Autologous hematopoietic stem cell transplantation improves disease-free survival in pediatric extraocular retinoblastoma patients
Children With Eye Cancer Now Have a New Hope for Survival
This retrospective analysis included 12 children with extraocular retinoblastoma treated with autologous hematopoietic stem cell transplanta…
Children with advanced eye cancer can now achieve long-term survival using their own stem cells, especially if the disease has not reached t…
Reni-cel gene therapy shows transfusion independence in small phase 1-2 trial for β-thalassemia
New Gene Edit Stops Blood Transfusions
A single-group phase 1-2 trial in 9 adults with transfusion-dependent β-thalassemia found all participants achieved neutrophil engraftment a…
A new gene edit reactivates natural red blood cell production, potentially ending the need for lifelong transfusions in adults with severe b…
Renizgamglogene autotemcel infusion showed engraftment and hemoglobin increases in severe sickle cell disease patients.
One Gene Edit Stopped Painful Sickle Cell Crises in Most Patients
This single-group, open-label, multicenter Phase 1-2 trial enrolled 28 patients with severe sickle cell disease aged 12 to 50 years. Followi…
A single infusion of CRISPR-edited stem cells eliminated painful sickle cell crises in 27 out of 28 patients by switching the body back to p…