CN109121395B - 腺相关病毒载体递送β-肌聚糖和微RNA-29以及肌营养不良症的治疗 - Google Patents

腺相关病毒载体递送β-肌聚糖和微RNA-29以及肌营养不良症的治疗 Download PDF

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CN109121395B
CN109121395B CN201780023705.7A CN201780023705A CN109121395B CN 109121395 B CN109121395 B CN 109121395B CN 201780023705 A CN201780023705 A CN 201780023705A CN 109121395 B CN109121395 B CN 109121395B
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muscle
aav vector
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CN109121395A (zh
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L.罗迪诺-克拉帕
J.R.门德尔
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Nationwide Childrens Hospital Inc
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CN201780023705.7A 2016-04-15 2017-04-14 腺相关病毒载体递送β-肌聚糖和微RNA-29以及肌营养不良症的治疗 Active CN109121395B (zh)

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CN202210977746.2A CN117384964A (zh) 2016-04-15 2017-04-14 腺相关病毒载体递送β-肌聚糖和微RNA-29以及肌营养不良症的治疗

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US201662323333P 2016-04-15 2016-04-15
US62/323333 2016-04-15
US201662433548P 2016-12-13 2016-12-13
US62/433548 2016-12-13
PCT/US2017/027583 WO2017180976A1 (en) 2016-04-15 2017-04-14 Adeno-associated virus vector delivery of b-sarcoglycan and microrna-29 and the treatment of muscular dystrophy

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CN202210977746.2A Pending CN117384964A (zh) 2016-04-15 2017-04-14 腺相关病毒载体递送β-肌聚糖和微RNA-29以及肌营养不良症的治疗

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EP3020403A1 (en) 2014-11-14 2016-05-18 Universitat de Valéncia Compounds for the treatment of myotonic dystrophy
WO2016130600A2 (en) 2015-02-09 2016-08-18 Duke University Compositions and methods for epigenome editing
WO2017066497A2 (en) 2015-10-13 2017-04-20 Duke University Genome engineering with type i crispr systems in eukaryotic cells
KR102877920B1 (ko) 2015-11-16 2025-10-30 더 리서치 인스티튜트 앳 네이션와이드 칠드런스 하스피탈 티틴-기반 근증 및 다른 티틴성병증의 치료를 위한 물질 및 방법
EP3384055B1 (en) 2015-11-30 2025-07-16 Duke University Therapeutic targets for the correction of the human dystrophin gene by gene editing and methods of use
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FI3442600T3 (fi) * 2016-04-15 2024-05-14 Res Inst Nationwide Childrens Hospital Adeno-assosioidulla virusvektorilla b-sarkoglykaanin ja mikro-rna-29:n toimittaminen ja lihasdystrofian hoito
MA45477A (fr) * 2016-04-15 2019-02-20 Res Inst Nationwide Childrens Hospital Administration à vecteurs de virus adéno-associé de microarn-29 et micro-dystrophine pour traiter la dystrophie musculaire
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US11338045B2 (en) 2017-03-17 2022-05-24 Newcastle University Adeno-associated virus vector delivery of a fragment of micro-dystrophin to treat muscular dystrophy
KR20240164969A (ko) * 2017-10-20 2024-11-21 더 리서치 인스티튜트 앳 네이션와이드 칠드런스 하스피탈 Nt-3 유전자 치료를 위한 방법 및 물질
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US12377170B2 (en) * 2019-02-26 2025-08-05 Research Institute At Nationwide Children's Hospital Adeno-associated virus vector delivery of β-sarcoglycan and the treatment of muscular dystrophy
EP4017871B1 (en) * 2019-08-21 2024-01-17 Research Institute at Nationwide Children's Hospital Adeno-associated virus vector delivery of alpha-sarcoglycan and the treatment of muscular dystrophy
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