Single-base 2′OMe-modified LNA and MOE gapmers selectively silence ACVR1R206H in fibrodysplasia ossificans progressiva

Molecular Therapy — Nucleic Acids 2026 Open access

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Authors: Saeed Anwar, Sarah Hay, Hidenori Moriyama, Farin Mir, Rika Maruyama, Toshifumi Yokota

DOI: 10.1016/j.omtn.2026.102937

Cited by: 1

References: 74

Abstract

. Further studies in disease-relevant FOP models will be needed to establish therapeutic efficacy and long-term safety.

Orthopaedic implants and arthroplasty Polymer Surface Interaction Studies Osteoarthritis Treatment and Mechanisms

References 74