Systemic AAVrh10 provides higher transgene expression than AAV9 in the brain and the spinal cord of neonatal mice

Por Tanguy, Yannick, Biferi, Maria G., Besse, Aurore, Astord, Stephanie, Cohen-Tannoudji, Mathilde, Marais, Thibaut, Barkats, Martine
Publicado no Front Mol Neurosci (2015)

Intramuscular scAAV9-SMN Injection Mediates Widespread Gene Delivery to the Spinal Cord and Decreases Disease Severity in SMA Mice

Por Benkhelifa-Ziyyat, Sofia, Besse, Aurore, Roda, Marianne, Duque, Sandra, Astord, Stéphanie, Carcenac, Romain, Marais, Thibaut, Barkats, Martine
Publicado em 2013

Intravenous Administration of Self-complementary AAV9 Enables Transgene Delivery to Adult Motor Neurons

Por Duque, Sandra, Joussemet, Béatrice, Riviere, Christel, Marais, Thibaut, Dubreil, Laurence, Douar, Anne-Marie, Fyfe, John, Moullier, Philippe, Colle, Marie-Anne, Barkats, Martine
Publicado em 2009

Lentiviral vector-mediated overexpression of mutant ataxin-7 recapitulates SCA7 pathology and promotes accumulation of the FUS/TLS and MBNL1 RNA-binding proteins

Por Alves, Sandro, Marais, Thibaut, Biferi, Maria-Grazia, Furling, Denis, Marinello, Martina, El Hachimi, Khalid, Cartier, Nathalie, Ruberg, Merle, Stevanin, Giovanni, Brice, Alexis, Barkats, Martine, Sittler, Annie
Publicado no Mol Neurodegener (2016)

Efficacy and biodistribution analysis of intracerebroventricular administration of an optimized scAAV9-SMN1 vector in a mouse model of spinal muscular atrophy

Por Armbruster, Nicole, Lattanzi, Annalisa, Jeavons, Matthieu, Van Wittenberghe, Laetitia, Gjata, Bernard, Marais, Thibaut, Martin, Samia, Vignaud, Alban, Voit, Thomas, Mavilio, Fulvio, Barkats, Martine, Buj-Bello, Ana
Publicado no Mol Ther Methods Clin Dev (2016)