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Yuzhi Yin

National Institute of Allergy and Infectious Diseases

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JUL 14, 2026 · JOURNAL ARTICLE

Base Editing Hematopoietic Stem/Progenitor Cell Gene Therapy for Treatment of X-Linked Severe Combined Immunodeficiency

Journal of Human Immunity

First-in-human Phase I/II trial with only 3 patients treated and 6-month follow-up data reported for one patient; early data suggest safety and potential efficacy but remain insufficient for definitive clinical guidance.

PreliminaryHarry L. Malech Siyuan Liu Immunodeficiency and Autoimmune Disorders

Reported

Gene correction frequency in cell…80%

Gene correction in myeloid compar…70%

Gene correction in B cell compart…94%

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