Patricia Dickson  pdickson@wustl.edu

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Division Chief, Genetics and Genomic Medicine
Genetics and Genomic Medicine

phone: (314) 454-6093

Education

  • BA, College of the University of Chicago1995
  • MD, Columbia University College of Physicians and Surgeons1999

Training

  • Internship and Residency, Los Angeles County/Harbor-UCLA Medical Center1999 - 2002
  • Chief Residency, Los Angeles County/Harbor-UCLA Medical Center2002 - 2003
  • Fellowship, UCLA Intercampus Medical Genetics Training Program2003 - 2006

Licensure and Board Certification

  • CA, Medical Board of California 2000
  • CA, California medical license 2000
  • Diplomate, American Board of Pediatrics (General Pediatrics) 2002
  • Diplomate, American Board of Pediatrics (Clinical Genetics) 2005
  • Diplomate, American Board of Pediatrics (Medical Biochemical Genetics) 2011
  • MO, Missouri medical license 2018

Honors

  • Alpha Omega Alpha Student Essay Competition, Honorable Mention, Columbia University College of Physicians and Surgeons, New York, NY1996
  • Primary Care Fellowship, Columbia University College of Physicians and Surgeons, New York, NY1996
  • David Imagawa Memorial Fellowship Travel Award, Harbor-UCLA, Torrance, CA2001
  • Federation of Pediatric Organizations Women in Pediatrics Task Force2003 - 2006
  • NIH National Research Service Award Fellowship, UCLA Intercampus Genetics Training Program, Los Angeles, CA2005 - 2006
  • Liu Young Investigator Award Finalist, Los Angeles Biomedical Research Institute at Harbor-UCLA, Torrance, CA2007
  • Organizing Committee, Challenges in Research in CNS Manifestations of Inborn Errors of Metabolism Workshop, jointly sponsored by NIH ORDR, NIH NINDS, and FDA CDER2009 - 2009
  • Ad hoc member, NICHD Study Section: 2009/05 ZHD1 MRG-C2009
  • Ad hoc grant reviewer, Australian Government National Health and Medical Research Council (NHMRC)2009
  • Ad hoc Challenge Grant mail reviewer, 2009/10 ZRG1 GGG-F (58) CSR Special Emphasis Panel2009
  • Member, Therapeutics Committee, American College of Medical Genetics2011
  • CTSI Maternal Child and Adolescent Health Award, UCLA, Los Angeles, CA2011
  • Richard B. Weitzman Memorial Award for Meritorious Research, Los Angeles Biomedical Research Institute at Harbor-UCLA, Torrance, CA2011
  • Young Investigator Award, Los Angeles Biomedical Research Institute at Harbor-UCLA, Torrance, CA2011
  • Member, ad hoc Technical Expert Panel on Newborn Screening for MPS I, Condition Review Workgroup at Duke Clinical Research Institute by the U S Secretary of Health and Human Services Advisory Committee on Heritable Disorders in Newborns and Children2013
  • Member, NIH Special Emphasis Panel ZHG1 HGR-P (J2) for review of applications in response to RFA-RM-13-004, “Clinical Sites for an Undiagnosed Diseases Network”2013
  • Member, ad hoc committee on animal harmonization, UCLA CTSI2014 - 2015
  • Mail-in reviewer (ad hoc “Subject Expert”), NIH Special Emphasis Panel ZRG1 RPHB-W (53) R for review of applications in response to RFA-RM-13-009, “NIH Director’s Early Independence Awards (DP5)”2014
  • Member, Neurologic & Ophthalmic Gene & Cell Therapy Committee, American Society of Gene and Cell Therapy2015 - Pres
  • Standing member, NIH/NINDS study section NSD-B2015 - Pres
  • Ad hoc member, NINDS study section NSD-B2015 - 2015
  • Ad hoc member, NIH study section DBD2015 - 2016
  • Ad hoc mail-in reviewer, NIH Special Emphasis Panel ZRG1 BDCN-M2016
  • Chair, NIH Special Emphasis Panel for review or applications in response to RFA-RM-17-019, “Clinical Sites for the Undiagnosed Diseases Network (UDN) Phase II”2018

Recent Publications view all (53)


