Partial Restoration of Brain Dystrophin and Behavioral Deficits by Exon Skipping in the Muscular Dystrophy X-Linked (mdx) Mouse.

"Potential Conflicts of Interest TT is an employee of SQY Therapeutics which produces tricyclo‐DNA oligomers. LG is co‐founder of SQY Therapeutics."

"This work was supported by Agence Nationale de la Recherche (ANR, France; initiated and coordinated by CV; grant number ANR‐14‐CE13‐0037‐01 to CV and AG), Centre National de la Recherche Scientifique (CNRS, France), the Institut National de la santé et la recherche médicale (INSERM), Université Paris‐Saclay (France), Paris Ile‐de‐France Region, grants to F.Z. and C.V from Association Monégasque contre les Myopathies (AMM, Monaco), and PhD fellowships from Ministère de l'Enseignement Supérieur et de la Recherche (France) to F.Z. and F.B. We also gratefully acknowledge support to C.V. and A.G. from the European Union's Horizon 2020 research and innovation program “Brain Involvement iN Dystrophinopathies” under grant agreement No 847826. The authors are grateful to the Zootechnic platform of our institutes for mouse breeding, care, and genotyping."