Department of Pediatrics

Education

• BA, College of the University of Chicago, 1995
• MD, Columbia University College of Physicians and Surgeons, 1999

Training

• Internship and Residency, Los Angeles County/Harbor-UCLA Medical Center, 1999 - 2002
• Chief Residency, Los Angeles County/Harbor-UCLA Medical Center, 2002 - 2003
• Fellowship, UCLA Intercampus Medical Genetics Training Program, 2003 - 2006

Licensure and Board Certification

• 2000CA, Medical Board of California
• 2000 - 2020CA, California medical license
• 2002American Board of Pediatrics, General Pediatrics
• 2002 - PresAmerican Board of Pediatrics
• 2005American Board of Pediatrics, Clinical Genetics
• 2005 - PresAmerican Board of Medical Genetics, Clinical Genetics
• 2011American Board of Pediatrics, Medical Biochemical Genetics
• 2011 - PresAmerican Board of Medical Genetics, Medical Biochemical Genetics
• 2018 - PresMO, Missouri medical license

Honors and Awards

• Alpha Omega Alpha Student Essay Competition, Honorable Mention, Columbia University College of Physicians and Surgeons, New York, NY, 1996
• Primary Care Fellowship, Columbia University College of Physicians and Surgeons, New York, NY, 1996
• David Imagawa Memorial Fellowship Travel Award, Harbor-UCLA, Torrance, CA, 2001
• Federation of Pediatric Organizations Women in Pediatrics Task Force, 2003 - 2006
• NIH National Research Service Award Fellowship, UCLA Intercampus Genetics Training Program, Los Angeles, CA, 2005 - 2006
• Liu Young Investigator Award Finalist, Los Angeles Biomedical Research Institute at Harbor-UCLA, Torrance, CA, 2007
• Organizing Committee, Challenges in Research in CNS Manifestations of Inborn Errors of Metabolism Workshop, jointly sponsored by NIH ORDR, NIH NINDS, and FDA CDER, 2009 - 2009
• CTSI Maternal Child and Adolescent Health Award, UCLA, Los Angeles, CA, 2011
• Richard B. Weitzman Memorial Award for Meritorious Research, Los Angeles Biomedical Research Institute at Harbor-UCLA, Torrance, CA, 2011
• Young Investigator Award, Los Angeles Biomedical Research Institute at Harbor-UCLA, Torrance, CA, 2011
• Centennial Professor Endowed Chair, 2019
• American Society for Clinical Investigation, 2021

Recent Publications view all (83)

Publication Co-Authors

Dustin Baldridge

Marwan Shinawi, M.D.

Barbara B. Warner, M.D., MSc

Jennifer A Wambach, M.D.

Rachel Slaugh, MS, CGC

Jorge L Granadillo, MD, MSc.

Sakil S Kulkarni, MD

Dorothy Katherine Grange, M.D.

Jonathan D Cooper, Ph.D.

Tomi L. Toler, MS, CGC

Francis Sessions Cole, III, M.D.

Washington University has a long tradition of collaboration amongst colleagues.The faculty above have co-authored publications with Patricia Dickson

