Gene and Cell Therapies for Beta-Globinopathies By Punam Malik
English | PDF,EPUB | 2017 | 254 Pages | ISBN : 1493972979 | 5.02 MB
Hemoglobin defects, specifically sickle cell disease & thalassemia, combined, constitute the most common monogenic disorders in the world. In fact, nearly 2% of the world’s population carries a globin gene mutation. The transfer of the corrective globin gene through the HSC compartment by allogeneic HSC transplantation (HSCT) has already proven curative in both SCD and thalassemia patients, and provides the proof of concept that genetic manipulation of the defective organ might be equally therapeutic.