Publication Co-Authors

  1. IRF2BPL Is Associated with Neurological Phenotypes. Am J Hum Genet. 2018. PMID:30057031 
  2. A Humoral Immune Response Alters the Distribution of Enzyme Replacement Therapy in Murine Mucopolysaccharidosis Type I. Mol Ther Methods Clin Dev. 2018;8:42-51. PMCID:PMC5684429  PMID:29159202 
  3. Diffusion tensor imaging findings suggestive of white matter alterations in a canine model of mucopolysaccharidosis type I. Neuroradiol J. 2018;31(1):90-94. PMCID:PMC5789994  PMID:28695759 
  4. Macrophage enzyme and reduced inflammation drive brain correction of mucopolysaccharidosis IIIB by stem cell gene therapy. Brain. 2018;141(1):99-116. PMID:29186350 
  5. Treatment of brain disease in the mucopolysaccharidoses. Mol Genet Metab. 2017;122S:25-34. PMID:29153844 
  6. Quantitative diffusion tensor imaging analysis does not distinguish pediatric canines with mucopolysaccharidosis I from control canines. Neuroradiol J. 2017;30(5):454-460. PMCID:PMC5602339  PMID:28703635 
  7. Diffusion tensor imaging tensor shape analysis for assessment of regional white matter differences. Neuroradiol J. 2017;30(4):324-329. PMCID:PMC5524281  PMID:28631949 
  8. Novel region of interest interrogation technique for diffusion tensor imaging analysis in the canine brain. Neuroradiol J. 2017;30(4):339-346. PMCID:PMC5524282  PMID:28627967 
  9. Pilot study of the safety and effect of adalimumab on pain, physical function, and musculoskeletal disease in mucopolysaccharidosis types I and II. Mol Genet Metab Rep. 2017;10:75-80. PMCID:PMC5238608  PMID:28119823 
  10. Intrathecal enzyme replacement therapy reverses cognitive decline in mucopolysaccharidosis type I. Am J Med Genet A. 2017;173(3):780-783. PMCID:PMC5367919  PMID:28211988 
  11. Elevated cerebral spinal fluid biomarkers in children with mucopolysaccharidosis I-H. Sci Rep. 2016;6:38305. PMCID:PMC5133554  PMID:27910891 
  12. Behavioral deficits and cholinergic pathway abnormalities in male Sanfilippo B mice. Behav Brain Res. 2016;312:265-71. PMCID:PMC4970944  PMID:27340089 
  13. Elevated TNF-α is associated with pain and physical disability in mucopolysaccharidosis types I, II, and VI. Mol Genet Metab. 2016;117(4):427-30. PMCID:PMC4851859  PMID:26873528 
  14. Immune-Mediated Inflammation May Contribute to the Pathogenesis of Cardiovascular Disease in Mucopolysaccharidosis Type I. PLoS One. 2016;11(3):e0150850. PMCID:PMC4795702  PMID:26986213 
  15. Immune response to enzyme replacement therapies in lysosomal storage diseases and the role of immune tolerance induction. Mol Genet Metab. 2016;117(2):66-83. PMID:26597321 
  16. Predicting degree of myelination based on diffusion tensor imagining of canines with mucopolysaccharidosis type I. Neuroradiol J. 2015;28(6):562-73. PMCID:PMC4757141  PMID:26475483 
  17. Data from subjects receiving intrathecal laronidase for cervical spinal stenosis due to mucopolysaccharidosis type I. Data Brief. 2015;5:71-6. PMCID:PMC4573094  PMID:26484358 
  18. Diffusion tensor imaging and myelin composition analysis reveal abnormal myelination in corpus callosum of canine mucopolysaccharidosis I. Exp Neurol. 2015;273:1-10. PMCID:PMC4644459  PMID:26222335 
  19. Safety of laronidase delivered into the spinal canal for treatment of cervical stenosis in mucopolysaccharidosis I. Mol Genet Metab. 2015;116(1-2):69-74. PMCID:PMC4572891  PMID:26260077 
  20. The individual (single patient) IND for inborn errors of metabolism. Mol Genet Metab. 2015;116(1-2):1-3. PMID:25456747 
  21. Intrathecal 2-hydroxypropyl-beta-cyclodextrin in a single patient with Niemann-Pick C1. Mol Genet Metab. 2015;116(1-2):75-9. PMCID:PMC4633280  PMID:26189084 
  22. A novel, long-lived, and highly engraftable immunodeficient mouse model of mucopolysaccharidosis type I. Mol Ther Methods Clin Dev. 2015;2:14068. PMCID:PMC4449030  PMID:26052536 
  23. Severe vascular calcification and tumoral calcinosis in a family with hyperphosphatemia: a fibroblast growth factor 23 mutation identified by exome sequencing. Nephrol Dial Transplant. 2014;29(12):2235-43. PMCID:PMC4240183  PMID:25378588 
  24. Delivery of an enzyme-IGFII fusion protein to the mouse brain is therapeutic for mucopolysaccharidosis type IIIB. Proc Natl Acad Sci U S A. 2014;111(41):14870-5. PMCID:PMC4205671  PMID:25267636 
  25. Intra-articular enzyme replacement therapy with rhIDUA is safe, well-tolerated, and reduces articular GAG storage in the canine model of mucopolysaccharidosis type I. Mol Genet Metab. 2014;112(4):286-93. PMCID:PMC4122635  PMID:24951454 
