Dr. Pacak Receives $1.8 Million Grant

Dr. Christina Pacak recently received a $1.8 million grant for the manufacturing of a gene therapy for Cockayne Syndrome. Christina Pacak, PhD, is an assistant professor in the neuromuscular disease division of the Department of Neurology at the University of Minnesota Medical School. 

Cockayne Syndrome is a, “​​rare accelerated aging disorder that has no disease- altering treatments available at this time”. It is characterized by a range of symptoms including, “accelerated aging, short stature, microcephaly, photosensitivity, neurological dysfunction, liver and digestive problems and damaged kidneys”. Dr. Pacak’s interest in this stems from a personal connection with a family friend that had a child with this syndrome. 

This three-year grant, awarded by the Center for Biologics Evaluation and Research of the FDA will seek to optimize gene replacement therapy. The team led by Dr. Pacak additionally aims to understand the underlying process for diseases connected to aging. 

Read the full story on Dr. Pacak’s grant here.

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