Lipid nanoparticle delivery of antisense gapmers attenuates pathology in a mouse model of facioscapulohumeral muscular dystrophy

Saeed Anwar(University of Alberta), Toshifumi Yokota(University of Alberta), Yusuke Echigoya(Nihon University), Darko Bosnakovski(University of Minnesota), Abdullah Zia(University of Alberta), Karen Ying Tung Chan(University of British Columbia), Pieter R. Cullis(University of British Columbia), Umme Sabrina Haque(University of Alberta), Dominik Witzigmann(University of British Columbia), Md Nur Ahad Shah(Shahjalal University of Science and Technology), Samuel L. Beck(University of Alberta), Michael Kyba(University of Minnesota), Kenji Rowel Q. Lim(Washington University in St. Louis), Rika Maruyama(University of Alberta), Hidenori Moriyama(Keio University)
Molecular Therapy
May 1, 2026
Cited by 0


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