EA201890543A1 - Способы и материалы для генной терапии galgt2 - Google Patents

Способы и материалы для генной терапии galgt2

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Publication number
EA201890543A1
EA201890543A1 EA201890543A EA201890543A EA201890543A1 EA 201890543 A1 EA201890543 A1 EA 201890543A1 EA 201890543 A EA201890543 A EA 201890543A EA 201890543 A EA201890543 A EA 201890543A EA 201890543 A1 EA201890543 A1 EA 201890543A1
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EA
Eurasian Patent Office
Prior art keywords
methods
galgt2
gene therapy
materials
raav
Prior art date
Application number
EA201890543A
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English (en)
Inventor
Пол Тейлор Мартин
Original Assignee
Рисёрч Инститьют Эт Нэйшнвайд Чилдрен'С Хоспитэл
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Application filed by Рисёрч Инститьют Эт Нэйшнвайд Чилдрен'С Хоспитэл filed Critical Рисёрч Инститьют Эт Нэйшнвайд Чилдрен'С Хоспитэл
Publication of EA201890543A1 publication Critical patent/EA201890543A1/ru

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    • 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
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    • 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
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    • C12N2830/00—Vector systems having a special element relevant for transcription
    • C12N2830/42—Vector systems having a special element relevant for transcription being an intron or intervening sequence for splicing and/or stability of RNA

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Abstract

Настоящее изобретение относится к доставке с помощью рекомбинантного аденоассоциированного вируса (rAAV) полинуклеотида GALGT2. Данное изобретение предусматривает rAAV и способы использования rAAV для генной терапии нервно-мышечных заболеваний с использованием GALGT2. Типичные примеры нервно-мышечных заболеваний включают, без ограничений, мышечные дистрофии, такие как мышечная дистрофия Дюшенна, врожденная мышечная дистрофия 1А и тазово-плечевая мышечная дистрофия 2D.
EA201890543A 2015-09-17 2016-09-16 Способы и материалы для генной терапии galgt2 EA201890543A1 (ru)

Applications Claiming Priority (4)

Application Number Priority Date Filing Date Title
US201562220107P 2015-09-17 2015-09-17
US201562221068P 2015-09-20 2015-09-20
US201662301260P 2016-02-29 2016-02-29
PCT/US2016/052051 WO2017049031A1 (en) 2015-09-17 2016-09-16 Methods and materials for galgt2 gene therapy

Publications (1)

Publication Number Publication Date
EA201890543A1 true EA201890543A1 (ru) 2018-08-31

Family

ID=58289891

Family Applications (1)

Application Number Title Priority Date Filing Date
EA201890543A EA201890543A1 (ru) 2015-09-17 2016-09-16 Способы и материалы для генной терапии galgt2

Country Status (14)

Country Link
US (1) US10980897B2 (ru)
EP (1) EP3350331A4 (ru)
JP (3) JP7338970B2 (ru)
KR (1) KR20180084747A (ru)
CN (1) CN108136049A (ru)
AU (2) AU2016323575B2 (ru)
CA (1) CA2998636A1 (ru)
CO (1) CO2018003960A2 (ru)
EA (1) EA201890543A1 (ru)
HK (1) HK1257519A1 (ru)
IL (2) IL314884A (ru)
MX (2) MX2018003234A (ru)
SA (1) SA518391113B1 (ru)
WO (1) WO2017049031A1 (ru)

Families Citing this family (7)

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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
WO2019078916A1 (en) 2017-10-18 2019-04-25 Research Institute At Nationwide Children's Hospital ADENO-ASSOCIATED VIRUS VECTOR ADMINISTRATION OF MUSCLE-SPECIFIC MICRO-DYSTROPHINE TO TREAT MUSCLE DYSTROPHY
KR20200083495A (ko) 2017-10-20 2020-07-08 더 리서치 인스티튜트 앳 네이션와이드 칠드런스 하스피탈 Nt-3 유전자 치료를 위한 방법 및 물질
WO2019246125A1 (en) * 2018-06-18 2019-12-26 Research Institute At Nationwide Children's Hospital Recombinant adeno-associated virus products and methods for treating dystroglycanopathies and laminin-deficient muscular dystrophies
EP4051324A4 (en) * 2019-10-28 2023-11-29 University Of Florida Research Foundation, Incorporated GENE THERAPY VECTORS
KR20230049670A (ko) * 2020-08-07 2023-04-13 얀센 바이오테크 인코포레이티드 고도로 정제된 바이러스 입자를 위한 제형
EP4230196A1 (en) 2022-02-21 2023-08-23 Som Innovation Biotech, S.A. Compounds for use in the treatment of dystrophinopathies

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Also Published As

Publication number Publication date
US20210220484A1 (en) 2021-07-22
MX2018003234A (es) 2018-09-07
SA518391113B1 (ar) 2022-10-25
CA2998636A1 (en) 2017-03-23
MX2023013509A (es) 2024-01-08
IL258005A (en) 2018-05-31
BR112018005177A2 (pt) 2018-10-09
JP2022010079A (ja) 2022-01-14
JP7338970B2 (ja) 2023-09-05
JP2024109934A (ja) 2024-08-14
US20180250423A1 (en) 2018-09-06
EP3350331A4 (en) 2019-01-23
EP3350331A1 (en) 2018-07-25
CO2018003960A2 (es) 2018-10-10
AU2016323575A1 (en) 2018-04-05
KR20180084747A (ko) 2018-07-25
US10980897B2 (en) 2021-04-20
AU2016323575B2 (en) 2022-10-27
AU2023200499A1 (en) 2023-06-15
HK1257519A1 (zh) 2019-10-25
IL314884A (en) 2024-10-01
JP2018527365A (ja) 2018-09-20
WO2017049031A1 (en) 2017-03-23
IL258005B1 (en) 2024-09-01
CN108136049A (zh) 2018-06-08

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