Article

Precision RNAi for Fibrodysplasia Ossificans Progressiva: a combinatorial, unimolecular, allele selective approach

2026-06-15

Abstract excerpt

<title>Abstract</title> <p>Fibrodysplasia ossificans progressiva (FOP) is a rare genetic disorder caused by a dominant mutation in the ACVR1 gene (R206H, 97% of cases), leading to debilitating heterotopic ossification (HO) characterized by abnormal bone growth triggered by inflammatory flare-ups. Here, we report the development of disease-modifying, allele-selective small interfering RNA (siRNA) targeting ACVR1R2...

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Literature Corpus work: a0562099-e480-527c-b1c4-3cb0ed6f9fcd
DOI: 10.21203/rs.3.rs-9854026/v1

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