptor/ligand system for delivery of therapeutic agents
EP3258955A1 (en) * 2015-02-20 2017-12-27 Institut Pasteur Prevention and/or treatment of hearing loss or impairment
AU2016324317A1 (en) 2015-09-17 2018-03-08 Coda Biotherapeutics, Inc. Compositions and methods for treating neurological disorders
SG10201913266UA (en) 2015-12-11 2020-02-27 Massachusetts Eye & Ear Infirmary Materials and methods for delivering nucleic acids to cochlear and vestibular cells
JP7007273B2 (ja) 2015-12-22 2022-01-24 アンセルム（アンスティチュート・ナシオナル・ドゥ・ラ・サンテ・エ・ドゥ・ラ・ルシェルシュ・メディカル） 遺伝子治療用の改良された複合型二重組換えａａｖベクターシステム
EP3472328A1 (en) 2016-06-15 2019-04-24 Oxford University Innovation Limited Dual overlapping adeno-associated viral vector system for expressing abc4a
WO2018162748A1 (en) 2017-03-10 2018-09-13 Genethon Treatment of glycogen storage disease iii
KR102606810B1 (ko) 2017-05-05 2023-11-27 유니버시티 오브 플로리다 리서치 파운데이션, 인코포레이티드 오토펄린을 발현시키기 위한 조성물 및 방법
IL325122A (en) 2018-02-22 2026-02-01 Akouos Inc Compositions and methods for treating non-age-related hearing impairment in humans
BR112020019079A2 (pt) 2018-03-23 2020-12-29 Massachusetts Eye And Ear Infirmary Abordagem de salto de éxon mediada por crispr/cas9 para síndrome de usher associada a ush2a
WO2020093018A1 (en) 2018-11-01 2020-05-07 University Of Florida Research Foundation, Incorporated A codon optimized otoferlin aav dual vector gene therapy
EP4295913A3 (en) 2019-01-18 2024-03-06 Institut Pasteur Aav-mediated gene therapy restoring the otoferlin gene
KR20220130093A (ko) 2019-10-30 2022-09-26 데시벨 테라퓨틱스, 인크. 오토펄린 듀얼 벡터 시스템을 사용한 감각신경성 난청을 치료하기 위한 조성물 및 방법
KR20220157944A (ko) 2020-02-21 2022-11-29 아카우오스, 인크. 인간 대상체에서 비-연령-연관 청각 장애를 치료하기 위한 조성물 및 방법
CN115666658A (zh) 2020-04-01 2023-01-31 佛罗里达州大学研究基金会 具有提高的治疗ush1b的安全性的双aav-myo7a载体

Patent Citations (3)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US20100003218A1 (en) * 2008-05-28 2010-01-07 Dongsheng Duan Hybrid-aav vectors to deliver large gene expression cassette
WO2016139321A1 (en) * 2015-03-03 2016-09-09 Fondazione Telethon Multiple vector system and uses thereof
WO2018039375A1 (en) * 2016-08-23 2018-03-01 Akouos, Inc. Compositions and methods for treating non-age-associated hearing impairment in a human subject

Non-Patent Citations (2)

* Cited by examiner, † Cited by third party
Title
ALEMI ET AL: "AOS research grant: Restoration of Hearing in the Otoferliu Knockout Mouse using Viral Gene Therapy", vol. 145th, 21 April 2012 (2012-04-21), pages 68, XP009513930, Retrieved from the Internet *
ARKASUBHRA GHOSH ET AL: "Efficient Transgene Reconstitution with Hybrid Dual AAV Vectors Carrying the Minimized Bridging Sequences", HUMAN GENE THERAPY, vol. 22, no. 1, 1 January 2011 (2011-01-01), GB, pages 77 - 83 *

Also Published As

Publication number Publication date
JP2023070682A (ja) 2023-05-19
JP2025063057A (ja) 2025-04-15
KR102606810B1 (ko) 2023-11-27
JP7851563B2 (ja) 2026-04-27
MX2019013151A (es) 2020-02-05
MX2023013435A (es) 2023-12-12
AU2024203084A1 (en) 2024-05-30
EP3635100A4 (en) 2021-03-24
CN110892062A (zh) 2020-03-17
KR20230167138A (ko) 2023-12-07
WO2018204734A8 (en) 2018-12-06
US20200157573A1 (en) 2020-05-21
US12359221B2 (en) 2025-07-15
JP7240675B2 (ja) 2023-03-16
KR20200003881A (ko) 2020-01-10
JP2020518268A (ja) 2020-06-25
CA3061955A1 (en) 2018-11-08
US20250367325A1 (en) 2025-12-04
WO2018204734A1 (en) 2018-11-08
AU2018261769A1 (en) 2019-11-21
JP7617600B2 (ja) 2025-01-20
EP3635100A1 (en) 2020-04-15
AU2024203084B2 (en) 2026-04-02

Similar Documents

Publication Publication Date Title
AU2018261769B2 (en) Compositions and methods for expressing Otoferlin
KR102946735B1 (ko) 오토페를린 유전자를 회복시키는 aav-매개된 유전자 치료요법
KR102446169B1 (ko) 리소좀 저장 장애의 치료를 위한 아데노연관 바이러스 벡터
CN109863243B (zh) 用于治疗粘多糖症的腺相关病毒载体
ES2819976T3 (es) Composiciones y usos médicos para la reprogramación de TCR con proteínas de fusión
CA2462455C (en) Development of a preventive vaccine for filovirus infection in primates
CN108138201A (zh) 包含2a肽的重组载体
KR20190025910A (ko) 조류 숙주 세포에 대한 crispr-cas 시스템
KR20230054840A (ko) rAAV 비리온의 유도 생산을 위한 안정화된 세포주
KR20220016485A (ko) 미엘린 단백질 제로 프로모터를 갖는 aav 벡터, 및 샤르코-마리-투스 질환과 같은 슈반 세포-관련 질환을 치료하기 위한 이의 용도
AU2016378480A1 (en) Endothelium-specific nucleic acid regulatory elements and methods and use thereof
KR20220121844A (ko) 유전자의 발현을 동시에 조절하기 위한 조성물 및 방법
KR20110126586A (ko) 단일 재조합 계 및 그의 사용 방법
KR20240021906A (ko) 발현 벡터, 박테리아 서열-무함유 벡터, 및 이를 제조하고 사용하는 방법
KR102346159B1 (ko) 고효율 발현 벡터 및 이의 용도
AU2017233862B2 (en) Methods and compositions for increased double stranded RNA production
KR20200104343A (ko) 라싸 백신
CN114364792B (zh) 溶瘤牛痘病毒
CN117881788A (zh) 表达载体、无细菌序列载体及其制备和使用方法
KR102749159B1 (ko) 재조합 구제역 바이러스 및 이를 포함하는 구제역 백신 조성물
KR20240023100A (ko) 유전자 발현을 조절하기 위한 조성물 및 방법
KR20240024172A (ko) 유전자의 발현을 조절하기 위한 조성물 및 방법
HK40111561A (zh) 表达载体、无细菌序列载体及其制备和使用方法
HK40061400A (en) Recombinant vectors comprising genes for binding domains and secretable peptides
CN113677800A (zh) 包含用于结合结构域和可分泌肽的基因的重组载体