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Journal of Cell Science|August 17, 2021
Titin M-line insertion sequence 7 is required for proper cardiac function in miceAriane Biquand, Simone Spinozzi, Paola Tonino, et al.Proceedings of the National Academy of Sciences of the United States of America|June 16, 2026
Restoring the interplay between the endoplasmic reticulum and mitochondria by gene therapy improves Charcot-Marie-Tooth type 2A diseaseMarine Tessier, Zeinab Hamze, Nathalie Bonello-Palot, et al.Annals of Clinical and Translational Neurology|April 26, 2019
Correction of pseudoexon splicing caused by a novel intronic dysferlin mutationJanice A Dominov, Özgün Uyan, Diane McKenna-Yasek, et al.Journal of Cachexia, Sarcopenia and Muscle|May 26, 2021
Cholesterol metabolism is a potential therapeutic target in Duchenne muscular dystrophyFatima Amor, Ai Vu Hong, Guillaume Corre, et al.Life Science Alliance|October 20, 2022
Dlk1-Dio3 cluster miRNAs regulate mitochondrial functions in the dystrophic muscle in Duchenne muscular dystrophyAi Vu Hong, Nathalie Bourg, Peggy Sanatine, et al.Molecular Therapy : the Journal of the American Society of Gene Therapy|December 25, 2025
Transgene-Induced Cardiotoxicity In High-Dose AAV Gene TransferAriane Biquand, Evelyne Gicquel, Jerome Poupiot, et al.Human Molecular Genetics|April 17, 2015
CAPN3-mediated processing of C-terminal titin replaced by pathological cleavage in titinopathyKarine Charton, Jaakko Sarparanta, Anna Vihola, et al.Human Molecular Genetics|March 24, 2017
AAV-mediated transfer of FKRP shows therapeutic efficacy in a murine model but requires control of gene expressionEvelyne Gicquel, Natacha Maizonnier, Steven J Foltz, et al.Nature Communications|September 11, 2024
An engineered AAV targeting integrin alpha V beta 6 presents improved myotropism across speciesAi Vu Hong, Laurence Suel, Eva Petat, et al.Investigative Ophthalmology & Visual Science|May 26, 2007
Age-related cataracts in alpha3Cx46-knockout mice are dependent on a calpain 3 isoformYajun Tang, Xiangyang Liu, Rebecca K Zoltoski, et al.Pageof 23