Gene Editing In Hematopoietic Stem Cells For Monogenic Blood Cell Diseases

Hematopoietic stem cells (HSCs), responsible for generating all blood cells, hold significant potential for gene therapy treatments for various blood disorders.  A clinical trial is in progress, employing Cas9-mediated homology-directed repair to revert the mutation causing sickle cell disease. This technique is also being applied to two primary immune deficiencies, with results indicating restored gene expression and improved B cell functions. Additionally, adenine base editing is being utilized to rectify a mutation causing severe combined immune deficiency. These advancements in gene editing technologies may significantly broaden the treatment possibilities for monogenic blood cell diseases.