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Molecular Therapy : the Journal of the American Society of Gene Therapy|August 1, 2024
Development and IND-enabling studies of a novel Cas9 genome-edited autologous CD34+ cell therapy to induce fetal hemoglobin for sickle cell diseaseVarun Katta, Kiera O'Keefe, Yichao Li, et al.Genome Research|July 29, 2011
Dynamics of the epigenetic landscape during erythroid differentiation after GATA1 restorationWeisheng Wu, Yong Cheng, Cheryl A Keller, et al.Nature Communications|July 1, 2025
Imbalanced TGFβ signalling and autophagy drive erythroid priming of hematopoietic stem cells in β-thalassemiaMaria Rosa Lidonnici, Giulia Chianella, Nicole Mende, et al.Journal of the American College of Cardiology|February 11, 2026
Sirolimus-Eluting Iron Bioresorbable Scaffolds vs Everolimus-Eluting Stents for Percutaneous Coronary Intervention: A Randomized Trial (IRONMAN II)Runlin Gao, Lei Song, Guosheng Fu, et al.Molecular Therapy. Nucleic Acids|February 28, 2023
Forced enhancer-promoter rewiring to alter gene expression in animal modelsScott A Peslak, Selami Demirci, Vemika Chandra, et al.Pediatric Blood & Cancer|May 26, 2018
Sickle Cell Clinical Research and Intervention Program (SCCRIP): A lifespan cohort study for sickle cell disease progression from the pediatric stage into adulthoodJane S Hankins, Jeremie H Estepp, Jason R Hodges, et al.Nature Communications|April 8, 2026
RPS19 and RPL5 haploinsufficient models reveal divergent ribosomal subunit controls of fetal hematopoiesisYuefeng Tang, Te Ling, Rashid Mehmood, et al.Blood Advances|August 27, 2025
A mouse model for hemoglobin SC disease recapitulates characteristic human pathologiesJinbin Zhai, James B Papizan, Yu Yao, et al.Blood Advances|November 8, 2019
Genome editing of HBG1 and HBG2 to induce fetal hemoglobinJean-Yves Métais, Phillip A Doerfler, Thiyagaraj Mayuranathan, et al.The Journal of Clinical Investigation|September 8, 2020
BCL11A enhancer-edited hematopoietic stem cells persist in rhesus monkeys without toxicitySelami Demirci, Jing Zeng, Yuxuan Wu, et al.Pageof 32