JP2014511694A - 修飾型rna(mod−rna)を使用する効率的なインビボタンパク質発現 - Google Patents

修飾型rna(mod−rna)を使用する効率的なインビボタンパク質発現 Download PDF

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JP2014511694A
JP2014511694A JP2014503668A JP2014503668A JP2014511694A JP 2014511694 A JP2014511694 A JP 2014511694A JP 2014503668 A JP2014503668 A JP 2014503668A JP 2014503668 A JP2014503668 A JP 2014503668A JP 2014511694 A JP2014511694 A JP 2014511694A
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modified rna
rna molecule
synthetic modified
rna
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JP2014511694A5 (OSRAM
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ケネス アール. シーン
レオン エム. プタチェク
キャシー オワ−ラン リュイ
リオール ザンギー
渉 蝦名
デリック ジェイ. ロッシ
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ザ ジェネラル ホスピタル コーポレーション ドゥーイング ビジネス アズ マサチューセッツ ジェネラル ホスピタル
イミューン ディズィーズ インスティテュート インコーポレイテッド
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JP2014503668A 2011-04-03 2012-03-12 修飾型rna(mod−rna)を使用する効率的なインビボタンパク質発現 Withdrawn JP2014511694A (ja)

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Application Number Priority Date Filing Date Title
US201161471166P 2011-04-03 2011-04-03
US61/471,166 2011-04-03
US201161471584P 2011-04-04 2011-04-04
US61/471,584 2011-04-04
PCT/US2012/028802 WO2012138453A1 (en) 2011-04-03 2012-03-12 Efficient protein expression in vivo using modified rna (mod-rna)

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