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Huntington disease: update on ongoing therapeutic developments and a look toward the future
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Resumo(s)
Huntington’s disease (HD) is a genetic condition inherited in an autosomal dominant pattern characterized by neurodegeneration. The root cause of HD is a CAG tract expansion occurring in exon 1 of the huntingtin (HTT) gene. This mutation sets in motion a series of detrimental events that progressively lead to neuronal death and structural changes, mainly within the central nervous system but also affecting other organs. Currently, no disease-modifying treatments for HD exist, but an active research effort focuses on potential therapies, with
several programs in various stages of development. This review provides an overview of the HD targets that have genetic and mechanistic validation and the clinical programs that are pursuing such targets at the time of writing. Programs that are completed or terminated will not be extensively covered. Other therapeutic approaches under development target downstream processes and have potential applicability across various neurodegenerative diseases; a detailed discussion of these therapies is beyond the scope of this review. Cell-based therapies will not be reviewed due to the specificity of the topic. However, there are active programs in the preclinical stage.
Descrição
© 2024 Published by Elsevier Ltd.
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Contexto Educativo
Citação
Parkinsonism Relat Disord. 2024 May:122:106049
Editora
Elsevier