Researchers at UCLA have developed a new mouse model for HD that more closely resembles human HD than ever before. In humans, HD is caused by a region in the genetic code for huntingtin protein that codes for too many C-A-G’s. This region expands over the course of the disease in a way that is toxic for the body, especially the brain, but scientists have not yet figured out how this causes HD. This new mouse model, called BAC-CAG, mimics CAG-repeat expansion in humans more closely than other mouse models of the disease have before. Scientists believe this model of the disease could help us better understand how instability in the CAG repeat region of the huntingtin gene sets the course of HD in humans. Learn more from HDBuzz here.
uniQure doses first European participants in open-label study of AMT-130
Earlier this week, uniQure shared news that the first European patients were dosed in their open-label study of AMT-130. This huntingtin-lowering gene therapy is delivered directly to deep brain tissues in a single dose administered with an MRI-guided brain surgery. Safety data from the first group of 10 participants in the USA is expected in the second quarter of 2022. Read the full press release here.
HDSA Announces 55 Centers of Excellence for 2022
HDSA Centers of Excellence provide world-class multidisciplinary care to families all over the United States. On February 9th, HDSA announced the expansion of the Centers of Excellence Program to include 55 sites in 35 states plus the District of Columbia. Read more about our COEs and all locations in this week’s press release.
Professionals at these locations include neurologists, psychiatrists, social workers, therapists, and counselors with extensive HD experience who work together to provide care and support for HD families. Because HDSA is highly committed to supporting clinical research, HDSA Centers of Excellence must also demonstrate their ability to provide clinical research opportunities. We’re excited to continue the expansion of expert HD research and care nationwide.