Genome Editing for CNS Disorders

Fábio Duarte; Nicole Déglon

doi:10.3389/fnins.2020.579062

Genome Editing for CNS Disorders

Front Neurosci. 2020 Oct 22:14:579062. doi: 10.3389/fnins.2020.579062. eCollection 2020.

Authors

Fábio Duarte^{1

2}, Nicole Déglon^{1

2}

Affiliations

¹ Laboratory of Neurotherapies and NeuroModulation, Department of Clinical Neurosciences, Lausanne University Hospital and Lausanne University, Lausanne, Switzerland.
² Laboratory of Neurotherapies and NeuroModulation, Neuroscience Research Center, Lausanne University Hospital and Lausanne University, Lausanne, Switzerland.

Abstract

Central nervous system (CNS) disorders have a social and economic burden on modern societies, and the development of effective therapies is urgently required. Gene editing may prevent or cure a disease by inducing genetic changes at endogenous loci. Genome editing includes not only the insertion, deletion or replacement of nucleotides, but also the modulation of gene expression and epigenetic editing. Emerging technologies based on ZFs, TALEs, and CRISPR/Cas systems have extended the boundaries of genome manipulation and promoted genome editing approaches to the level of promising strategies for counteracting genetic diseases. The parallel development of efficient delivery systems has also increased our access to the CNS. In this review, we describe the various tools available for genome editing and summarize in vivo preclinical studies of CNS genome editing, whilst considering current limitations and alternative approaches to overcome some bottlenecks.

Keywords: CNS; CRISPR/Cas; TALEs; ZFs; genome editing.

Publication types

Review