MSKCC: Clinical Trial for Beta-Thalassemia Brings Important Insights for Treating Blood Disorders with Stem Cell Gene Therapy

After three decades of research, Memorial Sloan Kettering Cancer Center investigators may have found a new treatment option for patients with an inherited blood disorder called beta (β)-thalassemia. The approach, led by MSK physician-scientist Michel Sadelain, involves using a new stem-cell-based form of gene therapy. Results from a phase 1 clinical trial testing this treatment were reported in Nature Medicine on January 3, 2022.

View the rest of the article here

View the PDF