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CTSI Directory

Patricia Dickson, M.D. [ Edit Your Profile ]

Email Address: pdickson@ucla.edu
Work Email Address: pdickson@ucla.edu

Fax Number: (310) 328-9921
(310) 782-2999
Work Phone Number: (310) 222-3757

Work Address:
1124 West Carson Street
Torrance, CA

LABioMed Associate Professor In-Residence, MEDICAL GENETICS
UCLA Associate Professor In-Residence, David Geffen School of Medicine at UCLA

M.D., Columbia University, New York, 1995 - 1999
A.B., University of Chicago, Chicago, Illinois, 1991 - 1995
2005 - 2006 UCLA Intercampus Medical Genetics Training Program, Los Angeles, CA
2003 - 2005 UCLA Intercampus Medical Genetics Training Program, Los Angeles, CA
1999 - 2002 Harbor-UCLA Medical Center, Torrance, CA


Dr. Patricia Dickson's role in the UCLA Clinical and Translational Science Institute will be to serve on the Committee on Child Health Research, a transdisciplinary, collaborative group who will define a strategic plan for the CTSI's important investigations in these areas of research. Her experience with clinical and translational research in a rare disease affecting children is a good fit for the goals of this committee and the project as a whole. The research focus is enzyme replacement therapy for the mucopolysaccharidoses (MPS), a group of lysosomal storage diseases. She has been performing research in intrathecal enzyme replacement therapy for MPS I (Hurler syndrome), and her laboratory is the leader in this pursuit, worldwide. In addition to the animal work funded by NINDS (1R01 NS054242-05), she is also the sponsor-investigator of the first clinical trial of intrathecal enzyme replacement therapy for mucopolysaccharidosis I, for the treatment of spinal cord compression due to the disease (supported by the Ryan Foundation and the FDA orphan products development program (1R01 FD003450-02)). She has given 22 invited lectures on enzyme replacement therapy for the MPS disorders since 2005, including presentations at FDA CDER, as a State-of-the Art speaker for the Western Society for Pediatric Research and at the annual meeting of the American College of Medical Genetics (concurrent invited session). Her lab also collaborates with the University of Minnesota in an ongoing project of intrathecal enzyme replacement therapy for Hurler patients undergoing hematopoietic stem cell transplantation, and in collaboration with Drs. Agnes Chen (Harbor-UCLA) and Elsa Shapiro (U. Minnesota), they are conducting a clinical trial of intrathecal enzyme replacement in relation to learning and memory in mucopolysaccharidosis I (Lysosomal Disease Network, a Rare Disease Clinical Research Network U54, C. Whitley, PI). Their studies use the Harbor-UCLA GCRC (M01-RR00425). In December 2009, she co-chaired a workshop, jointly sponsored by the FDA CDER (Division of Gastroenterology Products), NIH NINDS and NIH ORDR. The workshop, entitled "Research in Challenges in CNS Therapies for Inborn Errors of Metabolism" focused on the difficulties in translating therapies for CNS manifestations of inborn errors, including the inadequacy of outcome measures, ethical issues, and small study populations. She has experience with data and safety monitoring boards (both as investigator and DSMB member), investigational new drug applications, high-risk interventional clinical trials, regulatory issues including GCP, and preclinical research.

