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Therapeutic strategies for Huntington's disease

30 Jun 2020

Abstract excerpt

PURPOSE OF REVIEW: Huntington's disease is a fatal autosomal dominant neurodegenerative disorder caused by a trinucleotide expansion in the HTT gene, and current therapies focus on symptomatic treatment. This review explores therapeutic approaches that directly target the pathogenic mutation, disrupt HTT mRNA or its translation. RECENT FINDINGS: Zinc-finger transcription repressors and CRISPR-Cas9 therapies...

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PMID: 32657893
DOI: 10.1097/wco.0000000000000835

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