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体内靶向造血干细胞(HSCs)的基因治疗研究进展
在众多体内基因治疗载体中,腺相关病毒(Adeno - associated virus,AAV)脱颖而出 ... 这可能是因为 AAV6 载体转导 HSCs 会导致细胞分化和植入能力降低,高表达的 Cre 会对细胞造成 DNA 损伤,从而影响转导 HSCs 的适应性。尽管如此,研究数据表明长期功能性 HSCs 可 ...
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