A small molecule that alters glucose metabolism in stressed brain cells may help prevent their death, representing a new strategy for treating brain diseases including amyotrophic lateral sclerosis, Alzheimer’s disease, and Huntington’s disease, among others. The potential therapeutic candidate was described in the journal Scientific Reports…
I mentioned the day that I received the results of my genetic test in my first column. It was a Friday. I had been told to make plans for that day, because not knowing what you’re going to do can contribute to stress. However, I didn’t arrange…
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, “…
Bear with me as I make an analogy. It’ll make sense in a moment. My wife, Jill, now sees genetic testing as the equivalent of a GPS. When you need to find out what illnesses run in your family, all you have to do is turn it on. Once…
As you may have read in a previous column, I decided to get tested for Huntington’s at 22 years old. For me, it was a no-brainer. I wanted to get tested because I can’t handle unknowns. They vex me. I have obsessive-compulsive disorder, so…
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…
Get regular updates to your inbox.
