CRISPR-Cas9 technology and its application in haematological disorders.
The recent advent of the Clustered Regularly Interspaced Short Palindromic Repeats (CRISPR)-CRISPR associated protein 9 (Cas9) system for precise genome editing has revolutionized methodologies in haematology and oncology studies. CRISPR-Cas9 technology can be used to remove and correct genes or mutations, and to introduce site-specific therapeutic genes in human cells. Inherited haematological disorders represent ideal targets for CRISPR-Cas9-mediated gene therapy. Correcting disease-causing mutations could alleviate disease-related symptoms in the near future. The CRISPR-Cas9 system is also a useful tool for delineating molecular mechanisms involving haematological malignancies. Prior to the use of CRISPR-Cas9-mediated gene correction in humans, appropriate delivery systems with higher efficiency and specificity must be identified, and ethical guidelines for applying the technology with controllable safety must be established. Here, the latest applications of CRISPR-Cas9 technology in haematological disorders, current challenges and future directions are reviewed and discussed.
The Brown Foundation Institute of Molecular Medicine for the Prevention of Human Diseases (IMM), University of Texas-Health Science Centre at Houston, Houston, TX, USA.
Han
H
Zhang
The Brown Foundation Institute of Molecular Medicine for the Prevention of Human Diseases (IMM), University of Texas-Health Science Centre at Houston, Houston, TX, USA. nami.mccarty@uth.tmc.edu.
Nami
N
McCarty
D000818:Animals; D064113:CRISPR-Cas Systems; D000072669:Gene Editing / Q000379:methods; D018390:Gene Targeting; D018014:Gene Transfer Techniques; D005818:Genetic Engineering / Q000379:methods; D015316:Genetic Therapy / Q000379:methods; D006402:Hematologic Diseases / Q000235:genetics* / Q000628:therapy; D006801:Humans
Clustered Regularly Interspaced Short Palindromic Repeats (CRISPR)-associated protein 9 (Cas9) system; gene editing; haematological disorders; haematological malignancies