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Stem Cell Research|December 2, 2025
Generation of WTD, a control human iPSC line for genetic researchBria L Macklin, Wendy V Runyon, Carissa M Feliciano, et al.Medrxiv : the Preprint Server for Health Sciences|April 3, 2026
Leveraging human genetic variation to therapeutically target hundreds of genes with dominant & dispensable disease allelesGrace D Ramey, Quinn T Cowan, Akshita G Saxena, et al.Nature Biomedical Engineering|November 3, 2025
A microphysiological system for screening lipid nanoparticle-mRNA complexes predicts in vivo heart transfection efficacyGabriel Neiman, Mauro W Costa, Hesong Han, et al.Biorxiv : the Preprint Server for Biology|January 7, 2025
Haplotype editing with CRISPR/Cas9 as a therapeutic approach for dominant-negative missense mutations in <i>NEFL</i>Poorvi H Dua, Bazilco M J Simon, Chiara B E Marley, et al.Frontiers in Cell and Developmental Biology|September 6, 2021
Allele-Specific Gene Editing Rescues Pathology in a Human Model of Charcot-Marie-Tooth Disease Type 2ECarissa M Feliciano, Kenneth Wu, Hannah L Watry, et al.Science (New York, N.Y.)|April 20, 2019
Unbiased detection of CRISPR off-targets in vivo using DISCOVER-SeqBeeke Wienert, Stacia K Wyman, Christopher D Richardson, et al.Molecular Therapy : the Journal of the American Society of Gene Therapy|November 21, 2025
Haplotype editing with CRISPR-Cas9 as a therapeutic approach for dominant-negative missense mutations in NEFLPoorvi H Dua, Bazilco M J Simon, Chiara B E Marley, et al.JCI Insight|July 21, 2017
A BAG3 chaperone complex maintains cardiomyocyte function during proteotoxic stressLuke M Judge, Juan A Perez-Bermejo, Annie Truong, et al.Tissue Engineering. Part C, Methods|October 22, 2014
Automated Video-Based Analysis of Contractility and Calcium Flux in Human-Induced Pluripotent Stem Cell-Derived Cardiomyocytes Cultured over Different Spatial ScalesNathaniel Huebsch, Peter Loskill, Mohammad A Mandegar, et al.Biomaterials|August 17, 2021
Bioengineered optogenetic model of human neuromuscular junctionOlaia F Vila, Miguel Chavez, Stephen P Ma, et al.Pageof 3