Biblio
CRISPR/Cas9n-mediated ELANE promoter editing for gene therapy of severe congenital neutropenia. Mol Ther. 2024.
Efficient designer nuclease-based homologous recombination enables direct PCR screening for footprintless targeted human pluripotent stem cells. Stem Cell Reports. 2014;2(1):107-18.
Epitope-engineered human hematopoietic stem cells are shielded from CD123-targeted immunotherapy. J Exp Med. 2023;220(12).
Quantitative evaluation of chromosomal rearrangements in gene-edited human stem cells by CAST-Seq. Cell Stem Cell. 2021.
Rescue of DNA-PK Signaling and T-Cell Differentiation by Targeted Genome Editing in a prkdc Deficient iPSC Disease Model. PLoS Genet. 2015;11(5):e1005239.
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