Article
Suppression of heterotopic ossification in fibrodysplasia ossificans progressiva using AAV gene delivery.
Nature communications - 19 Oct 2022
Yang Yeon-Suk, Kim Jung-Min, Xie Jun, Chaugule Sachin, Lin Chujiao, Ma Hong, Hsiao Edward, Hong Jaehyoung, Chun Hyonho, Shore Eileen M, Kaplan Frederick S, Gao Guangping, Shim Jae-Hyuck
Abstract excerpt
Heterotopic ossification is the most disabling feature of fibrodysplasia ossificans progressiva, an ultra-rare genetic disorder for which there is currently no prevention or treatment. Most patients with this disease harbor a heterozygous activating mutation (c.617 G > A;p.R206H) in ACVR1. Here, we identify recombinant AAV9 as the most effective serotype for transduction of the major cells-of-origin of...
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