Viral-Mediated Gene Replacement Therapy in the Developing Central Nervous System: Current Status and Future Directions.

Pediatric Neurology
Julie UchitelMohamad A Mikati

Abstract

The past few years have witnessed rapid developments in viral-mediated gene replacement therapy for pediatric central nervous system neurogenetic disorders. Here, we provide pediatric neurologists with an up-to-date, comprehensive overview of these developments and note emerging trends for future research. This review presents the different types of viral vectors used in viral-mediated gene replacement therapy; the fundamental properties of viral-mediated gene replacement therapy; the challenges associated with the use of this therapy in the central nervous system; the pathway for therapy development, from translational basic science studies to clinical trials; and an overview of the therapies that have reached clinical trials in patients. Current viral platforms under investigation include adenovirus vectors, adeno-associated viral vectors, lentiviral/retroviral vectors, and herpes simplex virus type 1 vectors. This review also presents an in-depth analysis of numerous studies that investigated these viral platforms in cultured cells and in transgenic animal models for pediatric neurogenetic disorders. Viral vectors have been applied to clinical trials for many different pediatric neurogenetic disorders, including Canavan dise...Continue Reading

Citations

Aug 17, 2021·Brain Communications·Julie UchitelMohamad A Mikati

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