1. Sams EI, Ng JK, Tate V, Claire Hou YC, Cao Y, Antonacci-Fulton L, Belhassan K, Neidich J, Mitra RD, Cole FS, Dickson P, Milbrandt J, Turner TN. From karyotypes to precision genomics in 9p deletion and duplication syndromes. HGG Adv. 2022;3(1):100081. PMCID:PMC8756500  PMID:35047865 
2. Takahashi K, Le SQ, Kan SH, Jansen MJ, Dickson PI, Cooper JD. Neuropathology of murine Sanfilippo D syndrome. Mol Genet Metab. 2021;134(4):323-329. PMID:34844863 
3. Rodan LH, Spillmann RC, Kurata HT, Lamothe SM, Maghera J, Jamra RA, Alkelai A, Antonarakis SE, Atallah I, Bar-Yosef O, Bilan F, Bjorgo K, Blanc X, Van Bogaert P, Bolkier Y, Burrage LC, Christ BU, Granadillo JL, Dickson P, Donald KA, Dubourg C, Eliyahu A, Emrick L, Engleman K, Gonfiantini MV, Good JM, Kalser J, Kloeckner C, Lachmeijer G, Macchiaiolo M, Nicita F, Odent S, O'Heir E, Ortiz-Gonzalez X, Pacio-Miguez M, Palomares-Bralo M, Pena L, Platzer K, Quinodoz M, Ranza E, Rosenfeld JA, Roulet-Perez E, Santani A, Santos-Simarro F, Pode-Shakked B, Skraban C, Slaugh R, Superti-Furga A, Thiffault I, van Jaabrsveld RH, Vincent M, Wang HG, Zacher P, Undiagnosed Diseases Network., Rush E, Pitt GS, Au PYB, Shashi V. Correction: Phenotypic expansion of CACNA1C-associated disorders to include isolated neurological manifestations. Genet Med. 2021;23(10):2016. PMID:34522029 
4. Reynolds M, Linneman LA, Luna S, Warner BB, Turmelle YP, Kulkarni SS, Jiang X, Khanna G, Shinawi M, Porter FD, Ory DS, Cole FS, Dickson PI. A phase 1/2 open label nonrandomized clinical trial of intravenous 2-hydroxypropyl-β-cyclodextrin for acute liver disease in infants with Niemann-Pick C1. Mol Genet Metab Rep. 2021;28:100772. PMCID:PMC8170172  PMID:34113546 
5. Hurt SC, Dickson PI, Curiel DT. Mucopolysaccharidoses type I gene therapy. J Inherit Metab Dis. 2021. PMID:34189746 
6. Cheng KW, Wang F, Lopez GA, Singamsetty S, Wood J, Dickson PI, Chou TF. Evaluation of artificial signal peptides for secretion of two lysosomal enzymes in CHO cells. Biochem J. 2021;478(12):2309-2319. PMID:34032266 
7. Yang X, Yang X, Yu H, Na L, Ghosh T, McArthur JB, Chou TF, Dickson P, Chen X. A GH89 human α-N-acetylglucosaminidase (hNAGLU) homologue from gut microbe Bacteroides thetaiotaomicron capable of hydrolyzing heparosan oligosaccharides. AMB Express. 2021;11(1):94. PMCID:PMC8225759  PMID:34165649 
8. Rodan LH, Spillmann RC, Kurata HT, Lamothe SM, Maghera J, Jamra RA, Alkelai A, Antonarakis SE, Atallah I, Bar-Yosef O, Bilan F, Bjorgo K, Blanc X, Van Bogaert P, Bolkier Y, Burrage LC, Christ BU, Granadillo JL, Dickson P, Donald KA, Dubourg C, Eliyahu A, Emrick L, Engleman K, Gonfiantini MV, Good JM, Kalser J, Kloeckner C, Lachmeijer G, Macchiaiolo M, Nicita F, Odent S, O'Heir E, Ortiz-Gonzalez X, Pacio-Miguez M, Palomares-Bralo M, Pena L, Platzer K, Quinodoz M, Ranza E, Rosenfeld JA, Roulet-Perez E, Santani A, Santos-Simarro F, Pode-Shakked B, Skraban C, Slaugh R, Superti-Furga A, Thiffault I, van Jaabrsveld RH, Vincent M, Wang HG, Zacher P, Undiagnosed Diseases Network., Rush E, Pitt G, Au PYB, Shashi V. Phenotypic expansion of CACNA1C-associated disorders to include isolated neurological manifestations. Genet Med. 2021. PMID:34163037 
9. Kan SH, Elsharkawi I, Le SQ, Prill H, Mangini L, Cooper JD, Lawrence R, Sands MS, Crawford BE, Dickson PI. Biochemical evaluation of intracerebroventricular rhNAGLU-IGF2 enzyme replacement therapy in neonatal mice with Sanfilippo B syndrome. Mol Genet Metab. 2021;133(2):185-192. PMCID:PMC8195848  PMID:33839004 
10. Daly MB, Pal T, Berry MP, Buys SS, Dickson P, Domchek SM, Elkhanany A, Friedman S, Goggins M, Hutton ML, CGC., Karlan BY, Khan S, Klein C, Kohlmann W, CGC., Kurian AW, Laronga C, Litton JK, Mak JS, LCGC., Menendez CS, Merajver SD, Norquist BS, Offit K, Pederson HJ, Reiser G, CGC., Senter-Jamieson L, CGC., Shannon KM, Shatsky R, Visvanathan K, Weitzel JN, Wick MJ, Wisinski KB, Yurgelun MB, Darlow SD, Dwyer MA. Genetic/Familial High-Risk Assessment: Breast, Ovarian, and Pancreatic, Version 2.2021, NCCN Clinical Practice Guidelines in Oncology. J Natl Compr Canc Netw. 2021;19(1):77-102. PMID:33406487 
