HD Research

Huntington's Disease Research

“A major focus of research on HD is to understand the toxicity of mutant huntingin protein to brain cells and to develop potential drugs for counteracting it. Animal models of the disorder allow scientists to study mechanisms of the disease and to move forward with strategies most likely to work and least likely to cause harm for individuals. The HD gene discovery is allowing scientists to recruit individuals who carry the HD gene into clinical studies early — before they become ill. Researchers hope to understand how the defective gene affects various structures in the brain and the body's chemistry and metabolism. Since some of the clinical symptoms in neurodegenerative diseases may be caused by the ultimate malfunctioning of neuronal circuits rather than by the loss of individual cells, scientists are using cutting-edge methods such as optogenetics (where neurons are activated or silenced in the brains of living animals using light beams) to probe the cause and progression of such circuit defects in HD. Scientists are also using stem cells to study disease mechanisms and test potential therapeutic drugs.” From the
NIH Huntington’s Disease Information Page

Huntington's research areas include:
“New drug discovery may stop Huntington’s disease”

In December 2017 the HD community was thrilled to hear the results of the drug trial for ISIS-HTT. “For the first time we have the potential, we have the hope, of a therapy that one day may slow or prevent Huntington’s disease.” Click here to read more.


Philly Cure HD Supports Research

Philly Cure HD financially supports research through the Davidson Laboratory.

More Online Resources about HD Research
For more information contact
Sherri McElfatrick
215-219-3521
socialworker@phillycurehd.org