Vite Charles H, Nestrasil Igor, Mlikotic Anton, Jens Jackie K, Snella Elizabeth M, Gross William, Shapiro Elsa G, Kovac Victor, Provenzale James M, Chen Steven, Le Steven Q, Kan Shih-hsin, Banakar Shida, Wang Raymond Y, Haskins Mark E, Ellinwood N Matthew, Dickson Patricia I, Features of brain MRI in dogs with treated and untreated mucopolysaccharidosis type I. Comparative medicine. 2013; 63(2): 163-73.
Vera Moin, Lester Thomas, Zhao Bin, Tiger Pascale, Clarke Scott, Tippin Brigette L, Passage Merry B, Le Steven Q, Femenia Javier, Lemontt Jeffrey F, Kakkis Emil D, Dickson Patricia I, Mannose 6-phosphate conjugation is not sufficient to allow induction of immune tolerance to phenylalanine ammonia-lyase in dogs. JIMD reports. 2013; 8(2): 63-72.
Tippin Brigette L, Troitskaya Larisa, Kan Shih-hsin, Todd Amanda K, Le Steven Q, Dickson Patricia I, Biochemical characterization of fluorescent-labeled recombinant human alpha-L-iduronidase in vitro. Biotechnology and applied biochemistry. 2012; 58(6): 391-6.
Dickson Patricia I, Ellinwood N Matthew, Brown Jillian R, Witt Robert G, Le Steven Q, Passage Merry B, Vera Moin U, Crawford Brett E, Specific antibody titer alters the effectiveness of intrathecal enzyme replacement therapy in canine mucopolysaccharidosis I. Molecular genetics and metabolism. 2012; 106(1): 68-72.
Chen Agnes, Vogler Carole, McEntee Michael, Hanson Stephen, Ellinwood N Matthew, Jens Jackie, Snella Elizabeth, Passage Merry, Le Steven, Guerra Catalina, Dickson Patricia, Glycosaminoglycan storage in neuroanatomical regions of mucopolysaccharidosis I dogs following intrathecal recombinant human iduronidase. APMIS : acta pathologica, microbiologica, et immunologica Scandinavica. 2011; 119(8): 513-21.
Dickson Patricia I, Chen Agnes H, Intrathecal enzyme replacement therapy for mucopolysaccharidosis I: translating success in animal models to patients. Current pharmaceutical biotechnology. 2011; 12(6): 946-55.
Dickson Patricia I, Hanson Stephen, McEntee Michael F, Vite Charles H, Vogler Carole A, Mlikotic Anton, Chen Agnes H, Ponder Katherine P, Haskins Mark E, Tippin Brigette L, Le Steven Q, Passage Merry B, Guerra Catalina, Dierenfeld Ashley, Jens Jackie, Snella Elizabeth, Kan Shih-Hsin, Ellinwood N Matthew, Early versus late treatment of spinal cord compression with long-term intrathecal enzyme replacement therapy in canine mucopolysaccharidosis type I. Molecular genetics and metabolism. 2010; 101(2-3): 115-22.
Ezra Navid, Tetteh Beatrice, Diament Michael, Jonas Adam J, Dickson Patricia, Hereditary multiple exostoses with spine involvement in a 4-year-old boy. American journal of medical genetics. Part A. 2010; 152A(5): 1264-7.
Chen Agnes, Dickson Patricia, Intrathecal enzyme replacement therapy to treat spinal cord compression in mucopolysaccharidosis: Overview and rationale. Journal of pediatric rehabilitation medicine. 2010; 3(1): 7-11.
Dierenfeld AD, McEntee MF, Passage M, Le S, Jens JK, Snella EM, Kline KL, Parkes JD, Ware WA, Moran LE, Wengert JA, Whitley RD, Betts DM, Boal AM, Riedesel EA, Gross W, Ellinwood NM, Dickson PI, Replacing the enzyme α-L-iduronidase at birth ameliorates symptoms in the brain and periphery of dogs with mucopolysaccharidosis type I. Science Translational Medicine. 2010; 2(60): ra89.
Passage M B, Krieger A W, Peinovich M C, Lester T, Le S Q, Dickson P I, Kakkis E D, Continuous infusion of enzyme replacement therapy is inferior to weekly infusions in MPS I dogs. Journal of inherited metabolic disease. 2009; 110(3): .
Carlstrom Lucas P, Jens Jackie K, Dobyns Marley E, Passage Merry, Dickson Patricia I, Ellinwood N Matthew, Inadvertent propagation of factor VII deficiency in a canine mucopolysaccharidosis type I research breeding colony. Comparative medicine. 2009; 59(4): 378-82.
Dickson Patricia, Peinovich Maryn, McEntee Michael, Lester Thomas, Le Steven, Krieger Aimee, Manuel Hayden, Jabagat Catherine, Passage Merry, Kakkis Emil D, Immune tolerance improves the efficacy of enzyme replacement therapy in canine mucopolysaccharidosis I. The Journal of clinical investigation. 2008; 118(8): 2868-76.
