Last Updated: 09/26/2026
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Found 66 publications
Prime Editing for p47phox-Deficient Chronic Granulomatous Disease.
Journal: The New England journal of medicine
Published: December 08, 2025
Long-Term Safety and Efficacy of Gene Therapy for Adenosine Deaminase Deficiency.
Journal: The New England journal of medicine
Published: October 15, 2025
A novel frameshift mutation in Phosphoinositide 3-kinase regulatory subunit 1 (PIK3R1) causes immunodeficiency and Amyotrophic Lateral Sclerosis (ALS).
Journal: bioRxiv : the preprint server for biology
Published: June 26, 2025
Lymphedema in patients with X-linked severe combined immunodeficiency.
Journal: The journal of allergy and clinical immunology. Global
Published: June 10, 2025
Multiomics dissection of human RAG deficiency reveals distinctive patterns of immune dysregulation but a common inflammatory signature.
Journal: Science immunology
Published: January 10, 2025
High-fidelity PAMless base editing of hematopoietic stem cells to treat chronic granulomatous disease.
Journal: Science translational medicine
Published: October 16, 2024
Custom CRISPR-Cas9 PAM variants via scalable engineering and machine learning.
Journal: Nature
Published: April 11, 2024
Sensitive and unbiased genome-wide profiling of base-editor-induced off-target activity using CHANGE-seq-BE.
Journal: Nature biotechnology
Published: March 24, 2024
Exonic knockout and knockin gene editing in hematopoietic stem and progenitor cells rescues RAG1 immunodeficiency.
Journal: Science translational medicine
Published: February 07, 2024
Last Updated: 09/26/2026