  26. Insulin-like growth factor II peptide fusion enables uptake and lysosomal delivery of α-N-acetylglucosaminidase to mucopolysaccharidosis type IIIB fibroblasts. Biochem J. 2014;458(2):281-9. PMCID:PMC3993987  PMID:24266751 
  27. Glycan-based biomarkers for mucopolysaccharidoses. Mol Genet Metab. 2014;111(2):73-83. PMCID:PMC3769472  PMID:23958290 
  28. Immune response to intrathecal enzyme replacement therapy in mucopolysaccharidosis I patients. Pediatr Res. 2013;74(6):712-20. PMCID:PMC3855632  PMID:24002329 
  29. fMRI responses to Jung's Word Association Test: implications for theory, treatment and research. J Anal Psychol. 2013;58(3):409-431. PMID:23750943 
  30. Features of brain MRI in dogs with treated and untreated mucopolysaccharidosis type I. Comp Med. 2013;63(2):163-73. PMCID:PMC3625057  PMID:23582423 
  31. WITHDRAWN: Glycan-based biomarkers for mucopolysaccharidoses. Dis Markers. 2013. PMID:23396297 
  32. Mannose 6-phosphate conjugation is not sufficient to allow induction of immune tolerance to phenylalanine ammonia-lyase in dogs. JIMD Rep. 2013;8:63-72. PMCID:PMC3565636  PMID:23430522 
  33. Specific antibody titer alters the effectiveness of intrathecal enzyme replacement therapy in canine mucopolysaccharidosis I Mol Genet Metab. 2012;106(1):68-72. 
  34. Index of suspicion, case 3: Hyperimmunoglobulin D syndrome Pediatr Rev. 2011;32:299-305. 
  35. Research challenges in CNS manifestations of inborn errors of metabolism Mol Genet Metab. 2011;102(3):326-38. 
  36. Arterial pathology in canine mucopolysaccharidosis-I and response to therapy Lab Invest. 2011;91:665-674. 
  37. Glycosaminoglycan storage in neuroanatomical regions of mucopolysaccharidosis I dogs following intrathecal recombinant human iduronidase. , 119(8):513-521, 2011. APMIS. 2011;119(8):513-521. 
  38. Ocular lesions in canine mucopolysaccharidosis I and response to enzyme replacement therapy. , 52(8):5130-5, 2011. Invest Ophthalmol Vis Sci. 2011;52(8):5130-5. 
  39. Biochemical characterization of fluorescent-labeled recombinant human alpha-L-iduronidase in vitro Biotechnol Appl Biochem. 2011;58:391-6. 
  40. Hereditary Multiple Exostoses with spine involvement in a four year old boy, ,152A(5):1264-7, 2010. Am J Med Genet A. 2010;152A(5):1264-7. 
  41. Early versus late treatment of spinal cord compression with long-term intrathecal enzyme replacement therapy in canine mucopolysaccharidosis type I Mol Genet Metab. 2010;101:115-122. 
  42. Replacing the enzyme α-L-iduronidase at birth ameliorates symptoms in the brain and periphery of dogs with mucopolysaccharidosis type I Sci Transl Med. 2010;2:60ra89. 
  43. Continuous infusion of enzyme replacement therapy is inferior to weekly infusions in MPS I dogs J Inh Metab Dis. 2009;epub. 
  44. Canine factor VII deficiency: Propagation of inadvertent inherited genetic disease within a canine mucopolysaccharidosis type I research breeding colony Comp Med 59. 2009;378-382. 
  45. Intrathecal enzyme replacement therapy in a patient with mucopolysaccharidosis type I and symptomatic spinal cord compression Am J Med Genet A. 2008;epub. 
  46. Immune tolerance improves the efficacy of enzyme replacement therapy in the canine model of mucopolysaccharidosis I. J Clin Invest. 2008;118:2868-76 with introduction by K. Ponder at pp. 2686-89. 
  47. Intrathecal enzyme replacement therapy: Successful treatment of brain disease via the cerebrospinal fluid. Mol Genet Metab. 2007;91:61-68. 
  48. ETHE1 mutations are specific to Ethylmalonic Encephalopathy. J Med Genet. 2006;43:340-6. 
  49. Penetration, diffusion and uptake of recombinant human α-L-iduronidase after intraventricular injection into the rat brain. 86:141-149, 2005 Mol Genet Metab. 2005;86:141-149. 
  50. Costello syndrome with pancreatic islet cell hyperplasia: comparison to disorders of insulin or insulin-like growth factor pathways. , 130:402-405, 2004 Am J Med Genet A. 2004;130:402-405. 
  51. Intrathecal enzyme replacement therapy reduces lysosomal storage in the brain and meninges of the canine model of MPS I. ol Genet Metab. 2004;83:163-174. 
  52. Normal right and left ventricular mass development during early infancy. Am J Cardiology. 2004;93:797-801. 
  53. Test characteristics of the respiratory syncytial virus enzyme-linked immunoabsorbent assay in febrile infants < or = 60 days of age. Clinics in Pediatrics. 2002;41:415-8. 
Last updated: 08/03/2018
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