Gene Therapy with covalently-closed-end AAV vector for Spinal Muscular Atrophy.

已完结 20 由 ? 发布于 2025/7/25 9:17:54

DOI:10.1016/j.ymthe.2025.06.028
作者：Haolin Duan,Ciliu Zhang,Zhongliang Zhang,Xiaole Wang,Junping Zhang,Lifen Yang,Fang He,Leilei Mao,Li Yang,Zou Pan,Renzhi Han,Weiming Wang,Dao Pan,Fei Yin,Weidong Xiao,Jing Peng
文献类型：期刊论文
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Elsevier

Abstract：Covalently closed-end adeno-associated virus vector (cceAAV) is a new generation of self-complementary vector (scAAV) which does not utilize a mutant ITR for vector production. Importantly,

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