Application and Research Advances of CRISPR/Cas9 in the Treatment of Sickle Cell Anaemia

Abstract

The conventional treatment of sickle cell anaemia is based on the traditional methods including blood transfusions, the use of hydroxyurea and supportive measures. These various strategies improve symptoms, but neither is a cure, and all are associated with various limitations such as transfusion-related infections and drug toxicities. In gene therapy, the strategy used currently is based on the addition of genes into cells by means of lentiviral vector, with the more promising approach being that of gene editing which has been pioneered by the CRISPR/Cas9 technology. By means of this technology, the editors can be targeted towards the hematopoietic stem cells of the patients either by the actual repairs of the causal (sickle mutation) or the aerobic change which involves a reactivation of the fetal Hb production so as to restore natural function. With a very high targeting ability as well as prospects for long lasting effects from a single procedure, the CRISPR/Cas9 technology opens up a possible way of correcting the genetic defect of hereditary disease. The core challenges are primarily fourfold: low editing efficiency, off-target effects, the difficulty of expanding hematopoietic stem cells in vitro, and high treatment costs. This might be a possible solution to a form of treatment for sickle cell anaemia, and mainly lays down the theory of how to treat various forms of monogenic disorders.