A comparison of AAV strategies distinguishes overlapping vectors for efficient systemic delivery of the 6.2 kb Dysferlin coding sequence

by Pryadkina, Marina, Lostal, William, Bourg, Nathalie, Charton, Karine, Roudaut, Carinne, Hirsch, Matthew L, Richard, Isabelle
Published 2015

Lack of Correlation between Outcomes of Membrane Repair Assay and Correction of Dystrophic Changes in Experimental Therapeutic Strategy in Dysferlinopathy

by Lostal, William, Bartoli, Marc, Roudaut, Carinne, Bourg, Nathalie, Krahn, Martin, Pryadkina, Marina, Borel, Perrine, Suel, Laurence, Roche, Joseph A., Stockholm, Daniel, Bloch, Robert J., Levy, Nicolas, Bashir, Rumaisa, Richard, Isabelle
Published 2012