JP2016533709A5 - - Google Patents

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JP2016533709A5
JP2016533709A5 JP2016517431A JP2016517431A JP2016533709A5 JP 2016533709 A5 JP2016533709 A5 JP 2016533709A5 JP 2016517431 A JP2016517431 A JP 2016517431A JP 2016517431 A JP2016517431 A JP 2016517431A JP 2016533709 A5 JP2016533709 A5 JP 2016533709A5
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amino acid
acid residue
seq
capsid protein
aav2 capsid
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JP2016517431A 2013-09-26 2014-09-26 標的遺伝子治療のための合成コンビナトリアルaavカプシドライブラリー Active JP6985795B2 (ja)

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US201361883063P 2013-09-26 2013-09-26
US61/883,063 2013-09-26
PCT/US2014/057842 WO2015048534A1 (en) 2013-09-26 2014-09-26 Synthetic combinatorial aav capsid library for targeted gene therapy

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JP2019137247A Division JP7007337B2 (ja) 2013-09-26 2019-07-25 標的遺伝子治療のための合成コンビナトリアルaavカプシドライブラリー

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JP2016533709A JP2016533709A (ja) 2016-11-04
JP2016533709A5 true JP2016533709A5 (enExample) 2017-09-21
JP6985795B2 JP6985795B2 (ja) 2021-12-22

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JP2016517431A Active JP6985795B2 (ja) 2013-09-26 2014-09-26 標的遺伝子治療のための合成コンビナトリアルaavカプシドライブラリー
JP2019137247A Active JP7007337B2 (ja) 2013-09-26 2019-07-25 標的遺伝子治療のための合成コンビナトリアルaavカプシドライブラリー

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US (2) US20160369298A1 (enExample)
EP (2) EP3633041A3 (enExample)
JP (2) JP6985795B2 (enExample)
AU (1) AU2014324717B2 (enExample)
WO (1) WO2015048534A1 (enExample)

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