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Leukemia|July 18, 2023
Dual intron-targeted CRISPR-Cas9-mediated disruption of the AML RUNX1-RUNX1T1 fusion gene effectively inhibits proliferation and decreases tumor volume in vitro and in vivoSigne Neldeborg, Johannes Frasez Soerensen, Charlotte Thornild Møller, et al.Molecular Therapy : the Journal of the American Society of Gene Therapy|March 1, 2025
A differentiated β-globin gene replacement strategy uses heterologous introns to restore physiological expressionKirby A Wallace, Trevor L Gerstenberg, Craig L Ennis, et al.Molecular Therapy. Methods & Clinical Development|May 2, 2020
DNA Barcoding in Nonhuman Primates Reveals Important Limitations in Retrovirus Integration Site AnalysisJennifer E Adair, Mark R Enstrom, Kevin G Haworth, et al.Plastic and Reconstructive Surgery|August 28, 2014
Genome editing of mouse fibroblasts by homologous recombination for sustained secretion of PDGF-B and augmentation of wound healingJenny C Barker, Adam D Barker, Jessica Bills, et al.Nature Communications|August 11, 2022
Ultra-deep sequencing validates safety of CRISPR/Cas9 genome editing in human hematopoietic stem and progenitor cellsM Kyle Cromer, Valentin V Barsan, Erich Jaeger, et al.Molecular Therapy : the Journal of the American Society of Gene Therapy|June 13, 2020
Macrophage Subpopulation Dynamics Shift following Intravenous Infusion of Mesenchymal Stromal CellsNina Kosaric, Waracharee Srifa, Clark A Bonham, et al.Nature Biotechnology|December 4, 2020
Generation of human striatal organoids and cortico-striatal assembloids from human pluripotent stem cellsYuki Miura, Min-Yin Li, Fikri Birey, et al.Nature Communications|March 7, 2024
CRISPR/Cas9 model of prostate cancer identifies Kmt2c deficiency as a metastatic driver by Odam/Cabs1 gene cluster expressionHuiqiang Cai, Bin Zhang, Johanne Ahrenfeldt, et al.Nature|April 5, 2005
Highly efficient endogenous human gene correction using designed zinc-finger nucleasesFyodor D Urnov, Jeffrey C Miller, Ya-Li Lee, et al.Nucleic Acids Research|August 20, 2013
Receptor-mediated delivery of engineered nucleases for genome modificationZhong Chen, Lahcen Jaafar, Davies G Agyekum, et al.Pageof 20