Tara Deans wins National Institute of Heath Trailblazer award.

University of Utah bioengineering assistant professor Tara Deans, has received the National Institutes of Health (NIH) Trailblazer Award to research methods for treating a family of metabolic diseases called lysosomal storage diseases (LSDs). Deans’ award, which was titled “Engineering novel delivery systems with synthetic biology,” was given through the NIH’s National Institute of Biomedical Imaging and Bioengineering.

LSDs are inherited-diseases characterized by an abnormal buildup of toxic materials in the body’s cells as a result of lysosomal protein deficiencies. These malfunctioning proteins represent a group of about 50 different genetic diseases such as Tay–Sachs disease, which destroys nerve cells in the brain and spinal cord, and Krabbe disease, which attacks the nervous system and can often lead to death. Diseases from lysosomal deficiencies occur in an estimated one in every 8,000 births.

“Different organs can have cells that lack the appropriate lysosomal enzymes to break down waste molecules within the cells. When the lysosome is missing these enzymes, the cells in that organ die, and the patient eventually undergoes organ failure,” Deans said about these diseases. “It’s like when the garbage truck doesn’t show up, and your garbage piles up. This is what happens within cells that are lacking the proper enzymes within the lysosome — waste builds up and the cells eventually can’t handle all of the trash, so they die.”

With the grant, which is $400,000 over three years, Deans’ research group will look into an effective method for the controlled and sustained delivery of lysosomal proteins at functional levels to the needed organ or organs.