Future of genetic therapies for rare genetic diseases: what to expect for the next 15 years?

ABSTRACT:

Introduction

Rare genetic diseases affect millions of people worldwide. Most of them are caused by defective genes that impair quality of life and can lead to premature death. As genetic therapies aim to fix or replace defective genes, they are considered the most promising treatment for rare genetic diseases. Yet, as these therapies are still under development, it is still unclear whether they will be successful in treating these diseases. This study aims to address this gap by assessing researchers' opinions on the future of genetic therapies for the treatment of rare genetic diseases.

Methods

We conducted a global cross-sectional web-based survey of researchers who recently authored peer-reviewed articles related to rare genetic diseases.

Results

We assessed the op

SUBMITTER: Braga LAM 

PROVIDER: S-EPMC10032453 | biostudies-literature | 2022 Jan-Dec

REPOSITORIES: biostudies-literature