CN110997923B - 腺相关病毒载体递送肌肉特异性微肌营养不良蛋白以治疗肌营养不良症 - Google Patents

腺相关病毒载体递送肌肉特异性微肌营养不良蛋白以治疗肌营养不良症 Download PDF

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CN110997923B
CN110997923B CN201880018893.9A CN201880018893A CN110997923B CN 110997923 B CN110997923 B CN 110997923B CN 201880018893 A CN201880018893 A CN 201880018893A CN 110997923 B CN110997923 B CN 110997923B
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muscle
dystrophin
micro
aav
raav
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CN110997923A (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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CN201880018893.9A 2017-03-17 2018-03-16 腺相关病毒载体递送肌肉特异性微肌营养不良蛋白以治疗肌营养不良症 Active CN110997923B (zh)

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US201762473148P 2017-03-17 2017-03-17
US62/473148 2017-03-17
PCT/US2018/022881 WO2018170408A1 (en) 2017-03-17 2018-03-16 Adeno-associated virus vector delivery of muscle specific micro-dystrophin to treat muscular dystrophy

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CN202311798281.5A Pending CN118497275A (zh) 2017-03-17 2018-03-16 腺相关病毒载体递送肌肉特异性微肌营养不良蛋白以治疗肌营养不良症

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JP7642809B2 (ja) * 2020-11-12 2025-03-10 プレシジョン バイオサイエンシズ，インク． ジストロフィン遺伝子内の認識配列に特異性を有する遺伝子操作されたメガヌクレアーゼ
EP4086276A1 (en) 2021-05-03 2022-11-09 Université d'Aix-Marseille Composition for treating dysferlinopathy
EP4108263A3 (en) * 2021-06-02 2023-03-22 Research Institute at Nationwide Children's Hospital Recombinant adeno-associated virus products and methods for treating limb girdle muscular dystrophy 2a
US20250041450A1 (en) * 2021-08-05 2025-02-06 Insmed Incorporated Adeno-associated virus particles and methods of use thereof
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CN114316070B (zh) * 2021-12-29 2022-11-15 上海勉亦生物科技有限公司 用于治疗肌营养不良症的转基因表达盒
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CN118460618A (zh) * 2022-04-19 2024-08-09 康霖生物科技（杭州）有限公司 一种用于遗传性凝血因子缺乏病治疗的核酸构建体
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