Development of treatments for rare genetic diseases, Mucopolysaccharidoses (MPS)
Research DescriptionMPS research lab focuses on finding treatments for the brain in the mucopolysaccharidoses. The mucopolysaccharidoses (MPS) are a group of rare genetic diseases. Current projects include intrathecal enzyme replacement therapy for mucopolysaccharidosis type I, immune tolerance to enzyme replacement, and developing enzyme replacement therapy for mucopolysaccharidosis type III.
Dr. Patricia Dickson performs clinical trials and laboratory research on enzyme replacement therapy for the brain in the mucopolysaccharidoses (MPS), a group of rare genetic pediatric neurodegenerative diseases that are as yet untreatable. Her current projects include clinical trials of intrathecal enzyme replacement therapy for MPS type I, immune tolerance to enzyme replacement therapy, and developing enzyme replacement therapy for MPS type III.
Dr. Dickson serves on the scientific advisory board of the National MPS Society, is a member of the Committee on Maternal, Child and Adolescent Health at the UCLA Clinical and Translational Science Institute (CTSI), and is a member of the Therapeutics Committee of the American College of Medical Genetics. She supports LA BioMed as a participant on the Vision Committee and in the Website Development Planning Group.
- AB, 1995, University of Chicago, Chicago, Illinois
- MD, 1999, Columbia University, New York
Recent and/or Significant Publications
- Dickson PI, Ellinwood NM, Brown JR, Witt RG, Le SQ, Passage MB, Vera MU, Crawford BE. Specific antibody titer alters the effectiveness of intrathecal enzyme replacement therapy in canine mucopolysaccharidosis I. Mol Genet Metab. 2012 May;106(1):68-72. Epub 2012 Feb 8.
- Tippin BL, Troitskaya L, Kan SH, Todd AK, Le SQ, Dickson PI. Biochemical characterization of fluorescent-labeled recombinant human alpha-L-iduronidase in vitro. Biotechnol Appl Biochem. 2011 Nov-Dec;58(6):391-6. doi: 10.1002/bab.52. Epub 2011 Oct 28.
- Chen A, Vogler C, McEntee M, Hanson S, Ellinwood NM, Jens J, Snella E, Passage M, Le S, Guerra C, Dickson P. Glycosaminoglycan storage in neuroanatomical regions of mucopolysaccharidosis I dogs following intrathecal recombinant human iduronidase. APMIS. 2011 Aug;119(8):513-21. doi: 10.1111/j.1600-0463.2011.02760.x. Epub 2011 May 14.