Munoz-Rojas Maria-Veronica, Vieira Taiane, Costa Ronaldo, Fagondes Simone, John Angela, Jardim Laura Bannach, Vedolin Leonardo M, Raymundo Marcia, Dickson Patricia I, Kakkis Emil, Giugliani Roberto, Intrathecal enzyme replacement therapy in a patient with mucopolysaccharidosis type I and symptomatic spinal cord compression. American journal of medical genetics. Part A. 2008; 146A(19): 2538-44.
Dickson Patricia, McEntee Michael, Vogler Carole, Le Steven, Levy Beth, Peinovich Maryn, Hanson Stephen, Passage Merry, Kakkis Emil, Intrathecal enzyme replacement therapy: successful treatment of brain disease via the cerebrospinal fluid. Molecular genetics and metabolism. 2007; 91(1): 61-8.
Belichenko P V, Dickson P I, Passage M, Jungles S, Mobley W C, Kakkis E D, Penetration, diffusion, and uptake of recombinant human alpha-L-iduronidase after intraventricular injection into the rat brain. Molecular genetics and metabolism. 2005; 86(1-2): 141-9.
Dickson Patricia I, Briones Norman Y, Baylen Barry G, Jonas Adam J, French Samuel W, Lin Henry J, Costello syndrome with pancreatic islet cell hyperplasia. American journal of medical genetics. Part A. 2004; 130A(4): 402-5.
Kakkis E, McEntee M, Vogler C, Le S, Levy B, Belichenko P, Mobley W, Dickson P, Hanson S, Passage M, Intrathecal enzyme replacement therapy reduces lysosomal storage in the brain and meninges of the canine model of MPS I. Molecular genetics and metabolism. 2004; 83(1-2): 163-74.
Joyce James J, Dickson Patricia I, Qi Ning, Noble Julie E, Raj J Usha, Baylen Barry G, Normal right and left ventricular mass development during early infancy. The American journal of cardiology. 2004; 93(6): 797-801.
Dickson Patricia I, Delivering drugs to the central nervous system: an overview. Drug delivery and translational research. 2012; 2(3): 145-51.
Choi Joshua, Dickson Patricia, Calabrese Evan, Chen Steven, White Leonard, Ellingwood Matthew, Provenzale James M, Predicting degree of myelination based on diffusion tensor imagining of canines with mucopolysaccharidosis type I. The neuroradiology journal. 2015; 28(6): 562-73.
Dierenfeld Ashley D, McEntee Michael F, Vogler Carole A, Vite Charles H, Chen Agnes H, Passage Merry, Le Steven, Shah Sahil, Jens Jackie K, Snella Elizabeth M, Kline Karen L, Parkes Jennifer D, Ware Wendy A, Moran Lori E, Fales-Williams Amanda J, Wengert Jane A, Whitley R David, Betts Daniel M, Boal Amy M, Riedesel Elizabeth A, Gross William, Ellinwood N Matthew, Dickson Patricia I, Replacing the enzyme alpha-L-iduronidase at birth ameliorates symptoms in the brain and periphery of dogs with mucopolysaccharidosis type I. Science translational medicine. 2010; 2(60): 60ra89.
Lyons Jeremiah A, Dickson Patricia I, Wall Jonathan S, Passage Merry B, Ellinwood N Matthew, Kakkis Emil D, McEntee Michael F, Arterial pathology in canine mucopolysaccharidosis-I and response to therapy. Laboratory investigation; a journal of technical methods and pathology. 2011; 91(5): 665-74.
Kishnani Priya S, Dickson Patricia I, Muldowney Laurie, Lee Jessica J, Rosenberg Amy, Abichandani Rekha, Bluestone Jeffrey A, Burton Barbara K, Dewey Maureen, Freitas Alexandra, Gavin Derek, Griebel Donna, Hogan Melissa, Holland Stephen, Tanpaiboon Pranoot, Turka Laurence A, Utz Jeanine J, Wang Yow-Ming, Whitley Chester B, Kazi Zoheb B, Pariser Anne R, Immune response to enzyme replacement therapies in lysosomal storage diseases and the role of immune tolerance induction. Molecular genetics and metabolism. 2015; 28(6): .
Provenzale James M, Nestrasil Igor, Chen Steven, Kan Shih-Hsin, Le Steven Q, Jens Jacqueline K, Snella Elizabeth M, Vondrak Kristen N, Yee Jennifer K, Vite Charles H, Elashoff David, Duan Lewei, Wang Raymond Y, Ellinwood N Matthew, Guzman Miguel A, Shapiro Elsa G, Dickson Patricia I, Diffusion tensor imaging and myelin composition analysis reveal abnormal myelination in corpus callosum of canine mucopolysaccharidosis I. Experimental neurology. 2015; 273(6): 1-10.
