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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.
Science Translational Medicine|October 16, 2024
High-fidelity PAMless base editing of hematopoietic stem cells to treat chronic granulomatous diseaseVera Bzhilyanskaya, Linyuan Ma, Siyuan Liu, et al.
Nature Communications|June 28, 2022
Lentivector cryptic splicing mediates increase in CD34+ clones expressing truncated HMGA2 in human X-linked severe combined immunodeficiencySuk See De Ravin, Siyuan Liu, Colin L Sweeney, et al.
The Journal of Clinical Investigation|August 25, 2022
Mast cell-derived factor XIIIA contributes to sexual dimorphic defense against group B streptococcal infectionsAdrian M Piliponsky, Kavita Sharma, Phoenicia Quach, et al.
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