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Designer AAV muscle up
Cell ( IF 64.5 ) Pub Date : 2021-09-16 , DOI: 10.1016/j.cell.2021.08.031 Carsten G Bönnemann 1
中文翻译:
设计师 AAV 增肌
更新日期:2021-09-17
Cell ( IF 64.5 ) Pub Date : 2021-09-16 , DOI: 10.1016/j.cell.2021.08.031 Carsten G Bönnemann 1
Affiliation
Directed evolution of AAV capsids has been a successful strategy for generating bespoke serotypes to target gene therapies more specifically to the intended tissue. This has now been achieved for the largest organ, skeletal muscle, by selecting for an RGD containing integrin binding heptamer in a hypervariable region of the capsid of AAV9.
中文翻译:
设计师 AAV 增肌
AAV 衣壳的定向进化已成为生成定制血清型以更具体地针对预期组织进行基因治疗的成功策略。现在,通过在 AAV9 衣壳的高变区中选择包含整合素结合七聚体的 RGD,现在已经为最大的器官骨骼肌实现了这一点。