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Molecular Therapy. Methods & Clinical Development|March 16, 2023
Enrichment of transgene integrations by transient CRISPR activation of a silent reporter geneNanna S Mikkelsen, Sabina S Hernandez, Trine I Jensen, et al.Current Gene Therapy|October 3, 2014
Gene/cell therapy approaches for Immune Dysregulation Polyendocrinopathy Enteropathy X-linked syndromeLaura Passerini, Francesca R Santoni de Sio, Matthew H Porteus, et al.Nucleic Acids Research|October 26, 2013
Nuclease-mediated gene editing by homologous recombination of the human globin locusRichard A Voit, Ayal Hendel, Shondra M Pruett-Miller, et al.Molecular Therapy : the Journal of the American Society of Gene Therapy|January 30, 2013
Generation of an HIV resistant T-cell line by targeted "stacking" of restriction factorsRichard A Voit, Moira A McMahon, Sara L Sawyer, et al.Molecular Therapy : the Journal of the American Society of Gene Therapy|April 15, 2010
Gene correction by homologous recombination with zinc finger nucleases in primary cells from a mouse model of a generic recessive genetic diseaseJon P Connelly, Jenny C Barker, Shondra Pruett-Miller, et al.Molecular Therapy. Nucleic Acids|February 28, 2013
Efficient sleeping beauty DNA transposition from DNA minicirclesNynne Sharma, Yujia Cai, Rasmus O Bak, et al.Physiological Reports|March 19, 2026
Glycolytic alterations as biomarkers in polycystic kidney disease: A study using a PKD1 knockout model in NRK-52E rat kidney epithelial cellsIda Kjær Mieritz, Christoffer Laustsen, Esben Axelgaard, et al.Science (New York, N.Y.)|March 5, 2016
Activation of proto-oncogenes by disruption of chromosome neighborhoodsDenes Hnisz, Abraham S Weintraub, Daniel S Day, et al.Plos One|September 10, 2015
Use of Genome Engineering to Create Patient Specific MLL Translocations in Primary Human Hematopoietic Stem and Progenitor CellsErin H Breese, Corina Buechele, Catherine Dawson, et al.Molecular Therapy. Methods & Clinical Development|August 28, 2023
Molecular dynamics of genome editing with CRISPR-Cas9 and rAAV6 virus in human HSPCs to treat sickle cell diseaseLiwen Xu, Premanjali Lahiri, Jason Skowronski, et al.Pageof 20