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Comment
. 2021 Sep 16;184(19):4845-4847.
doi: 10.1016/j.cell.2021.08.031.

Designer AAV muscle up

Carsten G Bönnemann  1
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Designer AAV muscle up

Carsten G Bönnemann. Cell. .
Free article

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.

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Conflict of interest statement

Declaration of interests In his position at the NIH, C.G.B. serves on several scientific advisory boards (MDUK Neuromuscular Center, NeuroMyoGene Lyon, Généthon, Sarepta Therapeutics, CureCMD, CMD-IR, RYR1 Foundation, MTM and CNM Registry) and receives no personal compensation for his contributions.

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