Research collaborators and institutions
Dr. Michael Wangler
Baylor College of Medicine
A metabolomics resource for peroxisomal disorders and related conditions such as D-bifunctional protein deficiency and Rhizomelic chondrodysplasia punctata (RCDP) is being created.
The National Center for Advancing Translational Sciences (NCATS) at the National Institutes of Health (NIH) has hired Di Wu, a postdoctoral fellow to work on drug screening projects. GFPD is one of only 10 patient advocacy groups/nonprofit public charities in the world to be accepted into this program at NCATS
Dr. Joseph Hacia
Los Angeles, California
University of Southern California
Dr. Hacia has spearheaded a multi-year project developing research resources for neurodegeneration and vision loss in peroxisomal disorders. Currently, a rat with a central nervous system defect is being developed with Dr. Hacia’s collaborator, Dr. Quilong Ying. Development of this rat will allow treatments for the brain to be tested in an animal model prior to moving to human clinical testing.
Bar Harbor, Maine
A mouse model will be used to provide proof of concept for gene therapy for the central nervous system.
Dr. Nancy Braverman
Montreal, Quebec, Canada
A PEX 1 mouse model is being used to (1) optimize our retinal gene therapy projects and (2) test candidate drugs for potential therapies at McGill University in Montreal, Quebec, Canada.