JP7347932B2 - ムコリピドーシスｉｉ型を治療するためのアデノ随伴ウイルスベクター - Google Patents

ムコリピドーシスｉｉ型を治療するためのアデノ随伴ウイルスベクター Download PDF

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JP7347932B2
JP7347932B2 JP2018531240A JP2018531240A JP7347932B2 JP 7347932 B2 JP7347932 B2 JP 7347932B2 JP 2018531240 A JP2018531240 A JP 2018531240A JP 2018531240 A JP2018531240 A JP 2018531240A JP 7347932 B2 JP7347932 B2 JP 7347932B2
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aav
raav
gnptab
nucleic acid
pharmaceutical composition
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JP2019500034A (ja
JP2019500034A5 (enExample
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ネルソン・イュー
チン・トン－キュ
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Genzyme Corp
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Genzyme Corp
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JP2018531240A 2015-12-15 2016-12-14 ムコリピドーシスｉｉ型を治療するためのアデノ随伴ウイルスベクター Active JP7347932B2 (ja)

Priority Applications (2)

Application Number Priority Date Filing Date Title
JP2021210071A JP7878799B2 (ja) 2015-12-15 2021-12-24 ムコリピドーシスｉｉ型を治療するためのアデノ随伴ウイルスベクター
JP2024019140A JP2024056832A (ja) 2015-12-15 2024-02-13 ムコリピドーシスｉｉ型を治療するためのアデノ随伴ウイルスベクター

Applications Claiming Priority (3)

Application Number Priority Date Filing Date Title
US201562267502P 2015-12-15 2015-12-15
US62/267,502 2015-12-15
PCT/US2016/066611 WO2017106313A1 (en) 2015-12-15 2016-12-14 Adeno-associated viral vectors for treating mucolipidosis type ii

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JP2021210071A Division JP7878799B2 (ja) 2015-12-15 2021-12-24 ムコリピドーシスｉｉ型を治療するためのアデノ随伴ウイルスベクター

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JP2019500034A JP2019500034A (ja) 2019-01-10
JP2019500034A5 JP2019500034A5 (enExample) 2020-01-30
JP7347932B2 true JP7347932B2 (ja) 2023-09-20

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JP2021210071A Active JP7878799B2 (ja) 2015-12-15 2021-12-24 ムコリピドーシスｉｉ型を治療するためのアデノ随伴ウイルスベクター
JP2024019140A Pending JP2024056832A (ja) 2015-12-15 2024-02-13 ムコリピドーシスｉｉ型を治療するためのアデノ随伴ウイルスベクター

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JP2024019140A Pending JP2024056832A (ja) 2015-12-15 2024-02-13 ムコリピドーシスｉｉ型を治療するためのアデノ随伴ウイルスベクター

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RU2020118342A (ru) 2017-11-07 2021-12-08 Дзе Юниверсити Оф Норт Каролина Эт Чепел Хилл Оптимизированные гены aga и экспрессионные кластеры и их применение
MX2021005997A (es) * 2018-11-21 2021-08-11 Vectores virales recombinantes y acidos nucleicos para producirlos.
JP7781057B2 (ja) * 2019-10-18 2025-12-05 リサーチ インスティチュート アット ネイションワイド チルドレンズ ホスピタル Ｉｒｆ２ｂｐｌ遺伝子の変異に関連する障害の治療のための材料および方法
FI4103214T3 (fi) * 2020-02-13 2026-07-03 Tenaya Therapeutics Inc Geeniterapian vektorit sydänsairauden hoitoon

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WO2015060722A1 (en) 2013-10-24 2015-04-30 Uniqure Ip B.V. Aav-5 pseudotyped vector for gene therapy for neurological diseases

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