Breaking Boundaries in the Brain-Advances in Editing Tools for Neurogenetic Disorders.

ABSTRACT: Monogenic neurological disorders are devastating, affecting hundreds of millions of people globally and present a substantial burden to individuals, carers, and healthcare systems. These disorders are predominantly caused by inherited or de novo variants that result in impairments to nervous system development, neurodegeneration, or impaired neuronal function. No cure exists for these disorders with many being refractory to medication. However, since monogenic neurological disorders have a single causal factor, they are also excellent targets for innovative, therapies such as gene therapy. Despite this promise, gene transfer therapies are limited in that they are only suitable for neurogenetic disorders that fit within the technological reach of these therapies. The limitations incl

SUBMITTER: Coorey BA 

PROVIDER: S-EPMC8525368 | biostudies-literature | 2021

REPOSITORIES: biostudies-literature