AAV-Mediated Gene Delivery in Adult GM1-Gangliosidosis Mice Corrects Lysosomal Storage in CNS and Improves Survival

Por Baek, Rena C., Broekman, Marike L. D., Leroy, Stanley G., Tierney, Laryssa A., Sandberg, Michael A., d'Azzo, Alessandra, Seyfried, Thomas N., Sena-Esteves, Miguel
Publicado em 2010

Sustained normalization of neurological disease after intracranial gene therapy in a feline model**

Por McCurdy, Victoria J., Johnson, Aime K., Gray-Edwards, Heather, Randle, Ashley N., Brunson, Brandon L., Morrison, Nancy E., Salibi, Nouha, Johnson, Jacob A., Hwang, Misako, Beyers, Ronald J., Leroy, Stanley G., Maitland, Stacy, Denney, Thomas S., Cox, Nancy R., Baker, Henry J., Sena-Esteves, Miguel, Martin, Douglas R.
Publicado no Sci Transl Med (2014)

Therapeutic Response in Feline Sandhoff Disease Despite Immunity to Intracranial Gene Therapy

Por Bradbury, Allison M, Cochran, J Nicholas, McCurdy, Victoria J, Johnson, Aime K, Brunson, Brandon L, Gray-Edwards, Heather, Leroy, Stanley G, Hwang, Misako, Randle, Ashley N, Jackson, Laura S, Morrison, Nancy E, Baek, Rena C, Seyfried, Thomas N, Cheng, Seng H, Cox, Nancy R, Baker, Henry J, Cachón-González, M Begona, Cox, Timothy M, Sena-Esteves, Miguel, Martin, Douglas R
Publicado em 2013