The prestigious Science magazine published an article on Huntington’s disease this week, describing the huntingtin-lowering ASO drug developed by Ionis, and profiling the very first trial participant. The article covered her family history with HD and her current experience in the open-label trial, and featured discussions with her physician Dr. Blair Leavitt, a researcher at the University of British Columbia and an HDSA Scientific Advisory Board member.
HD Featured in the Guardian’s Science Weekly Podcast
The Guardian’s Science Weekly podcast features stories about discoveries and debates in biology, chemistry, and physics. This week they featured a story about HD and the science behind it, with interviews from Dr. Ed Wild of HDBuzz and HDSA Human Biology Fellow Dr. Amber Southwell. Especially interesting is evidence that longer CAG repeat tracts could have benefits for health, intelligence, and evolution. From the Guardian:
“Hannah Devlin investigates the link between Huntington’s and its genetic history by talking to Dr. Amber Southwell, from the University of Central Florida, about the evolution of the gene. She also speaks with Dr. Ed Wild, from UCL, about Huntington’s and whether a new drug could slow its progression. And she hears from Roger, who was diagnosed with Huntington’s four years ago, and his wife, Brenda.”
Final Chance to Participate in CRISPR Survey
This week is your last chance to participate in a university-approved survey about CRISPR available on HDSA’s research survey page. The survey will close at midnight on Friday, August 31st. CRISPR is a technique for editing genes that has been extremely useful in laboratory settings to help us understand more about the function of particular genes, including the HD gene.
One long-term goal of CRISPR is to use it therapeutically, but it’s currently limited by huge safety concerns – DNA editing gone wrong could have disastrous medical consequences. Nevertheless, its great potential has raised some interesting ethical questions that are the focus of a graduate research project by Dylan Platt at Augustana University. He is asking families affected by HD and other genetic disorders about their opinions around CRISPR technology as a possible future therapy. Participate in the survey to add your voice to the conversation about CRISPR and HD.
We also encourage you to learn more about this technique through some great HDBuzz articles about genome editing and CRISPR in HD mice.
