Therapeutic benefit after intracranial gene therapy delivered during the symptomatic stage in a feline model of Sandhoff disease

por McCurdy, Victoria J., Johnson, Aime K., Gray-Edwards, Heather L., Randle, Ashley N., Bradbury, Allison M., Morrison, Nancy E., Hwang, Misako, Baker, Henry J., Cox, Nancy R., Sena-Esteves, Miguel, Martin, Douglas R.
Publicado 2020