In this episode, Dr. Jennifer Leiding, a pediatric clinical immunologist, discusses Chronic Granulomatous Disease (CGD) with Jack and Elie. CGD is an inherited immune deficiency that leads to severe infections and autoimmunity. She explains the dual nature of immune deficiency and autoimmunity in CGD patients, the role of transplantation as a treatment option, and the expectations for families receiving a CGD diagnosis. Dr. Leiding also shares insights from her research on CGD, the complexities surrounding the concept of a cure, and the emotional burden faced by families. The conversation touches on the potential of gene therapy as an experimental treatment and highlights the often-overlooked symptoms experienced by carrier mothers of X-linked CGD. Overall, the episode provides a comprehensive overview of CGD, its challenges, and the ongoing efforts to improve patient outcomes.
The Primary Immune Deficiency Treatment Consortium (PIDTC) is a part of the Rare Diseases Clinical Research Network of the Office of Rare Diseases Research (ORDR), National Center for Advancing Translational Sciences (NCATS), National Institutes of Health (NIH).
This work was supported by the Division of Allergy, Immunology and Transplantation, National Institute of Allergy and Infectious Diseases (DAIT, NIAID) and the ORDR under cooperative agreements U54-AI082973 and U54-NS064808 and grants R13-AI094943 and 1R24AI184316-01.
The content and opinions expressed are solely the responsibility of the authors and do not represent the official policy or position of the NIAID, ORDR, NCATS, NIH, HRSA, or any other agency of the US Government.
Keywords: Chronic Granulomatous Disease, CGD, immune deficiency, transplantation, gene therapy, pediatric immunology, autoimmune disease, PIDTC, patient care, immunology
