A Genetic Lottery - a Column by Becky Field

The CHDI Foundation, a privately-funded nonprofit research organization dedicated to fighting Huntington’s disease, has awarded a research grant to a team of scientists from the University of Rochester Medical Center (URMC) to study the role of specific cells in the development of the disease. Dr. Steve Goldman, MD, PhD, co-director…

Abnormal energy production may contribute to the progression of Huntington’s disease, according to a new study. The study, entitled “The Phasor-FLIM Fingerprints Reveal Shifts From OXPHOS To Enhanced Glycolysis In Huntington Disease,” was recently published in the journal Scientific Reports. The mechanisms underlying neurodegeneration in Huntington’s disease remain…

Progressive neurological diseases, such as Huntington’s disease, are associated with pathological changes in the retina and the brain. According to Henri Leinonen from the University of Eastern Finland (UEF) and colleagues, such disorders might be detected earlier by using simple and non-invasive eye examination techniques. In the study “Early…

A survey of 1,700 Huntington’s disease patients that questioned issues such as symptoms, treatment, and disease management, revealed some surprising facts that can impact how physicians and researchers view and treat the condition. Particularly, the survey revealed that patients often lack support during the diagnostic process. GeneFo is an…

A recent review summarizes the scientific literature on behavioral and psychiatric symptoms of Huntington’s disease, noting that plenty of research is still needed to better understand and treat such symptoms, which traditionally have taken second stage to the disease’s movement problems. The review, “Changes in mental state…

Researchers have developed a new gene-editing tool that may improve the precision of future gene therapy efforts in Huntington’s disease, as well as numerous other genetic conditions. The study, “A chemical-inducible CRISPR–Cas9 system for rapid control of genome editing,” published in the journal Nature Chemical Biology,…

Teva Pharmaceutical presented encouraging top-line data from PRIDE-HD, a Phase 2 clinical trial evaluating the impact of pridopidine, an oral medication, on motor impairment in patients with Huntington’s disease (HD). Teva is now preparing for a larger clinical study in Huntington’s. The presentation took place at the 9th European…

Mice models of Huntington’s disease, infected with a parasite commonly found in humans, react in a different way to the infection than normal mice, researchers have revealed. The study suggests that infection and immune activation can impact the course of the neurodegenerative disease. Although researchers behind the study declined to speculate…

Attempts to block the activity of the mutant huntingtin gene, which causes Huntington’s disease, may have taken a big step forward. Scientists report they successfully silenced the gene for six months after a single injection in a mouse model of the disease. The study, “Deimmunization for gene therapy:…

Voyager Therapeutics has partnered with the CHDI Foundation to advance its VY-HTT01 gene-silencing therapy for Huntington’s disease (HD). The research alliance with CHDI builds upon an earlier partnership between CHDI and Sanofi Genzyme and includes financial support from CHDI to help in the preparation and filing of an FDA…