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Scientists at Boston Children’s Hospital have revealed the cellular and biological mechanisms involved in synapse loss in Alzheimer’s disease. By blocking certain agents involved in this process, scientists could halt synaptic loss, a discovery with therapeutic potential not only for Alzheimer’s, but also other diseases with loss of neuronal connections, including Huntington’s disease. The…

uniQure recently published results from preclinical studies of its gene therapy program, AMT-130, indicating that a one-time administration of AAV5-delivered therapy into the central nervous system can block the mutant HTT gene that causes Huntington’s disease (HD). These findings, in the article “Design, Characterization, and Lead Selection of Therapeutic miRNAs…

The English Parliament recently listened to a young scientist, Emma Yhnell, 25, explain her research into the potential of computer games to “train the brain” of Huntington’s disease (HD) patients. The research associate from Cardiff University in Wales attended Parliament on March 7 to present her novel approach to a range of politicians and…

Researchers at Philadelphia’s Thomas Jefferson University investigated the physiological and pathological role of MICU1 (Mitochondrial Calcium Uptake 1), a protein that controls calcium by the mitochondria, a process involved in cell survival. Results suggest that strengthening this protein’s function and inhibiting mitochondrial calcium uptake could be a novel therapeutic approach…

New Huntington’s research sheds light on the link between the effects of the disease’s underlying mutation on brain structure and function, and consequent behavior anomalies. Studying the gene mutation’s expression in a precise brain area of songbirds, scientists observed how the mutation affects specific neurons and changes behavior, findings that could lead to the…