Expanding Horizons of CRISPR/Cas Technology: Clinical Advancements, Therapeutic Applications, and Challenges in Gene Therapy

Ahmad Bairqdar; Polina E Karitskaya; Grigory A Stepanov

doi:10.3390/ijms252413321

Expanding Horizons of CRISPR/Cas Technology: Clinical Advancements, Therapeutic Applications, and Challenges in Gene Therapy

Int J Mol Sci. 2024 Dec 12;25(24):13321. doi: 10.3390/ijms252413321.

Authors

Ahmad Bairqdar¹, Polina E Karitskaya², Grigory A Stepanov¹

Affiliations

¹ Institute of Chemical Biology and Fundamental Medicine of the Siberian Branch of the Russian Academy of Sciences, Novosibirsk 630090, Russia.
² Department of Natural Sciences, Novosibirsk State University, Novosibirsk 630090, Russia.

Abstract

CRISPR-Cas technology has transformed the field of gene editing, opening new possibilities for treatment of various genetic disorders. Recent years have seen a surge in clinical trials using CRISPR-Cas-based therapies. This review examines the current landscape of CRISPR-Cas implementation in clinical trials, with data from key registries, including the Australian New Zealand Clinical Trials Registry, the Chinese Clinical Trial Register, and ClinicalTrials.gov. Emphasis is placed on the mechanism of action of tested therapies, the delivery method, and the most recent findings of each clinical trial.

Keywords: CRISPR–Cas; gene therapy; genome editing; xenotransplantation.

Publication types

Review

MeSH terms

Animals
CRISPR-Cas Systems*
Clinical Trials as Topic
Gene Editing* / methods
Genetic Therapy* / methods
Humans

Grants and funding

075-15-2021-1085/Ministry of Science and Higher Education of the Russian Federation