JP2019523648A5 - - Google Patents

Download PDF

Info

Publication number
JP2019523648A5
JP2019523648A5 JP2018565324A JP2018565324A JP2019523648A5 JP 2019523648 A5 JP2019523648 A5 JP 2019523648A5 JP 2018565324 A JP2018565324 A JP 2018565324A JP 2018565324 A JP2018565324 A JP 2018565324A JP 2019523648 A5 JP2019523648 A5 JP 2019523648A5
Authority
JP
Japan
Prior art keywords
nucleic acid
acid sequence
aav vector
seq
nucleotides
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
JP2018565324A
Other languages
English (en)
Japanese (ja)
Other versions
JP2019523648A (ja
Filing date
Publication date
Priority claimed from GBGB1610448.1A external-priority patent/GB201610448D0/en
Priority claimed from GBGB1707261.2A external-priority patent/GB201707261D0/en
Application filed filed Critical
Priority claimed from PCT/GB2017/051741 external-priority patent/WO2017216560A1/en
Publication of JP2019523648A publication Critical patent/JP2019523648A/ja
Publication of JP2019523648A5 publication Critical patent/JP2019523648A5/ja
Pending legal-status Critical Current

Links

JP2018565324A 2016-06-15 2017-06-14 Abc4aを発現させるためのデュアル重複アデノ随伴ウイルスベクターシステム Pending JP2019523648A (ja)

Applications Claiming Priority (5)

Application Number Priority Date Filing Date Title
GB1610448.1 2016-06-15
GBGB1610448.1A GB201610448D0 (en) 2016-06-15 2016-06-15 Adeno-associated viral vector system
GB1707261.2 2017-05-05
GBGB1707261.2A GB201707261D0 (en) 2017-05-05 2017-05-05 Adeno-associated viral vector system
PCT/GB2017/051741 WO2017216560A1 (en) 2016-06-15 2017-06-14 Dual overlapping adeno-associated viral vector system for expressing abc4a

Publications (2)

Publication Number Publication Date
JP2019523648A JP2019523648A (ja) 2019-08-29
JP2019523648A5 true JP2019523648A5 (enExample) 2020-07-27

Family

ID=59101505

Family Applications (1)

Application Number Title Priority Date Filing Date
JP2018565324A Pending JP2019523648A (ja) 2016-06-15 2017-06-14 Abc4aを発現させるためのデュアル重複アデノ随伴ウイルスベクターシステム

Country Status (13)

Country Link
US (1) US20190309326A1 (enExample)
EP (1) EP3472328A1 (enExample)
JP (1) JP2019523648A (enExample)
KR (1) KR20190020745A (enExample)
CN (1) CN109642242A (enExample)
AU (1) AU2017286623A1 (enExample)
BR (1) BR112018075855A2 (enExample)
CA (1) CA3025445A1 (enExample)
IL (1) IL263523A (enExample)
MX (1) MX2018015629A (enExample)
RU (1) RU2765826C2 (enExample)
SG (1) SG11201811244SA (enExample)
WO (1) WO2017216560A1 (enExample)

Families Citing this family (13)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
ES2704677T3 (es) 2013-04-18 2019-03-19 Fond Telethon Suministro eficaz de genes grandes por vectores de AAV duales
CA3061955A1 (en) 2017-05-05 2018-11-08 University Of Florida Research Foundation, Incorporated Compositions and methods for expressing otoferlin
US20210147870A1 (en) * 2018-04-05 2021-05-20 Nightstarx Limited Compositions and methods for the treatment of stargardt disease
BR112021007221A2 (pt) * 2018-10-15 2021-08-10 Fondazione Telethon proteínas inteínas e seus usos
GB201817469D0 (en) * 2018-10-26 2018-12-12 Univ Oxford Innovation Ltd Gene therapy for retinal disease
US12188041B2 (en) 2018-11-01 2025-01-07 University Of Florida Research Foundation, Incorporated Codon optimized otoferlin AAV dual vector gene therapy
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
US20220267802A1 (en) * 2019-07-15 2022-08-25 President And Fellows Of Harvard College Methods and compositions for gene delivery
AU2021247172A1 (en) 2020-04-01 2022-09-15 University Of Florida Research Foundation, Incorporated Dual AAV-MY07A vectors with improved safety for the treatment of USH1B
US20230338580A1 (en) * 2020-08-14 2023-10-26 Case Western Reserve University Plasmid vectors and nanoparticles for treating ocular disorders
EP4334447A1 (en) * 2021-05-07 2024-03-13 UCL Business Ltd Abca4 genome editing
US20240425876A1 (en) * 2022-02-25 2024-12-26 Chigenovo Co., Ltd. Expression cassette combinations and uses thereof
WO2024257061A1 (en) * 2023-06-16 2024-12-19 Arkasubhra Ghosh A hybrid dual aav vector system with splice enhancer elements for expression of large genes

Family Cites Families (5)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
EP2311967B1 (en) * 2005-10-20 2017-09-20 UniQure IP B.V. Improved AAV vectors produced in insect cells
US20090214478A1 (en) * 2008-02-21 2009-08-27 Alberto Auricchio Method of treating ocular diseases by gene therapy
KR20120052352A (ko) * 2009-08-07 2012-05-23 트랜스진 에스.에이. Hbv 감염을 치료하는 조성물
WO2013075008A1 (en) * 2011-11-16 2013-05-23 University Of Florida Research Foundation Inc. Aav dual vector systems for gene therapy
ES2704677T3 (es) * 2013-04-18 2019-03-19 Fond Telethon Suministro eficaz de genes grandes por vectores de AAV duales

Similar Documents

Publication Publication Date Title
JP2019523648A5 (enExample)
RU2019100525A (ru) Векторная система на основе аденоассоциированного вируса
DE69429260T2 (de) Adenovirale vektoren tierischen ursprungs und ihre verwendung bei der gentherapie
JP2018520646A5 (enExample)
JP2018520997A5 (enExample)
JP2017512466A5 (enExample)
JP2020514286A5 (enExample)
JP2015508760A5 (enExample)
JP2020513814A5 (enExample)
KR20210028162A (ko) 지대근 이영양증 2a형을 치료하기 위한 재조합 아데노 연관 바이러스 생성물 및 방법
JP2016538885A5 (enExample)
Hollinger et al. Viral vector-mediated gene therapies
US20240002462A1 (en) Treatment of neuropathy with igf-1-encoding dna constructs and hgf-encoding dna constructs
JP2019524090A5 (enExample)
JP2018509154A5 (enExample)
JP2017523239A5 (enExample)
KR20250105716A (ko) 간에서의 유전자 발현을 위한 핵산 조절 요소 및 사용 방법
JP2020527167A5 (enExample)
JP2021502978A5 (enExample)
Chauhan et al. AAV-DJ is superior to AAV9 for targeting brain and spinal cord, and de-targeting liver across multiple delivery routes in mice
JP2018526994A5 (enExample)
IL308645A (en) Viral vectors comprising rdh12 coding regions and methods of treating retinal dystrophies
JP2022060514A (ja) ガバペンチノイドからの干渉が減少し、hgf異型体を発現するdnaコンストラクトを用いる神経病症の治療
JP2021500880A5 (enExample)
JP2020517268A5 (enExample)