Gene Therapy in Cerebrovascular Diseases

Rodney   Allanigue   Gabriel; Guo-Yuan      Yang

Abstract

Several studies utilize gene therapy technology to study the molecular mechanisms and therapeutic approaches to a variety of neurological diseases. Although gene therapy for neuro-oncology and neurodegenerative diseases has advanced to the clinical arena, those of cerebrovascular nature have remained in the experimental stage and demonstrate promising results. Before gene therapy in cerebrovascular disorders can be successful, various obstacles need to be addressed. In this review, we focus on the fundamentals of gene therapy adapted to cerebrovascular diseases, which include cerebral ischemia, post-subarachnoid hemorrhagic vasospasm, arteriovenous malformations, etc. We discuss: 1) the utilization of gene delivery vehicle, through viral, non-viral, or cellular vectors; 2) the routes and specific delivery of the vector to its target cells or tissue in the central nervous system; 3) the studies that have utilized in vivo and ex vivo gene delivery approaches and their success in the attenuation of cerebrovascular injuries; and finally 4) the future of gene therapy in this field.

Keywords: Adeno-associated virus, adenovirus, herpes simplex virus, ischemia, stroke, vasospasm

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