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Delivering a disease-modifying treatment for Huntington's disease.

Drug discovery today - 1 Jan 2015

Godinho Bruno M D C, Malhotra Meenakshi, O'Driscoll Caitriona M, Cryan John F

Abstract excerpt

Huntington's disease (HD) is an incurable genetic neurodegenerative disorder that leads to motor and cognitive decline. It is caused by an expanded polyglutamine tract within the Huntingtin (HTT) gene, which translates into a toxic mutant HTT (muHTT) protein. Although no cure has yet been discovered, novel therapeutic strategies, such as RNA interference (RNAi), antisense oligonucleotides (ASOs), ribozymes, DNA...

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Alleles
HTT gene
Gene Silencing
Genome, Human
Huntingtin Protein
Huntington Disease

Methodology Data sources Editorial policy Privacy About Contact Helena Bioinformatics

Nerve Tissue Proteins

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PMID: 25256777
DOI: 10.1016/j.drudis.2014.09.011

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HTT

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PubMed metadata and Literature Corpus relationships. Corpus snapshot updated 31 Aug 2026.

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