Last Updated: 09/26/2026
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Found 23 publications
A differentiated β-globin gene replacement strategy uses heterologous introns to restore physiological expression.
Journal: Molecular therapy : the journal of the American Society of Gene Therapy
Published: July 03, 2024
Functional screening in human HSPCs identifies optimized protein-based enhancers of Homology Directed Repair.
Journal: Nature communications
Published: August 07, 2023
Transient inhibition of 53BP1 increases the frequency of targeted integration in human hematopoietic stem and progenitor cells.
Journal: Nature communications
Published: February 23, 2023
Clinically relevant gene editing in hematopoietic stem cells for the treatment of pyruvate kinase deficiency.
Journal: Molecular therapy. Methods & clinical development
Published: February 02, 2021
Development of β-globin gene correction in human hematopoietic stem cells as a potential durable treatment for sickle cell disease.
Journal: Science translational medicine
Published: October 13, 2020
Cas9-AAV6 gene correction of beta-globin in autologous HSCs improves sickle cell disease erythropoiesis in mice.
Journal: Nature communications
Published: October 05, 2020
The TRACE-Seq method tracks recombination alleles and identifies clonal reconstitution dynamics of gene targeted human hematopoietic stem cells.
Journal: Nature communications
Published: April 06, 2020
Gene replacement of α-globin with β-globin restores hemoglobin balance in β-thalassemia-derived hematopoietic stem and progenitor cells.
Journal: Nature medicine
Published: October 08, 2019
Highly efficient editing of the β-globin gene in patient-derived hematopoietic stem and progenitor cells to treat sickle cell disease.
Journal: Nucleic acids research
Published: February 27, 2019
Diamond Blackfan anemia is mediated by hyperactive Nemo-like kinase.
Journal: Nature communications
Published: October 23, 2018
Last Updated: 09/26/2026