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Blood Advances|May 26, 2019
The E3 ligase adaptor molecule SPOP regulates fetal hemoglobin levels in adult erythroid cellsXianjiang Lan, Eugene Khandros, Peng Huang, et al.
Science (New York, N.Y.)|July 21, 2018
Domain-focused CRISPR screen identifies HRI as a fetal hemoglobin regulator in human erythroid cellsJeremy D Grevet, Xianjiang Lan, Nicole Hamagami, et al.
Molecular Therapy : the Journal of the American Society of Gene Therapy|January 30, 2021
Lentiviral vector ALS20 yields high hemoglobin levels with low genomic integrations for treatment of beta-globinopathiesLaura Breda, Valentina Ghiaccio, Naoto Tanaka, 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.
Scientific Reports|November 12, 2017
Structural and Functional Insight of Sphingosine 1-Phosphate-Mediated Pathogenic Metabolic Reprogramming in Sickle Cell DiseaseKaiqi Sun, Angelo D'Alessandro, Mostafa H Ahmed, et al.
Blood|July 1, 2024
Use of HSC-targeted LNP to generate a mouse model of lethal α-thalassemia and treatment via lentiviral gene therapyMaxwell E Chappell, Laura Breda, Lucas Tricoli, et al.
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