Current clinical treatments for central nervous system (CNS) diseases, such as Parkinson's
disease and glioblastoma do not halt disease progression and have significant treatment
morbidities. Gene therapy has the potential to “permanently” correct disease by bringing in a
normal gene to correct a mutant gene deficiency, knocking down mRNA of mutant alleles,
and inducing cell-death in cancer cells using transgenes encoding apoptosis-inducing
proteins. Promising results in clinical trials of eye disease (Leber's congenital aumorosis) …