Molecular Marvels: Small Molecules Paving the Way for Enhanced Gene Therapy.

ABSTRACT: In the rapidly evolving landscape of genetic engineering, the advent of CRISPR-Cas technologies has catalyzed a paradigm shift, empowering scientists to manipulate the genetic code with unprecedented accuracy and efficiency. Despite the remarkable capabilities inherent to CRISPR-Cas systems, recent advancements have witnessed the integration of small molecules to augment their functionality, introducing new dimensions to the precision and versatility of gene editing applications. This review delves into the synergy between CRISPR-Cas technologies based specifically on Cas9 and small-molecule drugs, elucidating the pivotal role of chemicals in optimizing target specificity and editing efficiency. By examining a diverse array of applications, ranging from therapeutic interventions to agricul

SUBMITTER: Hasselbeck S 

PROVIDER: S-EPMC10818943 | biostudies-literature | 2023 Dec

REPOSITORIES: biostudies-literature