A Genetic Lottery - a Column by Becky Field

A sluggish metabolism in the cells of the striatum, a brain region essential for motor function control, is associated with disease severity in Huntington’s patients, while a higher metabolism in other brain regions seems to be associated with more severe motor symptoms in patients, according to researchers. These findings…

Mutations of the MSH3 gene may be associated with the progression of Huntington’s disease, according to a British study. The gene could become a biomarker for the disease, and scientists may be able to develop Huntington’s therapies around it, the researchers from University College London and Wales’ Cardiff University said.

Mitoconix Bio has secured $20 million in financing to continue developing MTC-1203 as a treatment for mitochondrial impairment in Huntington’s and Parkinson’s diseases. Several pharmaceutical companies and medical-related venture-capital funds participated in the financing. They include Aril Bioscience, OrbiMed, Remiges Ventures and the Dementia Discovery Fund. “We are very…

Ionis Pharmaceuticals has completed enrollment of the Phase 1/2a study evaluating the safety, tolerability, properties and movement within the body of Ionis-HTTRx in patients with Huntington’s disease (HD). Patients who complete the randomized, placebo-controlled, dose escalation study (NCT02519036) will be eligible to enroll in an open-label…

Researchers have identified a variant in the gene MSH3 that is linked with Huntington’s disease (HD) progression. The study, “Identification of genetic variants associated with Huntington’s disease progression: a genome-wide association study,” was published in the journal The Lancet Neurology. TRACK-HD was a prospective and observational study which…

Inhibiting the activity of an enzyme preserved the movement capabilities of mice with Huntington’s, slowed the progression of the disease and extended the animals’ lives, according to a study. The dSETDB1/ESET enzyme can disrupt a mechanism that controls normal gene expression, the researchers discovered. Alterations in that expression, along with…

Low levels of a protein called Pyk2 may be associated with the development of Huntington’s disease, according to a new study. Researchers found that normalizing its levels in the hippocampus, the brain region responsible for learning and memory, rescues memory deficits. These findings were published in a study titled, “…

A factor called neurofilament light (NfL) may act as a biomarker of progression in Huntington’s disease, as blood levels of the molecule increase with more severe neurodegeneration. However, the factor also might offer a better means to predict disease onset in people carrying huntingtin mutations, as demonstrated in the study,…

A mutated form of the huntingtin (mHTT) protein has been acknowledged as the cause of Huntington’s disease, but researchers have not been fully clear about how it disrupts the function of brain cells. A study, “Mutant Huntingtin Disrupts the Nuclear Pore Complex,” that was published in the scientific journal…

Voyager Therapeutics has a new candidate for the treatment of Huntington’s disease. The selected gene therapy is called VY-HTT01 and aims to prevent the production of protein from the mutant huntingtin gene (HTT). The compound is now undergoing preclinical studies of drug characteristics and toxicology as the company plans to file…