Branden Baptiste, diagnosed with sickle cell disease at a young age, struggled with chronic complications affecting his kidneys, lungs, joints, and eyes. Seeking a cure, he participated in a groundbreaking gene therapy trial called BEACON at Boston Children’s Hospital. Through base editing, the most precise form of gene therapy, Branden’s red blood cells were edited to produce fetal hemoglobin, eliminating sickling. After successful treatment, Branden experienced significant improvement, discontinued sickle cell medication, and has been able to exercise without pain. Early results from the BEACON trial show promise in treating sickle cell disease and improving quality of life for patients.
Source link
Base editing, a new form of gene therapy, leaves patient feeling ‘more than fine’
