A Yale chemistry professor is closing in on a medical breakthrough that could help cure a rare genetic disorder as well as pioneer a novel way of treating disease.
Yale University Sterling professor Alanna Schepartz is working to send protein-based therapies into hard-to-reach areas of the cell. It’s an incredibly difficult task, one that has stumped the numerous scientists who’ve tried to apply it to treatments for cancer, HIV and other diseases. But this summer, with a new Yale grant in hand, Schepartz is beginning preclinical trials that could prove she’s done it.
If her treatment is successful, it would be a first for the long-studied drug-delivery method —based on what are called cell-penetrating miniature proteins — and a potential model for other therapies. Other researchers have sought to treat cancer, cardiac diseases, pain, and strokes with the method.
“Frankly, that’s pretty remarkable,” said Jon Soderstrom, director of Yale’s Office of Cooperative Research. It could be, he said, “like another arrow in the quiver for potentially developing new therapies.”
Beyond the vital medical research, Schepartz’s progress toward animal trials also illustrates how major universities are aggressively seeking markets for their faculty’s work.
Schepartz will soon begin mouse trials on a potential cure for Type 1 Citrullinemia, a rare genetic disorder in which ammonia and other toxins build up in the blood, causing lethargy, seizures, loss of consciousness and vomiting. It affects 1 in 56,000 people worldwide, and often presents itself in newborns.
click here to read more.