JP6985795B2 - 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 - Google Patents

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 Download PDF

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JP6985795B2
JP6985795B2 JP2016517431A JP2016517431A JP6985795B2 JP 6985795 B2 JP6985795 B2 JP 6985795B2 JP 2016517431 A JP2016517431 A JP 2016517431A JP 2016517431 A JP2016517431 A JP 2016517431A JP 6985795 B2 JP6985795 B2 JP 6985795B2
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マルシク,ダミアン
ゾロツキン,セルゲイ
アグバンジェ−マッケナ,メイビス
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ユニバーシティ オブ フロリダ リサーチ ファンデーション インコーポレーティッド
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    • C12N2750/14111Dependovirus, e.g. adenoassociated viruses
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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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PCT/US2014/057842 WO2015048534A1 (en) 2013-09-26 2014-09-26 Synthetic combinatorial aav capsid library for targeted gene therapy

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