The Hyper IgM Foundation Blog
Italian National TV – Gene Editing for X-Linked Hyper IgM Syndrome
A late 2024 scientific documentary on Italian National TV (RAI) showcased the work of Dr. Naldini at the San Raffaele Telethon Institute for Gene Therapy (SR-Tiget) in Milan. Dr. Naldini’s research focuses on gene-edited CD4+ T-cells at the CD40LG locus as a potential treatment for Hyper-IgM 1. This research is supported by the Hyper IgM Foundation, which provided a $100,000 grant to Dr. Daniele Canarutto and Prof. Luigi Naldini at SR-Tiget. The documentary indicates that they are nearing the launch of a first-of-its-kind gene-editing clinical trial for X-Linked Hyper IgM Syndrome, with the trial expected to open by the end of 2025. This development represents a potentially game-changing treatment option for patients. In the documentary (which is in Italian with AI-generated English subtitles), adult X-HIGM patient Chris Sheasby, connected to the researchers by the Foundation, shares his experiences with HIGM and his enthusiasm for the new therapy. Prof. Naldini also discusses the research and the planned trial.