查看文章

S26 factors in the development of the central nervous system (CNS) and severity of the neuropathology. We previously reported that early postnatal systemic delivery of the human aspartoacylase (hASPA) gene by recombinant adeno-associated virus (rAAV) to the CNS of a CD mouse model with neonatal death rescued lethality and partially restored motor function. Now in its 3rd generation, our Canavan gene therapy completely reverses the disease phenotype in the CD KO mouse. For clinical translation to treat juvenile and adult patients, we sought to study and understand the age-limitations for Canavan disease gene therapy. We hypothesized that there is a point-of-noreturn, when gene replacement therapy alone is no longer sufficient to successfully alter the disease outcome. First, Nur7 mice were treated at post-natal (p) day 1 as the gold standard. In the next step, experimental groups were dosed at 6, 12 …