Introduction: The recent approval of Spinraza (nusinersen), an antisense oligonucleotide,
by U.S. Food and Drug Administration to treat patients with spinal muscular atrophy, has reignited interests
of researchers in designing and testing new gene therapy approaches to treat neurological disorders,
in particular, to curb neurodegenerative diseases of the central nervous system which represent
an ever-increasing public health burden to today's society.
Conclusion: This review highlights several key factors to be taken into consideration to design successful
preclinical and clinical gene therapy experiments with respect to the vehicle of delivery and
the route of administration to CNS-specific targets, with an additional focus on antisense oligonucleotide
therapy and recent clinical trial developments.