Application of CRISPR-Cas9 technology in non-cancerous hematological disorders / 上海交通大学学报（医学版）

ABSTRACT

The recent discovery of the clustered regularly interspaced short palindromic repeats (CRISPR)-CRISPR associated protein 9 (Cas9) system for precise genome editing has revolutionized methodologies in hematology studies. CRISPR-Cas9 technology can remove and correct genes or mutations, and introduce site-specific therapeutic genes in human cells. Therefore, it has become the ideal targets for inherited haematological disorders and could be the choice to alleviate disease-related symptoms by correcting disease-causing mutations in the near future. Prior to the use of CRISPR-Cas9-mediated gene correction in humans, appropriate delivery systems with high efficiency and specificity must be identified, and ethical guidelines for applying the technology with controllable safety must be established. In this review, the latest applications of CRISPR-Cas9 technology in haematological disorders, current challenges and future directions were discussed.