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Science Translational Medicine
|
June 17, 2021
Development of β-globin gene correction in human hematopoietic stem cells as a potential durable treatment for sickle cell disease
Annalisa Lattanzi, Joab Camarena, Premanjali Lahiri, et al.
Cell Stem Cell
|
September 29, 2020
Loss of Extreme Long-Range Enhancers in Human Neural Crest Drives a Craniofacial Disorder
Hannah K Long, Marco Osterwalder, Ian C Welsh, et al.
Molecular Therapy : the Journal of the American Society of Gene Therapy
|
April 1, 2021
Targeted replacement of full-length CFTR in human airway stem cells by CRISPR-Cas9 for pan-mutation correction in the endogenous locus
Sriram Vaidyanathan, Ron Baik, Lu Chen, et al.
Cell Reports
|
January 4, 2025
Dual α-globin-truncated erythropoietin receptor knockin restores hemoglobin production in α-thalassemia-derived erythroid cells
Simon N Chu, Eric Soupene, Devesh Sharma, et al.
Molecular Therapy. Nucleic Acids
|
January 6, 2016
TALENs Facilitate Single-step Seamless SDF Correction of F508del CFTR in Airway Epithelial Submucosal Gland Cell-derived CF-iPSCs
Shingo Suzuki, R Geoffrey Sargent, Beate Illek, et al.
Nature Medicine
|
March 19, 2021
Gene replacement of α-globin with β-globin restores hemoglobin balance in β-thalassemia-derived hematopoietic stem and progenitor cells
M Kyle Cromer, Joab Camarena, Renata M Martin, et al.
Blood
|
February 24, 2021
Enhanced homology-directed repair for highly efficient gene editing in hematopoietic stem/progenitor cells
Suk See De Ravin, Julie Brault, Ronald J Meis, et al.
Blood Advances
|
December 14, 2023
Genetically corrected RAG2-SCID human hematopoietic stem cells restore V(D)J-recombinase and rescue lymphoid deficiency
Mara Pavel-Dinu, Cameron L Gardner, Yusuke Nakauchi, et al.
Cell Stem Cell
|
December 17, 2019
High-Efficiency, Selection-free Gene Repair in Airway Stem Cells from Cystic Fibrosis Patients Rescues CFTR Function in Differentiated Epithelia
Sriram Vaidyanathan, Ameen A Salahudeen, Zachary M Sellers, et al.
Developmental Cell
|
April 3, 2024
Lineage-tracing hematopoietic stem cell origins in vivo to efficiently make human HLF+ HOXA+ hematopoietic progenitors from pluripotent stem cells
Jonas L Fowler, Sherry Li Zheng, Alana Nguyen, et al.
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Search research articles
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Showing results (121-130 of 141) with videos related to
Sort By:
Page
of 15
Science Translational Medicine
|
June 17, 2021
Development of β-globin gene correction in human hematopoietic stem cells as a potential durable treatment for sickle cell disease
Annalisa Lattanzi, Joab Camarena, Premanjali Lahiri, et al.
Cell Stem Cell
|
September 29, 2020
Loss of Extreme Long-Range Enhancers in Human Neural Crest Drives a Craniofacial Disorder
Hannah K Long, Marco Osterwalder, Ian C Welsh, et al.
Molecular Therapy : the Journal of the American Society of Gene Therapy
|
April 1, 2021
Targeted replacement of full-length CFTR in human airway stem cells by CRISPR-Cas9 for pan-mutation correction in the endogenous locus
Sriram Vaidyanathan, Ron Baik, Lu Chen, et al.
Cell Reports
|
January 4, 2025
Dual α-globin-truncated erythropoietin receptor knockin restores hemoglobin production in α-thalassemia-derived erythroid cells
Simon N Chu, Eric Soupene, Devesh Sharma, et al.
Molecular Therapy. Nucleic Acids
|
January 6, 2016
TALENs Facilitate Single-step Seamless SDF Correction of F508del CFTR in Airway Epithelial Submucosal Gland Cell-derived CF-iPSCs
Shingo Suzuki, R Geoffrey Sargent, Beate Illek, et al.
Nature Medicine
|
March 19, 2021
Gene replacement of α-globin with β-globin restores hemoglobin balance in β-thalassemia-derived hematopoietic stem and progenitor cells
M Kyle Cromer, Joab Camarena, Renata M Martin, et al.
Blood
|
February 24, 2021
Enhanced homology-directed repair for highly efficient gene editing in hematopoietic stem/progenitor cells
Suk See De Ravin, Julie Brault, Ronald J Meis, et al.
Blood Advances
|
December 14, 2023
Genetically corrected RAG2-SCID human hematopoietic stem cells restore V(D)J-recombinase and rescue lymphoid deficiency
Mara Pavel-Dinu, Cameron L Gardner, Yusuke Nakauchi, et al.
Cell Stem Cell
|
December 17, 2019
High-Efficiency, Selection-free Gene Repair in Airway Stem Cells from Cystic Fibrosis Patients Rescues CFTR Function in Differentiated Epithelia
Sriram Vaidyanathan, Ameen A Salahudeen, Zachary M Sellers, et al.
Developmental Cell
|
April 3, 2024
Lineage-tracing hematopoietic stem cell origins in vivo to efficiently make human HLF+ HOXA+ hematopoietic progenitors from pluripotent stem cells
Jonas L Fowler, Sherry Li Zheng, Alana Nguyen, et al.
Page
of 15