JP6649265B2 - 糖原病の処置のためのアデノ随伴ウイルスベクター - Google Patents

糖原病の処置のためのアデノ随伴ウイルスベクター Download PDF

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JP6649265B2
JP6649265B2 JP2016554828A JP2016554828A JP6649265B2 JP 6649265 B2 JP6649265 B2 JP 6649265B2 JP 2016554828 A JP2016554828 A JP 2016554828A JP 2016554828 A JP2016554828 A JP 2016554828A JP 6649265 B2 JP6649265 B2 JP 6649265B2
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ジャニス ジェイ． チョウ，
ジャニス ジェイ． チョウ，
バリー ジェイ． バーン，
バリー ジェイ． バーン，
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    • 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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JP2016554828A 2013-11-26 2014-11-25 糖原病の処置のためのアデノ随伴ウイルスベクター Active JP6649265B2 (ja)

Applications Claiming Priority (3)

Application Number Priority Date Filing Date Title
US201361908861P 2013-11-26 2013-11-26
US61/908,861 2013-11-26
PCT/US2014/067415 WO2015081101A1 (en) 2013-11-26 2014-11-25 Adeno-associated virus vectors for treatment of glycogen storage disease

Publications (3)

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JP2016538885A JP2016538885A (ja) 2016-12-15
JP2016538885A5 JP2016538885A5 (https=) 2017-11-24
JP6649265B2 true JP6649265B2 (ja) 2020-02-19

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US (3) US9644216B2 (https=)
EP (2) EP3074510B1 (https=)
JP (1) JP6649265B2 (https=)
CN (1) CN105934515B (https=)
AU (1) AU2014354839B2 (https=)
CA (1) CA2930872C (https=)
ES (1) ES2690643T3 (https=)
IL (1) IL245659B (https=)
MX (1) MX375360B (https=)
WO (1) WO2015081101A1 (https=)

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KR20230145206A (ko) 2014-11-14 2023-10-17 보이저 테라퓨틱스, 인크. 조절성 폴리뉴클레오티드
WO2016077687A1 (en) 2014-11-14 2016-05-19 Voyager Therapeutics, Inc. Compositions and methods of treating amyotrophic lateral sclerosis (als)
US11697825B2 (en) 2014-12-12 2023-07-11 Voyager Therapeutics, Inc. Compositions and methods for the production of scAAV
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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EP3448874A4 (en) 2016-04-29 2020-04-22 Voyager Therapeutics, Inc. COMPOSITIONS FOR TREATING A DISEASE
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KR102427379B1 (ko) 2016-05-18 2022-08-02 보이저 테라퓨틱스, 인크. 헌팅톤 질환을 치료하기 위한 조성물 및 방법
WO2018044933A1 (en) 2016-08-30 2018-03-08 The Regents Of The University Of California Methods for biomedical targeting and delivery and devices and systems for practicing the same
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
JP2020518258A (ja) 2017-05-05 2020-06-25 ボイジャー セラピューティクス インコーポレイテッドＶｏｙａｇｅｒ Ｔｈｅｒａｐｅｕｔｉｃｓ，Ｉｎｃ． 筋萎縮性側索硬化症（ａｌｓ）治療組成物および方法
JP2020518259A (ja) 2017-05-05 2020-06-25 ボイジャー セラピューティクス インコーポレイテッドＶｏｙａｇｅｒ Ｔｈｅｒａｐｅｕｔｉｃｓ，Ｉｎｃ． ハンチントン病治療組成物および方法
JOP20190269A1 (ar) 2017-06-15 2019-11-20 Voyager Therapeutics Inc بولي نوكليوتيدات aadc لعلاج مرض باركنسون
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TW202413649A (zh) 2017-10-16 2024-04-01 美商航海家醫療公司 肌萎縮性脊髓側索硬化症(als)之治療
EP3697908A1 (en) 2017-10-16 2020-08-26 Voyager Therapeutics, Inc. Treatment of amyotrophic lateral sclerosis (als)
KR102398295B1 (ko) 2018-03-09 2022-05-17 다이이찌 산쿄 가부시키가이샤 당원병 Ia형 치료약
KR20210019996A (ko) 2018-05-15 2021-02-23 보이저 테라퓨틱스, 인크. 파킨슨병의 치료를 위한 조성물 및 방법
MY207355A (en) * 2018-05-16 2025-02-21 Spark Therapeutics Inc Codon-optimized acid lpha-glucosidase expression cassettes and methods of using same
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CA2930872C (en) 2022-05-31
IL245659A0 (en) 2016-06-30
CN105934515A (zh) 2016-09-07
WO2015081101A1 (en) 2015-06-04
US20190017069A1 (en) 2019-01-17
MX375360B (es) 2025-03-06
US20170233763A1 (en) 2017-08-17
CN105934515B (zh) 2020-08-04
AU2014354839B2 (en) 2020-06-04
JP2016538885A (ja) 2016-12-15
EP3415620A1 (en) 2018-12-19
AU2014354839A1 (en) 2016-06-09
US9644216B2 (en) 2017-05-09
BR112016011997A2 (pt) 2017-09-26
EP3415620B1 (en) 2021-03-31
US10113183B2 (en) 2018-10-30
US11060110B2 (en) 2021-07-13
WO2015081101A8 (en) 2016-06-16
EP3074510B1 (en) 2018-08-01
EP3074510A1 (en) 2016-10-05
CA2930872A1 (en) 2015-06-04
ES2690643T3 (es) 2018-11-21
IL245659B (en) 2020-01-30
US20160376608A1 (en) 2016-12-29
MX2016006774A (es) 2016-10-13

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