CN107636153B - 编码经修饰的g6pc的腺相关病毒载体及其用途 - Google Patents

编码经修饰的g6pc的腺相关病毒载体及其用途 Download PDF

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CN107636153B
CN107636153B CN201580076462.4A CN201580076462A CN107636153B CN 107636153 B CN107636153 B CN 107636153B CN 201580076462 A CN201580076462 A CN 201580076462A CN 107636153 B CN107636153 B CN 107636153B
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g6pc
g6pase
vector
alpha
seq
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CN107636153A (zh
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J·J·周
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US Department of Health and Human Services
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CN201580076462.4A 2014-12-23 2015-12-22 编码经修饰的g6pc的腺相关病毒载体及其用途 Active CN107636153B (zh)

Applications Claiming Priority (3)

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US201462096400P 2014-12-23 2014-12-23
US62/096,400 2014-12-23
PCT/US2015/067338 WO2016106303A1 (en) 2014-12-23 2015-12-22 Adeno-associated virus vectors encoding modified g6pc and uses thereof

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CN107636153A CN107636153A (zh) 2018-01-26
CN107636153B true CN107636153B (zh) 2020-12-11

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US (3) US10415044B2 (https=)
EP (1) EP3236984B1 (https=)
JP (2) JP6824169B2 (https=)
CN (1) CN107636153B (https=)
CA (1) CA2972038C (https=)
ES (1) ES2824829T3 (https=)
IL (1) IL253103B (https=)
WO (1) WO2016106303A1 (https=)

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WO2016106303A1 (en) 2014-12-23 2016-06-30 THE UNITED STATES OF AMERICA, as representd by THE SECRETARY, DEPARTMENT OF HEALTH AND HUMAN SERVICES Adeno-associated virus vectors encoding modified g6pc and uses thereof
CA3050917A1 (en) * 2017-01-30 2018-08-02 The Government Of 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
EP3594346A4 (en) 2017-03-10 2020-12-16 National Center For Child Health And Development ANTISENS OLIGONUCLEOTIDE AND COMPOSITION FOR THE PREVENTION OR TREATMENT OF TYPE IA GLYCOGEN STORAGE DISEASE
KR102398295B1 (ko) 2018-03-09 2022-05-17 다이이찌 산쿄 가부시키가이샤 당원병 Ia형 치료약
CN112654698A (zh) * 2018-08-08 2021-04-13 吉尼松公司 用于治疗糖原贮积病iii的小型gde
MX2021003015A (es) * 2018-09-13 2021-07-15 Modernatx Inc Polinucleotidos que codifican glucosa-6-fosfatasa para el tratamiento de la glucogenosis.
BR112021012104A2 (pt) * 2018-12-18 2021-09-08 Ultragenyx Pharmaceutical Inc. Métodos e composições para o tratamento de doenças de armazenamento de glicogênio
EP3913060A1 (en) * 2020-05-22 2021-11-24 Genethon Vectors encoding a glucose-6-phosphatase (g6pase-a) for gene therapy
CN111972355A (zh) * 2020-08-31 2020-11-24 中国医学科学院北京协和医院 GSDIa型糖原累积症小鼠模型及其构建方法
EP4504947A4 (en) * 2022-04-07 2026-03-25 Univ Duke COMPOSITIONS AND METHODS TO PROMOTE LIVER REGENERATION BY GENE EDITING IN METABOLIC LIVER DISEASE
WO2025165616A1 (en) * 2024-01-31 2025-08-07 The Johns Hopkins University Compositions and methods for treating and preventing cancer

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US6759050B1 (en) 1998-12-03 2004-07-06 Avigen, Inc. Excipients for use in adeno-associated virus pharmaceutical formulations, and pharmaceutical formulations made therewith
US6627425B1 (en) * 2000-06-02 2003-09-30 Millennium Pharmaceuticals, Inc. Human glucose-6-phosphatase molecules and uses thereof
US7261544B2 (en) 2003-05-21 2007-08-28 Genzyme Corporation Methods for producing preparations of recombinant AAV virions substantially free of empty capsids
ES2751999T3 (es) 2008-01-29 2020-04-02 Applied Genetic Tech Corporation Producción recombinante de virus adeno-asociados usando células BHK en suspensión
US20120093775A1 (en) 2009-03-27 2012-04-19 Proyecto De Biomedicina Cima, S.L. Methods and compositions for the treatment of cirrhosis and liver fibrosis
WO2010114948A2 (en) 2009-04-02 2010-10-07 University Of Florida Research Foundation, Inc. An inducible system for highly efficient production of recombinant adeno-associated virus (raav) vectors
WO2011094198A1 (en) 2010-01-28 2011-08-04 The Children's Hospital Of Philadelphia Research Institute, Abramson Research Center A scalable manufacturing platform for viral vector purification and viral vectors so purified for use in gene therapy
AU2014354839B2 (en) 2013-11-26 2020-06-04 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
WO2016106303A1 (en) 2014-12-23 2016-06-30 THE UNITED STATES OF AMERICA, as representd by THE SECRETARY, DEPARTMENT OF HEALTH AND HUMAN SERVICES Adeno-associated virus vectors encoding modified g6pc and uses thereof

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Publication number Publication date
US10415044B2 (en) 2019-09-17
US20190345502A1 (en) 2019-11-14
US20170362670A1 (en) 2017-12-21
EP3236984A1 (en) 2017-11-01
JP2020054367A (ja) 2020-04-09
US20230144834A1 (en) 2023-05-11
IL253103B (en) 2019-12-31
WO2016106303A1 (en) 2016-06-30
CA2972038C (en) 2023-05-16
US11535870B2 (en) 2022-12-27
IL253103A0 (en) 2017-08-31
JP6824169B2 (ja) 2021-02-03
US12440580B2 (en) 2025-10-14
JP2018501791A (ja) 2018-01-25
ES2824829T3 (es) 2021-05-13
EP3236984B1 (en) 2020-07-22
CN107636153A (zh) 2018-01-26
CA2972038A1 (en) 2016-06-30

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