2023
Albini S et Al. « Assessment of Therapeutic Potential of a Dual AAV Approach for Duchenne Muscular Dystrophy » International Journal of Molecular Sciences, 24,11421
Berling E et Al.
« Gene therapy review: Duchenne muscular dystrophy case study »
Revue neurologique 179, 90-105
Morin A et Al.
« Dystrophin myonuclear domain restoration governs treatment efficacy in dystrophic muscle »
Proceedings of the National Academy of Sciences of the United States of America 120, e2206324120
Prakasam R et Al.
« LSD1/PRMT6-targeting gene therapy to attenuate androgen receptor toxic gain-of-function ameliorates spinobulbar muscular atrophy phenotypes in flies and mice »
Nature communications 14, 603
Steicy Sobrino et Al
« Severe hematopoeitic stem cell inflammation compromises chronic granulomatous disease gene therapy »
Cell Reports Medicine
