Margarida Azevedo wites on HuntingtonDiseaseNews.com how Emory University researchers deleted the Huntington’s disease-causing protein, huntingtin, in the brains of adult mice, and observed that such deletion was safe in adults though not in relatively newborn mice. The study, “Ablation of huntingtin in adult neurons is nondeleterious but its depletion in young mice causes acute pancreatitis” and published in PNAS, appears to present a new therapeutic strategy to treating the disease.
Huntington’s disease, a neurodegenerative and debilitating condition, is caused by a known genetic mutation, the CAG repeat (polyglutamine) expansion, in the huntingtin (HTT) gene, leading to the formation of a mutated form of the protein. The loss of the functional huntingtin protein affects axonal transport, essential for synaptic formation, and leads to mitochondrial dysfunction and neuronal damage. Research has focused on a series of approaches, such as RNA interference (RNAi), to inactivate the mutant HTT gene. As such, a clear understanding of the protein’s function is vital.
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