Article

Gene replacement therapy for Piga GPI-anchor deficiency in the developing nervous system

2025-12-13

Abstract excerpt

Glycosylphosphatidylinositol (GPI) anchors are a class of post-translational modifications observed on over 150 proteins. Pathogenic variants in the GPI biosynthesis enzyme, PIGA , in humans are associated with several brain anomalies such as hypomyelination, cerebellar hypoplasia, ataxic gait, and can lead to premature mortality. We previously genetically deleted Piga from the embryonic mouse brain which led to...

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Literature Corpus work: 81317727-863f-516c-8ffa-031a9bb1810a
DOI: 10.64898/2025.12.11.693709

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