The Non-Malignant Channel is supported with funding from Agios (Gold).
The Thalassemia Channel is supported with funding from Agios (Gold).
VJHemOnc is an independent medical education platform. Supporters, including channel supporters, have no influence over the production of content. The levels of sponsorship listed are reflective of the amount of funding given to support the channel.
ASH 2025 | A case report used to evaluate gene therapy outcomes in transfusion-dependent β-thalassemia
In this video, Gonzalo De Luna, MD, Henri-Mondor University Hospital, Créteil, France, discusses real-world data from the first French patient treated with a CRISPR-Cas9–based gene therapy approach for transfusion-dependent β-thalassemia. Dr De Luna highlights that there was no evidence of clonal dominance or structural rearrangement at 16 weeks of follow-up, and the pre-existing DNMT3A mutation remained stable, suggesting no clonal expansion. This interview took place at the 67th ASH Annual Meeting and Exposition, held in Orlando, FL.
These works are owned by Magdalen Medical Publishing (MMP) and are protected by copyright laws and treaties around the world. All rights are reserved.
