Designer AAV muscle up.

Bönnemann, Carsten G · Cell · 2021

basic_science · Level V

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Abstract

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.

Medical subject headings