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Proceedings of the National Academy of Sciences of the United States of America|March 6, 2021
Homozygous IL37 mutation associated with infantile inflammatory bowel diseaseZinan Z Zhang, Yu Zhang, Tingyan He, et al.
Blood Advances|January 4, 2018
Gene-edited pseudogene resurrection corrects p47phox-deficient chronic granulomatous diseaseRandall K Merling, Douglas B Kuhns, Colin L Sweeney, et al.
Gene Therapy|March 13, 2021
Correction of X-CGD patient HSPCs by targeted CYBB cDNA insertion using CRISPR/Cas9 with 53BP1 inhibition for enhanced homology-directed repairColin L Sweeney, Mara Pavel-Dinu, Uimook Choi, et al.
Biorxiv : the Preprint Server for Biology|June 26, 2025
A novel frameshift mutation in Phosphoinositide 3-kinase regulatory subunit 1 (PIK3R1) causes immunodeficiency and Amyotrophic Lateral Sclerosis (ALS)Brice Calco, Colin L Sweeney, Joseph Steiner, et al.
Molecular Therapy : the Journal of the American Society of Gene Therapy|October 8, 2014
An AAVS1-targeted minigene platform for correction of iPSCs from all five types of chronic granulomatous diseaseRandall K Merling, Colin L Sweeney, Jessica Chu, et al.
Cytotherapy|October 14, 2020
MAGT1 messenger RNA-corrected autologous T and natural killer cells for potential cell therapy in X-linked immunodeficiency with magnesium defect, Epstein-Barr virus infection and neoplasia diseaseJulie Brault, Ronald J Meis, Linhong Li, et al.
Neurology|August 8, 2014
Examination of effects of corticosteroids on skeletal muscles of boys with DMD using MRI and MRSIshu Arpan, Rebecca J Willcocks, Sean C Forbes, et al.
The Lancet. Microbe|March 27, 2025
Evolving patterns of macrolide and fluoroquinolone resistance in Mycoplasma genitalium: an updated systematic review and meta-analysisTeck-Phui Chua, Lenka A Vodstrcil, Gerald L Murray, et al.
The Prostate|April 28, 2023
Comparison of circulating tumor DNA between African American and Caucasian patients with metastatic castrate-resistant prostate cancer post-abiraterone and/or enzalutamideAlbert Jang, Sree M Lanka, Minqi Huang, et al.
Molecular Therapy : the Journal of the American Society of Gene Therapy|January 28, 2017
Rhesus iPSC Safe Harbor Gene-Editing Platform for Stable Expression of Transgenes in Differentiated Cells of All Germ LayersSo Gun Hong, Ravi Chandra Yada, Kyujoo Choi, et al.
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