Allele-specific RNAi therapy corrects an extracellular matrix defect in Schuurs-Hoeijmakers syndrome

Journal:: American Journal of Human Genetics
Published:: 2025-09-18
DOI:: 10.1016/j.ajhg.2025.07.010
Affiliations:: 2
Authors:: 7

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Institutions	Authors	Share
Center of Research in Myology, France	5.000000	0.71
Institute of Myology, France	2.000000	0.29

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