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Revisiting gene delivery to the brain: silencing and editing

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Neurodegenerative disorders, ischemic brain diseases, and brain tumors are debilitating diseases that severely impact a person's life and could possibly lead to their demise if left untreated. Many of these diseases do not respond to small molecule therapeutics and have no effective long-term therapy. Gene therapy offers the promise of treatment or even a cure for both genetic and acquired brain diseases, mediated by either silencing or editing disease-specific genes. Indeed, in the last 5 years, significant progress has been made in the delivery of non-coding RNAs as well as gene-editing formulations to the brain. Unfortunately, the delivery is a major limiting factor for the success of gene therapies. Both viral and non-viral vectors have been used to deliver genetic information into a target cell, but they have limitations. Viral vectors provide excellent transduction efficiency but are associated with toxic effects and have limited packaging capacity; however, non-viral vectors are less toxic and show a high packaging capacity at the price of low transfection efficiency. Herein, we review the progress made in the field of brain gene therapy, particularly in the design of non-toxic and trackable non-viral vectors, capable of controlled release of genes in response to internal/external triggers, and in the delivery of formulations for gene editing. The application of these systems in the context of various brain diseases in pre-clinical and clinical tests will be discussed. Such promising approaches could potentially pave the way for clinical realization of brain gene therapies.

Graphical abstract: Revisiting gene delivery to the brain: silencing and editing

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Article information

31 Jul 2020
23 Nov 2020
First published
24 Nov 2020

Biomater. Sci., 2021, Advance Article
Article type
Review Article

Revisiting gene delivery to the brain: silencing and editing

J. Conniot, S. Talebian, S. Simões, L. Ferreira and J. Conde, Biomater. Sci., 2021, Advance Article , DOI: 10.1039/D0BM01278E

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