JP7256739B2 - 肝臓遺伝子のモジュレーション - Google Patents

肝臓遺伝子のモジュレーション Download PDF

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JP7256739B2
JP7256739B2 JP2019512750A JP2019512750A JP7256739B2 JP 7256739 B2 JP7256739 B2 JP 7256739B2 JP 2019512750 A JP2019512750 A JP 2019512750A JP 2019512750 A JP2019512750 A JP 2019512750A JP 7256739 B2 JP7256739 B2 JP 7256739B2
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domain
sequence
target
expression
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JP2019531068A (ja
JP2019531068A5 (enExample
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アンソニー コンウェイ，
ゲイリー ケー． リー，
デイビッド パッション，
レイ ジャン，
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Sangamo Therapeutics Inc
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    • A61K48/00—Medicinal preparations containing genetic material which is inserted into cells of the living body to treat genetic diseases; Gene therapy
    • A61K48/005—Medicinal preparations containing genetic material which is inserted into cells of the living body to treat genetic diseases; Gene therapy characterised by an aspect of the 'active' part of the composition delivered, i.e. the nucleic acid delivered
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    • C12N9/50—Proteinases, e.g. Endopeptidases (3.4.21-3.4.25)
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    • C12N9/14—Hydrolases (3)
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    • C12N9/50—Proteinases, e.g. Endopeptidases (3.4.21-3.4.25)
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    • C12N9/6424—Serine endopeptidases (3.4.21)
    • C12N9/6445—Kallikreins (3.4.21.34; 3.4.21.35)
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JP2019512750A 2016-09-07 2017-09-07 肝臓遺伝子のモジュレーション Active JP7256739B2 (ja)

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US201662384428P 2016-09-07 2016-09-07
US62/384,428 2016-09-07
PCT/US2017/050446 WO2018049009A2 (en) 2016-09-07 2017-09-07 Modulation of liver genes

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