Gene Therapy for Neurodegenerative Diseases: Slowing Down the Ticking Clock

N/ACitations
Citations of this article
201Readers
Mendeley users who have this article in their library.

Abstract

Gene therapy is an emerging and powerful therapeutic tool to deliver functional genetic material to cells in order to correct a defective gene. During the past decades, several studies have demonstrated the potential of AAV-based gene therapies for the treatment of neurodegenerative diseases. While some clinical studies have failed to demonstrate therapeutic efficacy, the use of AAV as a delivery tool has demonstrated to be safe. Here, we discuss the past, current and future perspectives of gene therapies for neurodegenerative diseases. We also discuss the current advances on the newly emerging RNAi-based gene therapies which has been widely studied in preclinical model and recently also made it to the clinic.

Cite

CITATION STYLE

APA

Martier, R., & Konstantinova, P. (2020, September 18). Gene Therapy for Neurodegenerative Diseases: Slowing Down the Ticking Clock. Frontiers in Neuroscience. Frontiers Media S.A. https://doi.org/10.3389/fnins.2020.580179

Register to see more suggestions

Mendeley helps you to discover research relevant for your work.

Already have an account?

Save time finding and organizing research with Mendeley

Sign up for free