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Article

Phosphorodiamidate morpholino oligomers suppress mutant huntingtin expression and attenuate neurotoxicity.

Human molecular genetics - 1 Dec 2014

Sun Xin, Marque Leonard O, Cordner Zachary, Pruitt Jennifer L, Bhat Manik, Li Pan P, Kannan Geetha, Ladenheim Ellen E, Moran Timothy H, Margolis Russell L, Rudnicki Dobrila D

Abstract excerpt

Huntington's disease (HD) is a neurodegenerative disorder caused by a CAG trinucleotide repeat expansion in the huntingtin (HTT) gene. Disease pathogenesis derives, at least in part, from the long polyglutamine tract encoded by mutant HTT. Therefore, considerable effort has been dedicated to the development of therapeutic strategies that significantly reduce the expression of the mutant HTT protein. Antisense...

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Identifiers

PMID: 25035419
DOI: 10.1093/hmg/ddu349
PMCID: PMC4222366

Genes

HTT

Provenance

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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