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

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 mo...

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Dettagli Bibliografici
Pubblicato in:Hum Mol Genet
Autore principale: Gonzalez-Alegre, Pedro
Natura: Artigo
Lingua:Inglês
Pubblicazione: Oxford University Press 2019
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Accesso online:https://ncbi.nlm.nih.gov/pmc/articles/PMC6796999/
https://ncbi.nlm.nih.gov/pubmed/31227833
https://ncbi.nlm.nih.govhttp://dx.doi.org/10.1093/hmg/ddz138
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