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该研究提出了一种能够利用AAV递送较大基因载荷的策略,从而为之前认为无法进行AAV基因治疗的疾病提供了治疗可能性。 治疗性基因的递送对于基因治疗至关重要。腺相关病毒(AAV)因其优越的基因分割灵活性和强大的基因重建效率,成为携带基因载荷的主要载体 ...
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