Voice Your Opinions About The Future Of CRISPR

Posted on August 2, 2018

Community research surveys are valuable ways for researchers to get input from HD patients, families and caregivers about a variety of topics, and can help guide strategies for HD research and care. HDSA works with scientists and students to make their university-approved research surveys available for easy online participation through our website. This week, there’s a new survey about CRISPR available on HDSA’s research survey page. 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.

Update on the LEGATO-HD study

LEGATO-HD is a clinical trial of a drug called laquinimod, made by Active Biotech.  Teva Pharmaeuticals has been conducting a Phase II study to see whether laquinimod could improve symptoms in people with HD. For 12 months, 352 participants took low, medium, or high doses of the drug, or a placebo control. On Tuesday, July 31st, Active Biotech announced that, unfortunately, the study did not meet its primary endpoint of improving Total Motor Score. This means that after a year of treatment, laquinimod was safe, but did not help significantly with people’s movement symptoms. However, the drug did prevent some brain shrinkage, as measured with imaging tests like MRI. Check out the HDBuzz coverage of this story. Additional analyses still need to be completed, and HDSA will provide more information about the future of the trial as details arise.

Application process for HDSA Center of Excellence status

HDSA Centers of Excellence undergo a thorough application and review process to gain and to maintain their designation as high-quality multidisciplinary HD care centers. Research is a central component in the evaluation process; Centers are evaluated not only for their ability to provide care, but for their ability to conduct meaningful HD clinical research and to provide referrals to research studies. Completing the 2019 Letter of Intent form, available this week, is the first step towards applying for HDSA Center of Excellence status.

Posted in Our opinion, Weekly News
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