# Genetic therapies for neurological diseases

## TL;DR

This paper reviews various genetic therapy approaches for neurological diseases, covering viral vectors, RNA-based therapies, and gene editing.

## Contribution

The paper provides a comprehensive overview of multiple genetic therapy modalities for neurological diseases.

## Key findings

- The paper discusses preclinical and clinical applications of viral vectors for genetic therapies.
- RNA-based therapies and antisense oligonucleotides are examined in both preclinical and clinical contexts.
- Gene editing strategies and neurosurgical techniques for clinical deployment are reviewed.

## Abstract

Often, gene therapy reviews concentrate upon specific therapeutic modalities—particularly either viral vector-mediated or a nonviral approach. Here, we draw together a comprehensive array of knowledge across the field of genetic therapy for genetic neurological disease. The sections on preclinical and clinical application of viral vectors are followed by sections on RNA-based therapies and then by antisense oligonucleotide approaches also in preclinical and clinical settings. We present a separate section on gene editing strategies and conclude with a section elaborating on the neurosurgical techniques and the expertise required for clinical application of many of these technologies.

Genetic therapies have significant potential to treat life-limiting neurological diseases. This review examines the different approaches, clinical successes, and considerations on how to deploy them.