Article
Retained chromosomal integrity following CRISPR-Cas9-based mutational correction in human embryos.
Molecular therapy : the journal of the American Society of Gene Therapy - 2 Aug 2023
Bekaert Bieke, Boel Annekatrien, De Witte Lisa, Vandenberghe Winter, Popovic Mina, Stamatiadis Panagiotis, Cosemans Gwenny, Tordeurs Lise, De Loore Athina-Maria, Chuva de Sousa Lopes Susana Marina, De Sutter Petra, Stoop Dominic, Coucke Paul, Menten Björn, Heindryckx Björn
Abstract excerpt
Human germline gene correction by targeted nucleases holds great promise for reducing mutation transmission. However, recent studies have reported concerning observations in CRISPR-Cas9-targeted human embryos, including mosaicism and loss of heterozygosity (LOH). The latter has been associated with either gene conversion or (partial) chromosome loss events. In this study, we aimed to correct a heterozygous...
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