Posted on June 14, 2018
HDSA’s 33rd Annual Convention in Los Angeles from June 7th-9th included the 2018 Research Forum, where ten speakers collaborated to tell a story about the HD gene and the continued work towards designing and testing a huntingtin-lowering therapy. Check it out here and stay tuned for a recap of research news at Convention!
CRISPR research techniques require extra caution
An experimental gene-editing technology called CRISPR is taking the heat in the news (and the stock market) this week due to a research paper showing that we need to take seriously the potential concerns about human use. Claims about it causing cancer, however, are overblown. To prevent the formation of tumors, our cells have natural defense mechanisms against DNA damage – and it turns out that our cells can fight off CRISPR, too, with the help of a protein called p53. Recent research shows that when CRISPR does work to edit a cell’s genes, it could mean that p53 has lost its strength, potentially making cells more vulnerable to DNA damage. This suggests that we’ll need to keep a close eye on p53 when experimenting with CRISPR, especially for human therapies. For Huntington’s disease, CRISPR is being explored in cells and in mice as a way to correct or turn off the HD gene – and these new findings won’t halt this work. We already know that CRISPR is still too experimental for human use in HD, but there are groups trying to improve its safety and efficiency moving forward. The recent research simply provides useful information about how to proceed.
Later onset of HD with high blood pressure?
Data from large observational studies like Enroll-HD can be used to answer questions about how additional health factors influence the course of Huntington’s disease. To ask whether a person’s blood pressure can have an effect on when they develop symptoms, a group of researchers analyzed data from 680 participants with HD in the REGISTRY study conducted by the European Huntington’s Disease Network. 86 of these participants had high blood pressure; 544 did not. To the researchers’ surprise, those with high blood pressure showed several years’ delay in symptom onset. This finding is difficult to explain. First of all, those with high blood pressure were on average older and under medical care to begin with, so a person’s age and general comfort with seeking medical care could influence the results. Second, since almost all of those with high blood pressure were on medication to treat it, the results suggest that blood pressure medication could have a protective effect in HD. Such medications can also positively influence symptoms of other neurological diseases, so it may be a useful approach to explore. However, analysis of a larger group of people and a controlled drug study will be required to determine if this is the case.