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See below for a daily digest of the latest Huntington's Disease news!

How Mutant Protein Drives Cellular Toxicity in Huntington’s Revealed in Study

Aug 17, 2017 12:02 pm | Alice Melão

 

mutant huntingtin proteinAggregates of mutant huntingtin protein have been associated with cellular toxicity in Huntington’s disease. Now researchers have discovered that isolated forms of mutated huntingtin are actually responsible for major molecular changes in cells that will drive toxicity. The study, “Transcriptional profiles for distinct aggregation states of mutant Huntingtin exon 1 protein unmask new Huntington’s disease pathways,” was […]

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UniQure Plans Tests That Could Lead to Clinical Trial of AMT-130 Gene Therapy for Huntington’s

Aug 15, 2017 06:28 am | Carolina Henriques

 

gene therapy AMT-130Dutch pharmaceutical company uniQure says it plans to conduct preclinical studies leading to clinical testing of a gene therapy in patients with Huntington’s disease. The company is developing AMT-130, an experimental therapy that aims to block production of the mutated and aggregation-prone huntingtin protein in patients. Composed of a small portion of man-made genetic material called microRNA (miRNA), […]

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Recent Posts

Using Gene Editing to Suppress Activity of Protein Mutations Could Be Way to Treat Huntington’s, Study Reports
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New Research Data Network Aims to Advance Discovery of Huntington’s Treatment
Wave Life Sciences to Start Trials of Two Therapies That Target Huntington’s Mutations

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