Sylvie Boutin

TL;DR: Characterization of the preexisting humoral responses to the AAV capsid and cross-reactivity will allow development of new strategies to circumvent AAV acquired immune responses, and vectors based on AAV5, AAV8, and AAV9 may have an advantage for gene therapy in humans.

TL;DR: CAV vectors were replication defective in all cell lines tested, transduced human-derived cells at an efficiency similar to that of a comparable human adenovirus type 5 vector, and are amenable to in vivo use.

TL;DR: It is shown that frequent sessions of PP result in drastic reduction of NAF specific for AAV1, 2, 6, and 8 to undetectable levels or titers <1:5, mainly when initial titers were ≤1:20.

TL;DR: It is demonstrated that CAV-2 transduction is augmented by CAR and possibly by αvβ5, though transduction can be CAR and αv β3/5independent but is αMβ2, MHC-I, and RGD independent, demonstrating a transduction mechanism which is distinct from that of Ad2/5.

TL;DR: The concept of a phase 1/2 trial of locoregional delivery into upper limbs of nonambulatory DMD patients is supported, as there was a dose relationship for the amount of exon skipping and histological, nuclear magnetic resonance pathological indices and strength improvement responded in a dose-dependent manner.