JP6867945B2 - 核内遺伝子出力の標的とされた増強 - Google Patents

核内遺伝子出力の標的とされた増強 Download PDF

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JP6867945B2
JP6867945B2 JP2017518228A JP2017518228A JP6867945B2 JP 6867945 B2 JP6867945 B2 JP 6867945B2 JP 2017518228 A JP2017518228 A JP 2017518228A JP 2017518228 A JP2017518228 A JP 2017518228A JP 6867945 B2 JP6867945 B2 JP 6867945B2
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intron
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mrna
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JP2017536338A5 (enExample
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クライナー，エイドリアン
アズナレズ，イザベル
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コールド スプリング ハーバー ラボラトリー
コールド スプリング ハーバー ラボラトリー
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JP2017518228A 2014-10-03 2015-10-03 核内遺伝子出力の標的とされた増強 Active JP6867945B2 (ja)

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US201462059847P 2014-10-03 2014-10-03
US62/059,847 2014-10-03
PCT/US2015/053896 WO2016054615A2 (en) 2014-10-03 2015-10-03 Targeted augmentation of nuclear gene output

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WO2015187825A2 (en) 2014-06-03 2015-12-10 University Of Massachusetts Compositions and methods for modulating dysferlin expression
GB201410693D0 (en) 2014-06-16 2014-07-30 Univ Southampton Splicing modulation
EP3201339A4 (en) * 2014-10-03 2018-09-19 Cold Spring Harbor Laboratory Targeted augmentation of nuclear gene output
EP3259356B1 (en) 2015-02-20 2021-12-01 Rosalind Franklin University of Medicine and Science Antisense compounds targeting genes associated with cystic fibrosis
US10525076B2 (en) 2015-02-20 2020-01-07 Rosalind Franklin University Of Medicine And Science Antisense compounds targeting genes associated with cystic fibrosis
KR20220105174A (ko) 2015-10-09 2022-07-26 유니버시티 오브 사우스앰톤 유전자 발현의 조절 및 탈조절된 단백질 발현의 스크리닝
US11096956B2 (en) 2015-12-14 2021-08-24 Stoke Therapeutics, Inc. Antisense oligomers and uses thereof
CA3005249A1 (en) * 2015-12-14 2017-06-22 Cold Spring Harbor Laboratory Compositions and methods for treatment of kidney diseases
EP3389782A4 (en) * 2015-12-14 2019-07-31 Cold Spring Harbor Laboratory ANTISENSE OLIGOMERS FOR THE TREATMENT OF A POLYCYSTIC KIDNEY DISEASE
WO2017106377A1 (en) 2015-12-14 2017-06-22 Cold Spring Harbor Laboratory Antisense oligomers for treatment of autosomal dominant mental retardation-5 and dravet syndrome
EP3390634A4 (en) * 2015-12-14 2019-08-14 Cold Spring Harbor Laboratory COMPOSITIONS AND METHODS FOR THE TREATMENT OF EYE DISEASES
EP3390635A4 (en) * 2015-12-14 2019-05-01 Cold Spring Harbor Laboratory Antisense oligomers for the treatment of a tuberous sclerosis complex
US11730828B2 (en) 2017-02-07 2023-08-22 The Regents Of The University Of California Gene therapy for haploinsufficiency
KR20240035631A (ko) 2017-08-25 2024-03-15 스톡 테라퓨틱스, 인크. 병태 및 질환 치료용 안티센스 올리고머
WO2019213525A1 (en) 2018-05-04 2019-11-07 Stoke Therapeutics, Inc. Methods and compositions for treatment of cholesteryl ester storage disease
US12163129B2 (en) 2018-06-08 2024-12-10 University Of Massachusetts Antisense oligonucleotides to restore dysferlin protein expression in dysferlinopathy patient cells
CN113329739B (zh) 2018-08-20 2024-06-25 罗贡股份有限公司 用于治疗scn1a脑病的靶向scn2a的反义寡核苷酸
AU2020210924A1 (en) * 2019-01-23 2021-09-16 The Florey Institute Of Neuroscience And Mental Health Antisense oligonucleotides targeting SCN2A retained introns
CA3162618A1 (en) 2019-03-20 2020-09-24 Peter Jungsoo PARK Antisense oligonucleotide-based progranulin augmentation therapy in neurodegenerative diseases
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