RU2765826C2 - Векторная система на основе аденоассоциированного вируса - Google Patents

Векторная система на основе аденоассоциированного вируса Download PDF

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RU2765826C2
RU2765826C2 RU2019100525A RU2019100525A RU2765826C2 RU 2765826 C2 RU2765826 C2 RU 2765826C2 RU 2019100525 A RU2019100525 A RU 2019100525A RU 2019100525 A RU2019100525 A RU 2019100525A RU 2765826 C2 RU2765826 C2 RU 2765826C2
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nucleic acid
abca4
seq
acid sequence
sequence
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RU2019100525A
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Russian (ru)
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RU2019100525A3 (enExample
RU2019100525A (ru
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Роберт МАКЛАРЕН
Мишелль МАККЛЕМЕНТС
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Охфорд Юниверсити Инновейшн Лимитед
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Priority claimed from GBGB1707261.2A external-priority patent/GB201707261D0/en
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    • C12N15/85—Vectors or expression systems specially adapted for eukaryotic hosts for animal cells
    • C12N15/86—Viral vectors
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    • 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
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    • C07K14/435—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from animals; from humans
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    • 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
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    • 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
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    • 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
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    • 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
    • C12N2750/14171—Demonstrated in vivo effect

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  • Ophthalmology & Optometry (AREA)
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  • Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
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  • Micro-Organisms Or Cultivation Processes Thereof (AREA)
  • Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
  • Medicines Containing Material From Animals Or Micro-Organisms (AREA)
  • Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)
RU2019100525A 2016-06-15 2017-06-14 Векторная система на основе аденоассоциированного вируса RU2765826C2 (ru)

Applications Claiming Priority (5)

Application Number Priority Date Filing Date Title
GBGB1610448.1A GB201610448D0 (en) 2016-06-15 2016-06-15 Adeno-associated viral vector system
GB1610448.1 2016-06-15
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 (3)

Publication Number Publication Date
RU2019100525A3 RU2019100525A3 (enExample) 2020-07-15
RU2019100525A RU2019100525A (ru) 2020-07-15
RU2765826C2 true RU2765826C2 (ru) 2022-02-03

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RU2019100525A RU2765826C2 (ru) 2016-06-15 2017-06-14 Векторная система на основе аденоассоциированного вируса

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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 (14)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
DK2986635T3 (en) 2013-04-18 2019-01-28 Fond Telethon EFFECTIVE DELIVERY OF BIG GENES THROUGH DUAL-AAV VECTORS
US12359221B2 (en) 2017-05-05 2025-07-15 The Regents Of The University Of California 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
CA3116606A1 (en) * 2018-10-15 2020-04-23 Fondazione Telethon Intein proteins and uses thereof
GB201817469D0 (en) * 2018-10-26 2018-12-12 Univ Oxford Innovation Ltd Gene therapy for retinal disease
WO2020093018A1 (en) 2018-11-01 2020-05-07 University Of Florida Research Foundation, Incorporated A 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
WO2021015997A1 (en) * 2019-07-15 2021-01-28 President And Fellows Of Harvard College Methods and compositions for gene delivery
TWI896570B (zh) 2019-10-30 2025-09-11 美商迪賽博治療公司 使用耳畸蛋白雙載體系統治療感覺神經性聽力損失之組合物及方法
IL295741A (en) * 2020-04-01 2022-10-01 Univ Florida Dual aav-myo7a 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
US20240425877A1 (en) * 2021-05-07 2024-12-26 Ucl Business Ltd Abca4 genome editing
WO2023160454A1 (zh) * 2022-02-25 2023-08-31 北京中因科技有限公司 表达盒组合及其应用
EP4728080A1 (en) * 2023-06-16 2026-04-22 Arkasubhra Ghosh A hybrid dual aav vector system with splice enhancer elements for expression of large genes

Citations (2)

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WO2014170480A1 (en) * 2013-04-18 2014-10-23 Fondazione Telethon Effective delivery of large genes by dual aav vectors
RU2555346C2 (ru) * 2009-08-07 2015-07-10 Трансген Са Композиция для лечения инфекции вирусом гепатита в

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PT3272872T (pt) * 2005-10-20 2020-06-26 Uniqure Ip Bv Vetores aav melhorados produzidos em células de insetos
US20090214478A1 (en) * 2008-02-21 2009-08-27 Alberto Auricchio Method of treating ocular diseases by gene therapy
WO2013075008A1 (en) * 2011-11-16 2013-05-23 University Of Florida Research Foundation Inc. Aav dual vector systems for gene therapy

Patent Citations (2)

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RU2555346C2 (ru) * 2009-08-07 2015-07-10 Трансген Са Композиция для лечения инфекции вирусом гепатита в
WO2014170480A1 (en) * 2013-04-18 2014-10-23 Fondazione Telethon Effective delivery of large genes by dual aav vectors

Non-Patent Citations (2)

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Title
IVANA TRAPANI et al., "Effective delivery of large genes to the retina by dual AAV vectors", EMBO MOLECULAR MEDICINE 2014,Vol. 6, No. 2, 16 December 2013 (2013-12-16), page n/a-n/a, XP055127533. *
IVANA TRAPANI ET AL: "Effective delivery of large genes to the retina by dual AAV vectors", EMBO MOLECULAR MEDICINE, WILEY-BLACKWELL, US, 1 December 2013 (2013-12-01), US , pages 194 - 211, XP055127533, ISSN: 1757-4676, DOI: 10.1002/emmm.201302948 *

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US20190309326A1 (en) 2019-10-10
IL263523A (en) 2019-01-31
WO2017216560A1 (en) 2017-12-21
BR112018075855A2 (pt) 2019-04-02
RU2019100525A3 (enExample) 2020-07-15
CA3025445A1 (en) 2017-12-21
KR20190020745A (ko) 2019-03-04
CN109642242A (zh) 2019-04-16
JP2019523648A (ja) 2019-08-29
RU2019100525A (ru) 2020-07-15
AU2017286623A1 (en) 2018-12-20
EP3472328A1 (en) 2019-04-24
MX2018015629A (es) 2019-09-26
SG11201811244SA (en) 2019-01-30

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