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Revisiting gene delivery to the brain: silencing and editing. | LitMetric

Revisiting gene delivery to the brain: silencing and editing.

Biomater Sci

NOVA Medical School, Faculdade de Ciências Médicas, Universidade Nova de Lisboa, 1169-056 Lisboa, Portugal.

Published: February 2021

AI Article Synopsis

  • * Gene therapy shows potential for treating genetic and acquired brain diseases by silencing or editing specific genes, with progress made in delivering non-coding RNAs and gene-editing technology to the brain over the last five years.
  • * The review highlights advances in developing non-toxic, trackable non-viral vectors for gene delivery, discussing their ability to release genes in response to triggers and their applications in various brain diseases during pre-clinical and clinical trials.

Article Abstract

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.

Download full-text PDF

Source
http://dx.doi.org/10.1039/d0bm01278eDOI Listing

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