Article

Increased FAN1 expression by mRNA-LNP attenuates CAG repeat expansion in Huntington patients’ iPSC-derived astrocytes

2023-11-24

Abstract excerpt

<h4>ABSTRACT</h4> Expansion of repeat sequences within the human genome can lead to disease pathogenesis, such as Huntington’s Disease, primarily affecting the nervous system. Genome-wide association studies (GWAS) of age-at-onset in Huntington’s disease (HD) patients demonstrated DNA mismatch repair (MMR) genes are modifiers of somatic expansion and may be potential therapeutic targets for repeat expansion (RE)...

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Literature Corpus work: f26d9e2c-6bd3-5104-ad86-c17b65a7bccd
DOI: 10.1101/2023.11.24.568451

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