Fetal Brain-directed AAV Gene Therapy Results in Rapid, Robust, and Persistent Transduction of Mouse Choroid Plexus Epithelia

por Haddad, Marie Reine, Donsante, Anthony, Zerfas, Patricia, Kaler, Stephen G
Publicado 2013

Cerebrospinal Fluid-Directed rAAV9-rsATP7A Plus Subcutaneous Copper Histidinate Advance Survival and Outcomes in a Menkes Disease Mouse Model

por Haddad, Marie Reine, Choi, Eun-Young, Zerfas, Patricia M., Yi, Ling, Martinelli, Diego, Sullivan, Patricia, Goldstein, David S., Centeno, Jose A., Brinster, Lauren R., Ralle, Martina, Kaler, Stephen G.
Publicado 2018