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Journal Articles

A First-in-Human Base Edit Gene Therapy for CD40L Deficiency X-Linked Hyper IgM (XHIGM) Syndrome

Suk See De Ravin, Tyra Estwick, Yuzhi Yin, Michelle Ma, Siyuan Liu, Xiaolin Wu, Justin Lack, Andres Zea Vera, Colin Sweeney, Stephen L. Highfill, Christian Wysocki, Ronald J. Meis, Gary A. Dahl, Benjamin P. Kleinstiver, Harry L. Malech

J Hum Immun (2026) 2 (CIS2026): eCIS2026abstract.1.

https://doi.org/10.70962/CIS2026abstract.1

Published: 01 May 2026

View article titled, A First-in-Human Base Edit Gene Therapy for CD40L Deficiency X-Linked Hyper IgM (XHIGM) Syndrome

Journal Articles

Treatment of Autosomal Recessive Interleukin-7 Deficiency with NT-I7 (Efineptakin Alfa), Long-Acting Recombinant Human Interleukin-7: An Expanded Access Protocol

Derek MacMath, Peiying Ye, Amay Shah, Jasmine Mensah, Triscia Martin, Megan Anderson, Andres Zea Vera, Alex Oh, Sara Ferrando Martinez, Klaus Warnatz, Luigi Notarangelo, Jean-Laurent Casanova, Jose Luis Franco, Anne Puel, Ken Natsuga, Andrea Lisco

J Hum Immun (2025) 1 (CIS2025): CIS2025abstract.117.

https://doi.org/10.70962/CIS2025abstract.117

Published: 25 April 2025

View article titled, Treatment of Autosomal Recessive Interleukin-7 Deficiency with NT-I7 (Efineptakin Alfa), Long-Acting Recombinant Human Interleukin-7: An Expanded Access Protocol