Seeking Brain Biomarkers for Preventive Therapy in Huntington Disease
نویسندگان
چکیده
منابع مشابه
Brain networks in Huntington disease.
Recent studies have focused on understanding the neural mechanisms underlying the emergence of clinical signs and symptoms in early stage Huntington disease (HD). Although cell-based assays have focused on cell autonomous effects of mutant huntingtin, animal HD models have revealed alterations in the function of neuronal networks, particularly those linking the cerebral cortex and striatum. The...
متن کاملProgress and challenges in RNA interference therapy for Huntington disease.
Huntington disease is an incurable, dominant neurodegenerative disorder caused by polyglutamine repeat expansion in the huntingtin protein. Reducing mutant huntingtin expression may offer a treatment for Huntington disease. RNA interference has emerged as a powerful method to silence dominant disease genes. As such, it is being developed as a prospective Huntington disease therapy. Here I discu...
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BACKGROUND AND PURPOSE The clinical symptoms of Huntington disease (HD) include progressive movement disorders, cognitive deficits, and behavioral changes, all of which affect an individual's ability to participate in activities of daily living. To date, very few quantitative or qualitative studies have been conducted to guide physical therapists working with people with HD. The objective of th...
متن کاملMicroRNAs in CSF as prodromal biomarkers for Huntington disease in the PREDICT-HD study
OBJECTIVE To investigate the feasibility of microRNA (miRNA) levels in CSF as biomarkers for prodromal Huntington disease (HD). METHODS miRNA levels were measured in CSF from 60 PREDICT-HD study participants using the HTG protocol. Using a CAG-Age Product score, 30 prodromal HD participants were selected based on estimated probability of imminent clinical diagnosis of HD (i.e., low, medium, h...
متن کاملGene therapy in mouse models of huntington disease.
Huntingtin, the protein that when mutated causes Huntington disease (HD), has many known interactors and participates in diverse cellular functions. Mutant Htt (mHtt) engages in a variety of aberrant interactions that lead to pathological gain of toxic functions as well as loss of normal functions. The broad symptomatology of HD, including diminished voluntary motor control, cognitive decline, ...
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ژورنال
عنوان ژورنال: CNS Neuroscience & Therapeutics
سال: 2010
ISSN: 1755-5930
DOI: 10.1111/j.1755-5949.2010.00157.x