CN112469421A - 减少剪接病和治疗rna显性疾病的组合物和方法 - Google Patents

减少剪接病和治疗rna显性疾病的组合物和方法 Download PDF

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CN112469421A
CN112469421A CN201980047374.XA CN201980047374A CN112469421A CN 112469421 A CN112469421 A CN 112469421A CN 201980047374 A CN201980047374 A CN 201980047374A CN 112469421 A CN112469421 A CN 112469421A
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nucleic acid
rna
acid sequence
interfering rna
viral vector
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J·张伯伦
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WASHINGTON, University of
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CN201980047374.XA 2018-05-15 2019-05-15 减少剪接病和治疗rna显性疾病的组合物和方法 Pending CN112469421A (zh)

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US201862671769P 2018-05-15 2018-05-15
US62/671,769 2018-05-15
PCT/US2019/032423 WO2019222354A1 (en) 2018-05-15 2019-05-15 Compositions and methods for reducing spliceopathy and treating rna dominance disorders

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US (1) US20210269825A1 (enExample)
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CN113166208B (zh) 2018-10-02 2024-11-22 沃雅戈治疗公司 重新定向aav衣壳的嗜性
US12630842B2 (en) 2020-05-13 2026-05-19 Voyager Therapeutics, Inc. Redirection of tropism of AAV capsids
US11833221B2 (en) 2021-09-01 2023-12-05 Ionis Pharmaceuticals, Inc. Oligomeric compounds for reducing DMPK expression
EP4426716A1 (en) 2021-11-02 2024-09-11 Voyager Therapeutics, Inc. Aav capsid variants and uses thereof
KR20240169111A (ko) * 2022-04-06 2024-12-02 젠자임 코포레이션 Dm-1 근긴장성 이영양증에 대한 표적화된 유전자 요법
US20250352672A1 (en) * 2022-05-13 2025-11-20 University Of Washington Method for treatment of myotonic dystrophy combining protein expression and rna interference vector delivery with tissue detargeting
TW202409289A (zh) * 2022-06-27 2024-03-01 美商安斯泰來基因治療股份有限公司 用於治療肌強直性營養不良之組合物及方法
WO2024211480A2 (en) * 2023-04-05 2024-10-10 Genzyme Corporation Targeted gene therapy for dm-1 myotonic dystrophy
WO2024229164A2 (en) * 2023-05-03 2024-11-07 Voyager Therapeutics, Inc. Compositions and methods for the treatment of disorders related to dystrophia myotonica protein kinase
WO2025038795A1 (en) * 2023-08-16 2025-02-20 Voyager Therapeutics, Inc. Compositions and methods for the treatment of disorders related to dystrophia myotonica protein kinase
WO2025122536A1 (en) * 2023-12-05 2025-06-12 Voyager Therapeutics, Inc. Compositions and methods for the treatment of disorders related to dystrophia myotonica protein kinase
TW202615058A (zh) * 2024-06-12 2026-04-16 美商艾拉倫製藥股份有限公司 失養強直性肌肉蛋白質激酶(dmpk)irna組成物及其使用方法

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MA51938A1 (fr) 2021-11-30
SG11202011151VA (en) 2020-12-30
PH12020551913A1 (en) 2021-06-14
JP2021522836A (ja) 2021-09-02
MA51938B1 (fr) 2022-10-31
AU2019268346A1 (en) 2020-12-24
KR20210010549A (ko) 2021-01-27
MX2020012269A (es) 2021-04-28
EP3793566A4 (en) 2022-03-16
CL2020002955A1 (es) 2021-04-23
WO2019222354A1 (en) 2019-11-21
BR112020023298A2 (pt) 2021-03-09
US20210269825A1 (en) 2021-09-02
EP3793566A1 (en) 2021-03-24
CO2020015239A2 (es) 2021-03-08
CA3098249A1 (en) 2019-11-21

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