Dickson Patricia I, Kaitila Ilkka, Harmatz Paul, Mlikotic Anton, Chen Agnes H, Victoroff Alla, Passage Merry B, Madden Jacqueline, Le Steven Q, Naylor David E, Naylor David E, Safety of laronidase delivered into the spinal canal for treatment of cervical stenosis in mucopolysaccharidosis I. Molecular genetics and metabolism. 2015; 116(1-2): 69-74.
Maarup Timothy J, Chen Agnes H, Porter Forbes D, Farhat Nicole Y, Ory Daniel S, Sidhu Rohini, Jiang Xuntian, Dickson Patricia I, Intrathecal 2-hydroxypropyl-beta-cyclodextrin in a single patient with Niemann-Pick C1. Molecular genetics and metabolism. 2015; 116(1-2): 75-9.
Dickson Patricia I, Tolar Jakub, The individual (single patient) IND for inborn errors of metabolism. Molecular genetics and metabolism. 2015; 116(1-2): 1-3.
Mendez Daniel C, Stover Alexander E, Rangel Anthony D, Brick David J, Nethercott Hubert E, Torres Marissa A, Khalid Omar, Wong Andrew Ms, Cooper Jonathan D, Jester James V, Monuki Edwin S, McGuire Cian, Le Steven Q, Kan Shih-Hsin, Dickson Patricia I, Schwartz Philip H, A novel, long-lived, and highly engraftable immunodeficient mouse model of mucopolysaccharidosis type I. Molecular therapy. Methods & clinical development. 2015; 2(1-2): 14068.
Shah Anuja, Miller Clinton J, Nast Cynthia C, Adams Mark D, Truitt Barbara, Tayek John A, Tong Lili, Mehtani Parag, Monteon Francisco, Sedor John R, Clinkenbeard Erica L, White Kenneth, Mehrotra Rajnish, LaPage Janine, Dickson Patricia, Adler Sharon G, Iyengar Sudha K, Severe vascular calcification and tumoral calcinosis in a family with hyperphosphatemia: a fibroblast growth factor 23 mutation identified by exome sequencing. Nephrology, dialysis, transplantation : official publication of the European Dialysis and Transplant Association - European Renal Association. 2014; 29(12): 2235-43.
Calias Pericles, Banks William A, Begley David, Scarpa Maurizio, Dickson Patricia, Intrathecal delivery of protein therapeutics to the brain: a critical reassessment. Pharmacology & therapeutics. 2014; 144(2): 114-22.
Kan Shih-Hsin, Aoyagi-Scharber Mika, Le Steven Q, Vincelette Jon, Ohmi Kazuhiro, Bullens Sherry, Wendt Daniel J, Christianson Terri M, Tiger Pascale M N, Brown Jillian R, Lawrence Roger, Yip Bryan K, Holtzinger John, Bagri Anil, Crippen-Harmon Danielle, Vondrak Kristen N, Chen Zhi, Hague Chuck M, Woloszynek Josh C, Cheung Diana S, Webster Katherine A, Adintori Evan G, Lo Melanie J, Wong Wesley, Fitzpatrick Paul A, LeBowitz Jonathan H, Crawford Brett E, Bunting Stuart, Dickson Patricia I, Neufeld Elizabeth F, Delivery of an enzyme-IGFII fusion protein to the mouse brain is therapeutic for mucopolysaccharidosis type IIIB. Proceedings of the National Academy of Sciences of the United States of America. 2014; 111(41): 14870-5.
Wang Raymond Y, Aminian Afshin, McEntee Michael F, Kan Shih-Hsin, Simonaro Calogera M, Lamanna William C, Lawrence Roger, Ellinwood N Matthew, Guerra Catalina, Le Steven Q, Dickson Patricia I, Esko Jeffrey D, Intra-articular enzyme replacement therapy with rhIDUA is safe, well-tolerated, and reduces articular GAG storage in the canine model of mucopolysaccharidosis type I. Molecular genetics and metabolism. 2014; 112(4): 286-93.
Kan Shih-hsin, Troitskaya Larisa A, Sinow Carolyn S, Haitz Karyn, Todd Amanda K, Di Stefano Ariana, Le Steven Q, Dickson Patricia I, Tippin Brigette L, Insulin-like growth factor II peptide fusion enables uptake and lysosomal delivery of α-N-acetylglucosaminidase to mucopolysaccharidosis type IIIB fibroblasts. The Biochemical journal. 2014; 458(2): 281-9.
Lawrence Roger, Brown Jillian R, Lorey Fred, Dickson Patricia I, Crawford Brett E, Esko Jeffrey D, Glycan-based biomarkers for mucopolysaccharidoses. Molecular genetics and metabolism. 2014; 111(2): 73-83.
Vera Moin, Le Steven, Kan Shih-Hsin, Garban Hermes, Naylor David, Mlikotic Anton, Kaitila Ilkka, Harmatz Paul, Chen Agnes, Dickson Patricia, Immune response to intrathecal enzyme replacement therapy in mucopolysaccharidosis I patients. Pediatric research. 2013; 74(6): 712-20.