CRISPR/Cas9-mediated genome editing: From basic research to translational medicine.

ABSTRACT: The recent development of the CRISPR/Cas9 system as an efficient and accessible programmable genome-editing tool has revolutionized basic science research. CRISPR/Cas9 system-based technologies have armed researchers with new powerful tools to unveil the impact of genetics on disease development by enabling the creation of precise cellular and animal models of human diseases. The therapeutic potential of these technologies is tremendous, particularly in gene therapy, in which a patient-specific mutation is genetically corrected in order to treat human diseases that are untreatable with conventional therapies. However, the translation of CRISPR/Cas9 into the clinics will be challenging, since we still need to improve the efficiency, specificity and delivery of this technology. In this revie

SUBMITTER: Jacinto FV 

PROVIDER: S-EPMC7171402 | biostudies-literature | 2020 Apr

REPOSITORIES: biostudies-literature