Article

Suppression of Heterotopic Ossification in Fibrodysplasia Ossificans Progressiva Using AAV Gene Delivery

2022-06-10

Abstract excerpt

<title>Abstract</title> <p>Progressive heterotopic ossification (HO) is the most disabling and defining feature of fibrodysplasia ossificans progressiva (FOP), an ultra-rare genetic disorder. More than 95% of FOP patients harbor a heterozygous activating mutation (c.617G>A;p.R206H) in the Activin A Receptor Type 1 (ACVR1). Currently, there are no definitive preventions or treatments. Gene therapy is thus an attra...

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Literature Corpus work: 26d921cf-7788-5ca7-92b7-7c05273a6b6e
DOI: 10.21203/rs.3.rs-1646941/v1

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