Frequent loss of heterozygosity in CRISPR-Cas9-edited early human embryos.

ABSTRACT: CRISPR-Cas9 genome editing is a promising technique for clinical applications, such as the correction of disease-associated alleles in somatic cells. The use of this approach has also been discussed in the context of heritable editing of the human germ line. However, studies assessing gene correction in early human embryos report low efficiency of mutation repair, high rates of mosaicism, and the possibility of unintended editing outcomes that may have pathologic consequences. We developed computational pipelines to assess single-cell genomics and transcriptomics datasets from OCT4 (POU5F1) CRISPR-Cas9-targeted and control human preimplantation embryos. This allowed us to evaluate on-target mutations that would be missed by more conventional genotyping techniques. We observed loss o

SUBMITTER: Alanis-Lobato G 

PROVIDER: S-EPMC8179174 | biostudies-literature | 2021 Jun

REPOSITORIES: biostudies-literature