JP7075597B2 - デュシェンヌ型筋ジストロフィーを治療するためのｃｒｉｓｐｒ／ｃａｓ関連の方法および組成物 - Google Patents

デュシェンヌ型筋ジストロフィーを治療するためのｃｒｉｓｐｒ／ｃａｓ関連の方法および組成物 Download PDF

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JP7075597B2
JP7075597B2 JP2018558287A JP2018558287A JP7075597B2 JP 7075597 B2 JP7075597 B2 JP 7075597B2 JP 2018558287 A JP2018558287 A JP 2018558287A JP 2018558287 A JP2018558287 A JP 2018558287A JP 7075597 B2 JP7075597 B2 JP 7075597B2
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grna molecule
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デイヴィッド エイ バムクロット
ニコラス シー ハストン
ジョシュア シー タイコー
ジャクリーン ロビンソン－ハム
チャールズ エイ ガーズバック
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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
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    • C12N15/09—Recombinant DNA-technology
    • C12N15/11—DNA or RNA fragments; Modified forms thereof; Non-coding nucleic acids having a biological activity
    • C12N15/113—Non-coding nucleic acids modulating the expression of genes, e.g. antisense oligonucleotides; Antisense DNA or RNA; Triplex- forming oligonucleotides; Catalytic nucleic acids, e.g. ribozymes; Nucleic acids used in co-suppression or gene silencing
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    • C12N2750/14111—Dependovirus, e.g. adenoassociated viruses
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JP2018558287A 2016-05-05 2017-05-05 デュシェンヌ型筋ジストロフィーを治療するためのｃｒｉｓｐｒ／ｃａｓ関連の方法および組成物 Active JP7075597B2 (ja)

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US201662332297P 2016-05-05 2016-05-05
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PCT/US2017/031351 WO2017193029A2 (en) 2016-05-05 2017-05-05 Crispr/cas-related methods and compositions for treating duchenne muscular dystrophy

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WO2017193029A2 (en) 2017-11-09
ES2957660T3 (es) 2024-01-23
EP3452498B1 (en) 2023-07-05
WO2017193029A3 (en) 2018-02-08
JP2019522461A (ja) 2019-08-15
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