Disease Focus: Hyper IgM Syndrome

---

Targeted Gene Editing in the Treatment of X-Linked Hyper-IgM Syndrome

Research Objective We are seeking to develop site-specific hematopoietic stem cell gene therapy with autologous transplant as a definitive treatment option for X-linked Hyper-IgM Syndrome. Impact These studies would bring stem cell gene therapy for X-HIGM closer to the clinic, as there are currently no options for those without an HLA match or with infections […]

Ex Vivo Gene Editing of Human Hematopoietic Stem Cells for the Treatment of X-Linked Hyper-IgM Syndrome

Translational Candidate Human hematopoietic stem cells that have been gene-corrected at the CD40L gene to treat patients with X-Linked Hyper-IgM Syndrome Area of Impact These studies will bring stem cell gene therapy for XHIM closer to the clinic especially those without an HLA match or infections too severe for HSCT. Mechanism of Action The CRISPR/Cas9 […]