Embryo and fetal gene editing: Technical challenges and progress toward clinical applications.

ABSTRACT: Gene modification therapies (GMTs) are slowly but steadily making progress toward clinical application. As the majority of rare diseases have an identified genetic cause, and as rare diseases collectively affect 5% of the global population, it is increasingly important to devise gene correction strategies to address the root causes of the most devastating of these diseases and to provide access to these novel therapies to the most affected populations. The main barriers to providing greater access to GMTs continue to be the prohibitive cost of developing these novel drugs at clinically relevant doses, subtherapeutic effects, and toxicity related to the specific agents or high doses required. In vivo strategy and treating younger patients at an earlier course of their disease could l

SUBMITTER: Mattar CNZ 

PROVIDER: S-EPMC10963250 | biostudies-literature | 2024 Jun

REPOSITORIES: biostudies-literature