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Recent advances in molecular therapies for neurological disease: triplet repeat disorders

11 Jun 2019

Abstract excerpt

Triplet repeat diseases (TRDs) are caused by pathogenic expansions of trinucleotide sequence repeats within coding and non-coding regions of different genes. They are typically progressive, very disabling and frequently involve the nervous system. Currently available symptomatic therapies provide modest benefit at best. The development of interventions that interfere with the natural history of these diseases is...

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PMID: 31227833
DOI: 10.1093/hmg/ddz138

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