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EBMT 2024 | Autologous gene editing using the CRISPR system to treat primary immunodeficiency disorders

Thomas Fox • 15 Apr 2024
EBMT 2024
General Rare Diseases

Thomas Fox, MbChB, MD, PhD, University College London Hospitals NHS Foundation Trust, London, UK, discusses CRISPR-based autologous gene therapy for treating primary immunodeficiency disorders (PIDs). This offers an alternative to bone marrow transplantation, avoiding the risks of graft-versus-host disease (GvHD) and the need for a compatible donor. However, there remain challenges in making gene editing strategies accessible to patients. This interview took place at the 50th Annual Meeting of the EBMT in Glasgow, Scotland.

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