M. Allais

TRPC3, but not TRPC1, as a good therapeutic target for standalone or complementary treatment of DMD

T. Larcher, I. Anegon, B. Fraysse, 2021, Journal of translational medicine.

Evaluation of the dystrophin carboxy-terminal domain for micro-dystrophin gene therapy in cardiac and skeletal muscles in the DMDmdx rat model

L. Popplewell, G. Dickson, T. Larcher, 2022, Gene Therapy.

Forelimb treatment in a large cohort of dystrophic dogs supports delivery of a recombinant AAV for exon skipping in Duchenne patients.

R. Kotin, L. Servais, J. Hogrel, 2014, Molecular therapy : the journal of the American Society of Gene Therapy.

RNA-Seq Analysis of an Antisense Sequence Optimized for Exon Skipping in Duchenne Patients Reveals No Off-Target Effect

L. Servais, T. Voit, Y. Audic, 2017, Molecular therapy. Nucleic acids.

Establishment of two quantitative nested qPCR assays targeting the human EPO transgene

Dirk Moser, P. Moullier, R. Snyder, 2016, Gene Therapy.

Long-term microdystrophin gene therapy is effective in a canine model of Duchenne muscular dystrophy

Thomas Voit, Jean-Yves Hogrel, Laurent Servais, 2017, Nature Communications.

Mannose-coupled AAV2: A second-generation AAV vector for increased retinal gene therapy efficiency

E. Ayuso, A. Galy, M. Mével, 2022, bioRxiv.

Evaluation of the dystrophin carboxy-terminal domain for micro-dystrophin gene therapy in cardiac and 1 skeletal muscles in the DMD mdx rat model 2 3

L. Popplewell, T. Larcher, S. Remy, 2021 .