Article
Gene-corrected regulatory T cell therapy for IL2RA deficiency.
Molecular therapy : the journal of the American Society of Gene Therapy - 4 Mar 2026
Ha Alvin S, Kalter Nechama, Rosenberg Michael, Acevedo Luis A, Liang Brianna, Liu Weihong, Paruthiyil Sreenivasan, Sinha Meenal, Vu Alexander, Nguyen Vinh, Qi Zhongxia, Krishnappa Netravathi, Shu Jinna, Yu Jingwei, Catanzaro Jason, Bluestone Jeffrey A, Tang Qizhi, Urnov Fyodor D, Marson Alexander, Hendel Ayal, Herold Kevan C, Shy Brian R, Esensten Jonathan H
Abstract excerpt
Bi-allelic germline deficiency of IL2RA causes a rare autoimmune disease with impaired regulatory T cell (Treg) function and interleukin-2 (IL-2) signaling. Definitive treatment is currently limited to allogeneic hematopoietic stem cell transplantation, which carries significant morbidity and mortality risks. We previously identified a family with three siblings affected by compound heterozygous mutations in...
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