Challenges and Advances in Gene Therapy Approaches for Neurodegenerative Disorders

E-ISSN： 1875-5631|17|3|187-193

ISSN： 1566-5232

Source： Current Gene Therapy, Vol.17, Iss.3, 2017-12, pp. : 187-193

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Abstract

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 interestsof 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 representan ever-increasing public health burden to today#39;s society.

Conclusion: This review highlights several key factors to be taken into consideration to design successfulpreclinical and clinical gene therapy experiments with respect to the vehicle of delivery andthe route of administration to CNS-specific targets, with an additional focus on antisense oligonucleotidetherapy and recent clinical trial developments.