Pre-clinical dose-escalation studies establish a therapeutic range for U7snRNA-mediated DMD exon 2 skipping

por Simmons, Tabatha R., Vetter, Tatyana A., Huang, Nianyuan, Vulin-Chaffiol, Adeline, Wein, Nicolas, Flanigan, Kevin M.
Publicado 2021

Lack of Toxicity in Nonhuman Primates Receiving Clinically Relevant Doses of an AAV9.U7snRNA Vector Designed to Induce DMD Exon 2 Skipping

por Gushchina, Liubov V., Frair, Emma C., Rohan, Natalie, Bradley, Adrienne J., Simmons, Tabatha R., Chavan, Hemantkumar D., Chou, Hsin-Jung, Eggers, Michelle, Waldrop, Megan A., Wein, Nicolas, Flanigan, Kevin M.
Publicado 2021

Systemic delivery of an AAV9 exon-skipping vector significantly improves or prevents features of Duchenne muscular dystrophy in the Dup2 mouse

por Wein, Nicolas, Vetter, Tatyana A., Vulin, Adeline, Simmons, Tabatha R., Frair, Emma C., Bradley, Adrienne J., Gushchina, Liubov V., Almeida, Camila F., Huang, Nianyuan, Lesman, Daniel, Rajakumar, Dhanarajan, Weiss, Robert B., Flanigan, Kevin M.
Publicado 2022

Systemic PPMO-mediated dystrophin expression in the Dup2 mouse model of Duchenne muscular dystrophy

por Gushchina, Liubov V., Vetter, Tatyana A., Frair, Emma C., Bradley, Adrienne J., Grounds, Kelly M., Lay, Jacob W., Huang, Nianyuan, Suhaiba, Aisha, Schnell, Frederick J., Hanson, Gunnar, Simmons, Tabatha R., Wein, Nicolas, Flanigan, Kevin M.
Publicado 2022

A first-in-human phase I/IIa gene transfer clinical trial for Duchenne muscular dystrophy using rAAVrh74.MCK.GALGT2

por Flanigan, Kevin M., Vetter, Tatyana A., Simmons, Tabatha R., Iammarino, Megan, Frair, Emma C., Rinaldi, Federica, Chicoine, Louis G., Harris, Johan, Cheatham, John P., Cheatham, Sharon L., Boe, Brian, Waldrop, Megan A., Zygmunt, Deborah A., Packer, Davin, Martin, Paul T.
Publicado 2022