Magdalena Kegel,  —

A virus that delivers UniQure‘s gene therapy candidate AMT-130 has done a good job of getting the Huntington’s disease treatment into the brains of primates, company research shows. Injections of the virus delivered the treatment with precision and without side effects, researchers said. AMT-130’s delivery component is an AAV5 viral vector. AAV5…

Although symptoms of Huntington’s disease don’t appear until later in life, new research suggests that the genetic flaw may alter neurons during early development — findings that may change ideas on how to treat the illness. For the study, “Developmental alterations in Huntington’s disease neural cells and pharmacological…

Changes in brain volume are linked to both movement problems and cognitive decline in people with huntingtin mutations who have not yet received a Huntington’s disease diagnosis, according to a new report. The study not only offers insights into how the neurodegeneration in Huntington’s affects the emergence of various symptoms,…

Caffeine boosts a neuroprotective protein in the brain that is lost in neurodegenerative conditions such as Huntington’s disease, according to a study. Researchers identified 23 other compounds that also act on the protein, known as NMNAT2. Since increasing the protein may help patients with Huntington’s or similar conditions, the researchers…

Mitochondrial abnormalities may not contribute to the degeneration of neurons in the striatum in patients with Huntington’s disease, according to a study that provides evidence contradicting several earlier findings. The study explored mitochondrial processes in a mouse model of Huntington’s disease and found that mitochondria from the striatum — the…

Two large surveys that explore the views of adult and juvenile Huntington’s disease patients and their caregivers on symptoms and treatments may help direct research efforts toward issues that matter most to those affected. Interestingly, the symptom-focused survey revealed large differences in how patients and caregivers perceive the frequency and…

Researchers may finally have tracked down an explanation for expansions of three-letter DNA sequences that disrupt genes, and lead to Huntington’s disease and numerous other neuromuscular and neurodegenerative conditions. The study, “The role of break-induced replication in large-scale expansions of (CAG)n/(CTG)n repeats,” published in the journal Nature Structural & Molecular…

A study using a mouse model suggests that some symptoms of Huntington’s disease are caused by muscle abnormalities, and are not related to dying neurons. The findings may present new opportunities for improving patient care by using muscle-specific drugs. The study, “Progressive Cl− channel defects reveal disrupted…

A drug in clinical development by Takeda for schizophrenia, called TAK-063, was seen to reduce neuron death and improve symptoms in mice with Huntington’s disease. But the data needs to be confirmed in other animal models of Huntington’s, and it is too early to say if the drug would also benefit…

By using computational modeling, researchers have demonstrated that at the early stages of mutant huntingtin (mHtt) aggregation, a molecular feedback loop works to prevent the effects of the toxic aggregates, until a tipping point is reached. Because the study, “Systemic study of a natural feedback loop in…