KR20210105390A - 글리코겐 저장 질환을 치료하기 위한 방법 및 조성물 - Google Patents

글리코겐 저장 질환을 치료하기 위한 방법 및 조성물 Download PDF

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KR20210105390A
KR20210105390A KR1020217022096A KR20217022096A KR20210105390A KR 20210105390 A KR20210105390 A KR 20210105390A KR 1020217022096 A KR1020217022096 A KR 1020217022096A KR 20217022096 A KR20217022096 A KR 20217022096A KR 20210105390 A KR20210105390 A KR 20210105390A
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raav
sequence
seq
nucleic acid
aav
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크리스토퍼 티퍼
켈리 리드 클락
사무엘 워즈워스
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울트라제닉스 파마수티컬 인코포레이티드
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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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    • C12N2830/00—Vector systems having a special element relevant for transcription
    • C12N2830/50—Vector systems having a special element relevant for transcription regulating RNA stability, not being an intron, e.g. poly A signal

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  • Animal Behavior & Ethology (AREA)
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  • Veterinary Medicine (AREA)
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  • Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
KR1020217022096A 2018-12-18 2019-12-18 글리코겐 저장 질환을 치료하기 위한 방법 및 조성물 Pending KR20210105390A (ko)

Applications Claiming Priority (3)

Application Number Priority Date Filing Date Title
US201862781380P 2018-12-18 2018-12-18
US62/781,380 2018-12-18
PCT/US2019/067247 WO2020132115A1 (en) 2018-12-18 2019-12-18 Methods and compositions for treating glycogen storage diseases

Publications (1)

Publication Number Publication Date
KR20210105390A true KR20210105390A (ko) 2021-08-26

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KR1020217022096A Pending KR20210105390A (ko) 2018-12-18 2019-12-18 글리코겐 저장 질환을 치료하기 위한 방법 및 조성물

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US (1) US20220017922A1 (https=)
EP (1) EP3898981B1 (https=)
JP (1) JP7486274B2 (https=)
KR (1) KR20210105390A (https=)
CN (1) CN113454226B (https=)
AU (1) AU2019405758B2 (https=)
BR (1) BR112021012104A2 (https=)
CA (1) CA3123841A1 (https=)
DK (1) DK3898981T5 (https=)
ES (1) ES2969222T3 (https=)
IL (1) IL283956A (https=)
MX (1) MX2021007379A (https=)
PL (1) PL3898981T3 (https=)
PT (1) PT3898981T (https=)
WO (1) WO2020132115A1 (https=)

Families Citing this family (1)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US20250222134A1 (en) * 2021-10-19 2025-07-10 University Of Connecticut Compositions and methods for the treatment of glycogen storage disorders

Family Cites Families (11)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US6759050B1 (en) 1998-12-03 2004-07-06 Avigen, Inc. Excipients for use in adeno-associated virus pharmaceutical formulations, and pharmaceutical formulations made therewith
JP2002191363A (ja) * 1999-07-29 2002-07-09 Herikkusu Kenkyusho:Kk 全長ｃＤＮＡ合成用プライマー、およびその用途
DE602004030327D1 (de) 2003-05-21 2011-01-13 Genzyme Corp Verfahren zur herstellung von präparationen rekombinanter aav-virionen, die weitgehend frei von leeren capsiden sind
DK2242840T3 (da) 2008-01-29 2019-10-21 Applied Genetic Tech Corporation Produktion af rekombinant adeno-associeret virus under anvendelse af bhk-celler i suspension
US8679837B2 (en) 2009-04-02 2014-03-25 University Of Florida Research Foundation, Inc. Inducible system for highly efficient production of recombinant Adeno-associated virus (rAAV) vectors
DK2529020T3 (en) 2010-01-28 2018-08-06 Childrens Hospital Philadelphia SCALABLE PREPARATION PLATF FOR CLEANING VIRAL VECTORS AND CLEANED VIRAL VECTORS FOR USE IN GENTHERAPY
AU2014216160B2 (en) 2013-02-15 2017-03-30 The United States Of America, As Represented By The Secretary, Department Of Health And Human Services AAV8 retinoschisin expression vector for treating X-linked retinoschisis
WO2015081101A1 (en) * 2013-11-26 2015-06-04 The United States Of America, As Represented By The Secretary Department Of Health And Human Services Adeno-associated virus vectors for treatment of glycogen storage disease
JP6824169B2 (ja) 2014-12-23 2021-02-03 ザ ユナイテッド ステイツ オブ アメリカ， アズ リプレゼンテッド バイ ザ セクレタリー， デパートメント オブ ヘルス アンド ヒューマン サービシーズ 改変ｇ６ｐｃをコードするアデノ随伴ウイルスベクターおよびその使用
PL3436593T3 (pl) 2016-03-28 2023-03-27 Ultragenyx Pharmaceutical Inc. Sposoby inaktywacji termicznej adenowirusów
AU2018212002A1 (en) * 2017-01-30 2019-08-29 The United States Of America, As Represented By The Secretary, Department Of Health And Human Services Recombinant virus vectors for the treatment of glycogen storage disease

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PL3898981T3 (pl) 2024-04-02
US20220017922A1 (en) 2022-01-20
ES2969222T3 (es) 2024-05-17
AU2019405758B2 (en) 2026-02-05
DK3898981T3 (da) 2024-01-22
IL283956A (en) 2021-07-29
AU2019405758A2 (en) 2022-05-12
CN113454226B (zh) 2025-10-31
PT3898981T (pt) 2023-12-28
CN113454226A (zh) 2021-09-28
EP3898981A1 (en) 2021-10-27
WO2020132115A1 (en) 2020-06-25
EP3898981B1 (en) 2023-10-18
JP7486274B2 (ja) 2024-05-17
MX2021007379A (es) 2021-07-15
JP2022514271A (ja) 2022-02-10
CA3123841A1 (en) 2020-06-25
DK3898981T5 (da) 2024-08-05
BR112021012104A2 (pt) 2021-09-08
WO2020132115A8 (en) 2021-05-14
AU2019405758A1 (en) 2021-07-01

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