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A new gene editing method allows researchers to target many different kinds of disease-causing mutations in multiple cell types, which could have implications for genetic disorders such as Huntington’s disease, a study suggests. The new method was described in the journal Cell Research in a study, titled “…

People at a pre-symptomatic, or premanifest, stage of Huntington’s disease (HD) have subtle speech alterations that may be used as a marker to evaluate new therapies intended to stop or slow disease progression before it becomes too debilitating. The study with that recent research, “…

Rare diseases deeply affect not only the children who experience them, but also their healthy brothers and sisters, as their parents can attest.    Two entries in November’s “Disorder: The Rare Disease Film Festival” will focus on what siblings go through, according to the San Francisco festival’s co-founder,…

Being less affected by loss is predictive of apathy in people with Huntington’s disease (HD), a new study has found. The study, “Insensitivity to Loss Predicts Apathy in Huntington’s Disease,” was published in the journal Movement Disorders. Apathy — the feeling of indifference, where a person just doesn’t care —…

Developing gene therapies for rare diseases is one thing. Creating gene-edited “designer babies” is quite another. German legal expert Timo Minssen outlined the potentially explosive ethical landmines surrounding such issues during a recent talk at the New York Genome Center. Minssen directs the Center for Advanced Studies in…

Tiny fatty (lipid) particles can be used to enhance the delivery of gene editing tools, such as CRISPR-Cas9, to targeted cells and considerably improve their ability to possibly treat human disorders like Huntington’s disease, researchers reported. Their study, “Fast and Efficient CRISPR/Cas9 Genome Editing In Vivo Enabled by…

Psychiatric and cognitive symptoms in Huntington’s disease share genetic traits with psychiatric disorders and intelligence, respectively, according to a study. These findings suggest that current treatments used for depression and psychotic symptoms in the overall population may also be able to treat these symptoms in Huntington’s patients. The…

Imagine living your whole life with a painful disease so rare that only 25 others worldwide have what you have. And that you’re one of just six such people who’ve made it to adulthood. Neena Nizar doesn’t have to imagine. The 41-year-old English professor at Metro Community College in Elkhorn,…

Patients at a pre-symptomatic stage of Huntington’s disease have marked difficulties recognizing and understanding negative and neutral mental states of other individuals, regardless of gender, a study has found. The results, “Effects of Stimulus-Related Variables on Mental States Recognition in Huntington’s Disease,” were published in the…

Eligible Huntington’s disease patients would likely participate in a gene therapy trial regardless of the study design, but they are more inclined toward those with less invasive interventions and without a placebo group, a questionnaire-based study suggests. The results may be useful to design more patient-centered clinical trials assessing…