NIH VideoCast - Demystifying Medicine — Primary Immunodeficiency: Mechanism and Management

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Demystifying Medicine — Primary Immunodeficiency: Mechanism and Management

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Air date:	Tuesday, January 9, 2024, 4:00:00 PM Time displayed is Eastern Time, Washington DC Local
Views:	Total views: 548 (271 Live, 277 On-demand)
Category:	Demystifying Medicine
Runtime:	02:00:12
Closed captioning:	Check box to display CC outside of the video Note: You can drag the captioning window around and resize it
Description:	In this first session of 2024, Dr. Notarangelo, chief of the NIAID Laboratory of Clinical Immunology and Microbiology, will deliver a lecture titled "Severe Combined Immune Deficiency in the Golden Era of Newborn Screening." He will describe exciting and very recent research on the efficacy of newborn screening for SCID, in which he led a team of investigators of the Primary Immune Deficiency Treatment Consortium (PIDTC) to demonstrate that newborn screening allowed to improve overall survival after transplantation for SCID from 73% up to 92.5%. For the second part of the session, Dr. Puck, a professor of pediatrics at the UCSF School of Medicine, will speak first and deliver a lecture titled "Advances in Gene Therapy for SCID." She will provide background about hematopoietic stem cell gene therapy, the evolution of integrating vectors from gamma retroviruses to current lentiviruses, and the development of lentiviral gene addition to autologous bone marrow CD34 cells to treat Artemis (DCLRE1C) deficient SCID. Most heartwarming, she will describe the case of the first patient treated, a Navajo infant who is now fully immune reconstituted, and as the family boasts, is back on the ‘Res. Come join us as we celebrate and learn from these success stories on January 9. And, as always, be prepared to be dazzled and demystified. The Demystifying Medicine Series includes presentations on pathology, diagnosis, and therapy in the context of major disease problems and current research. Primarily directed toward PhD students, clinicians and program managers, this series is designed to help bridge the gap between advances in biology and their application to major human diseases. Each session includes clinical and basic science components presented by NIH staff and invitees. All students, trainees, fellows, and staff are welcome to participate. For more information go to https://demystifyingmedicine.od.nih.gov

Author:	Luigi Notarangelo (NIAID) and Jennifer Puck (UCSF)

CIT Live ID:	54008
Permanent link:	https://videocast.nih.gov/watch=54008

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