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Molecular Therapy. Nucleic Acids|January 6, 2025
Precise template-free correction restores gene function in Tay-Sachs disease while reframing is ineffectiveJoshua E Hung, Reid A Brewer, Lujaina Elbakr, et al.
Genome Research|November 23, 2019
Modeling Niemann-Pick disease type C in a human haploid cell line allows for patient variant characterization and clinical interpretationSteven Erwood, Reid A Brewer, Teija M I Bily, et al.
Molecular Therapy. Methods & Clinical Development|June 4, 2020
Allele-Specific Prevention of Nonsense-Mediated Decay in Cystic Fibrosis Using Homology-Independent Genome EditingSteven Erwood, Onofrio Laselva, Teija M I Bily, et al.
Nature Biotechnology|February 22, 2022
Saturation variant interpretation using CRISPR prime editingSteven Erwood, Teija M I Bily, Jason Lequyer, et al.
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