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Article

Recapture Lysosomal Enzyme Deficiency via Targeted Gene Disruption in the Human Near-Haploid Cell Line HAP1.

Genes - 15 Jul 2021

Brown Annie, Zhang Jiayi, Lawler Brendan, Lu Biao

Abstract excerpt

BACKGROUND: Advancement in genome engineering enables rapid and targeted disruption of any coding sequences to study gene functions or establish human disease models. We explored whether this approach can be used to study Gaucher disease, one of the most common types of lysosomal storage diseases (LSDs) in a near-haploid human cell line (HAP1). RESULTS: CRISPR-Cas9 targeting to coding sequences of...

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Identifiers

PMID: 34356092
DOI: 10.3390/genes12071076
PMCID: PMC8308024

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PubMed metadata and Literature Corpus relationships. Corpus snapshot updated 7 Sept 2026. Manifest publication-graph-cohort-2026-09-07.1.

Source receipt: pubmed, retrieved 7 Sept 2026.

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