05/06/2026
As a pediatric hematologist and oncologist, it’s fascinating to witness what once seemed impossible gradually becoming part of modern medicine.
A landmark 2024 study published in the New England Journal of Medicine showed that most patients with transfusion-dependent beta thalassemia became free from regular blood transfusions after CRISPR-based gene therapy.
Imagine treating a genetic blood disease by editing the patient’s own DNA. What sounded like science fiction a decade ago is now a reality.
Full article here 👇👇👇
Exagamglogene autotemcel (exa-cel) is a nonviral cell therapy designed to reactivate fetal hemoglobin synthesis through ex vivo clustered regularly interspaced short palindromic repeats (CRISPR)–Ca...
