Huntington’s disease, an inherited condition that is invariably fatal, affects muscle coordination and causes movement, cognitive and behavioral problems. Currently, there is no effective cure or treatment to stop the progression of the disease.
Ghazaleh Sadri-Vakili, PhD, began studying Huntington’s disease when she joined Mass General as a post-doctoral fellow in 2003. Her laboratory aims to identify the underlying molecular mechanisms of neurodegenerative disorders and discover novel therapies for their treatment.
Sparing Entire Families
To translate her lab’s discoveries into new treatments, Dr. Sadri-Vakili is collaborating with other scientists and companies. A previous collaboration resulted in a library of over 50 compounds that her lab then screened in Huntington’s disease cell lines. “From there, we chose the most promising candidates, which have now been tested in fly and mouse models of Huntington’s disease,” she says.
Dr. Sadri-Vakili is optimistic that one or two of the more effective drugs will be used in clinical trials within the next few years.
Dr. Sadri-Vakili is optimistic that one or two of the more effective drugs will be used in clinical trials within the next few years. More recently, her laboratory is assessing a novel class of compounds known as microneurotrophins in cellular and animal models of Huntington’s disease and amyotrophic lateral sclerosis, or ALS. If she and her collaborators succeed, entire families may be spared the devastating effects of such neurodegenerative diseases.
Being a research scientist takes intellectual curiosity, discipline and patience — three characteristics that Dr. Sadri-Vakili has in abundance.
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