-
Article
Open AccessCorrection: PDGFB-expressing mesenchymal stem cells improve human hematopoietic stem cell engraftment in immunodeficient mice
An amendment to this paper has been published and can be accessed via a link at the top of the paper.
-
Article
Open AccessPDGFB-expressing mesenchymal stem cells improve human hematopoietic stem cell engraftment in immunodeficient mice
The bone marrow (BM) niche regulates multiple hematopoietic stem cell (HSC) processes. Clinical treatment for hematological malignancies by HSC transplantation often requires preconditioning via total body irr...
-
Article
Open AccessCuring hemophilia A by NHEJ-mediated ectopic F8 insertion in the mouse
Hemophilia A, a bleeding disorder resulting from F8 mutations, can only be cured by gene therapy. A promising strategy is CRISPR-Cas9-mediated precise insertion of F8 in hepatocytes at highly expressed gene loci,...
-
Article
Open AccessThrombopoietin knock-in augments platelet generation from human embryonic stem cells
Refinement of therapeutic-scale platelet production in vitro will provide a new source for transfusion in patients undergoing chemotherapy or radiotherapy. However, procedures for cost-effective and scalable p...
-
Article
Open AccessEfficient precise knockin with a double cut HDR donor after CRISPR/Cas9-mediated double-stranded DNA cleavage
Precise genome editing via homology-directed repair (HDR) after double-stranded DNA (dsDNA) cleavage facilitates functional genomic research and holds promise for gene therapy. However, HDR efficiency remains ...
