Efficacy and Safety of a Krabbe Disease Gene Therapy

por Hordeaux, Juliette, Jeffrey, Brianne A., Jian, Jinlong, Choudhury, Gourav R., Michalson, Kristofer, Mitchell, Thomas W., Buza, Elizabeth L., Chichester, Jessica, Dyer, Cecilia, Bagel, Jessica, Vite, Charles H., Bradbury, Allison M., Wilson, James M.
Publicado 2022

Treating Transthyretin Amyloidosis via Adeno-Associated Virus Vector Delivery of Meganucleases

por Greig, Jenny A., Breton, Camilo, Ashley, Scott N., Martins, Kelly M., Gorsuch, Cassandra, Chorazeczewski, Joanna K., Furmanak, Thomas, Smith, Melanie K., Zhu, Yanqing, Bell, Peter, Shoop, Wendy, Li, Hui, Smith, Jeff, Tomberlin, Ginger, Clark, Peter, Mitchell, Thomas W., Buza, Elizabeth L., Yan, Hanying, Jantz, Derek, Wilson, James M.
Publicado 2022