11. Wang F, Moen DR, Sauni C, Kan SH, Li S, Le SQ, Lomenick B, Zhang X, Ekins S, Singamsetty S, Wood J, Dickson PI, Chou TF. Enzyme Replacement Therapy for Mucopolysaccharidosis IIID using Recombinant Human α-N-Acetylglucosamine-6-Sulfatase in Neonatal Mice. Mol Pharm. 2021;18(1):214-227. PMID:33320673 
12. Clarke LA, Dickson P, Ellinwood NM, Klein TL. Newborn Screening for Mucopolysaccharidosis I: Moving Forward Learning from Experience. Int J Neonatal Screen. 2020;6(4). PMCID:PMC7712368  PMID:33227921 
13. Le SQ, Nestrasil I, Kan SH, Egeland M, Cooper JD, Elashoff D, Guo R, Tolar J, Yee JK, Dickson PI. Myelin and Lipid Composition of the Corpus Callosum in Mucopolysaccharidosis Type I Mice. Lipids. 2020;55(6):627-637. PMID:32537944 
14. Wongkittichote P, Watson JR, Leonard JM, Toolan ER, Dickson PI, Grange DK. Fatal COVID-19 infection in a patient with long-chain 3-hydroxyacyl-CoA dehydrogenase deficiency: A case report. JIMD Rep. 2020;56(1):40-45. PMCID:PMC7653242  PMID:33204595 
15. Zhang H, Dickson PI, Stiles AR, Chen AH, Le SQ, McCaw P, Beasley J, Millington DS, Young SP. Comparison of dermatan sulfate and heparan sulfate concentrations in serum, cerebrospinal fluid and urine in patients with mucopolysaccharidosis type I receiving intravenous and intrathecal enzyme replacement therapy. Clin Chim Acta. 2020;508:179-184. PMID:32442432 
16. Baldridge D, Spillmann RC, Wegner DJ, Wambach JA, White FV, Sisco K, Toler TL, Dickson PI, Cole FS, Shashi V, Grange DK. Phenotypic expansion of KMT2D-related disorder: Beyond Kabuki syndrome. Am J Med Genet A. 2020;182(5):1053-1065. PMID:32083401 
17. Daly MB, Pilarski R, Yurgelun MB, Berry MP, Buys SS, Dickson P, Domchek SM, Elkhanany A, Friedman S, Garber JE, Goggins M, Hutton ML, Khan S, Klein C, Kohlmann W, Kurian AW, Laronga C, Litton JK, Mak JS, Menendez CS, Merajver SD, Norquist BS, Offit K, Pal T, Pederson HJ, Reiser G, Shannon KM, Visvanathan K, Weitzel JN, Wick MJ, Wisinski KB, Dwyer MA, Darlow SD. NCCN Guidelines Insights: Genetic/Familial High-Risk Assessment: Breast, Ovarian, and Pancreatic, Version 1.2020. J Natl Compr Canc Netw. 2020;18(4):380-391. PMID:32259785 
18. Vera MU, Le SQ, Victoroff A, Passage MB, Brown JR, Crawford BE, Polgreen LE, Chen AH, Dickson PI. Evaluation of non-reducing end pathologic glycosaminoglycan detection method for monitoring therapeutic response to enzyme replacement therapy in human mucopolysaccharidosis I. Mol Genet Metab. 2020;129(2):91-97. PMCID:PMC7219480  PMID:31630958 
19. Chen AH, Harmatz P, Nestrasil I, Eisengart JB, King KE, Rudser K, Kaizer AM, Svatkova A, Wakumoto A, Le SQ, Madden J, Young S, Zhang H, Polgreen LE, Dickson PI. Intrathecal enzyme replacement for cognitive decline in mucopolysaccharidosis type I, a randomized, open-label, controlled pilot study. Mol Genet Metab. 2019. PMID:31839529 
20. Ahmed A, Ou L, Rudser K, Shapiro E, Eisengart JB, King K, Chen A, Dickson P, Whitley CB. A longitudinal study of neurocognition and behavior in patients with Hurler-Scheie syndrome heterozygous for the L238Q mutation. Mol Genet Metab Rep. 2019;20:100484. PMCID:PMC6603334  PMID:31304092 
21. Savarirayan R, Irving M, Bacino CA, Bostwick B, Charrow J, Cormier-Daire V, Le Quan Sang KH, Dickson P, Harmatz P, Phillips J, Owen N, Cherukuri A, Jayaram K, Jeha GS, Larimore K, Chan ML, Huntsman Labed A, Day J, Hoover-Fong J. C-Type Natriuretic Peptide Analogue Therapy in Children with Achondroplasia. N Engl J Med. 2019;381(1):25-35. PMID:31269546 
22. Labounek R, Mai K, Mueller B, Ellinwood NM, Dickson P, Nestrasil I. In-vivo cortical thickness estimation from high-resolution T₁w MRI scans in healthy and mucopolysaccharidosis affected dogs. Conf Proc IEEE Eng Med Biol Soc. 2019;2019:2848-2851. PMID:31946486 
23. Eisengart JB, Pierpont EI, Kaizer AM, Rudser KD, King KE, Pasquali M, Polgreen LE, Dickson PI, Le SQ, Miller WP, Tolar J, Orchard PJ, Lund TC. Intrathecal enzyme replacement for Hurler syndrome: biomarker association with neurocognitive outcomes. Genet Med. 2019. PMID:31019279 
24. Clarke D, Pearse Y, Kan SH, Le SQ, Sanghez V, Cooper JD, Dickson PI, Iacovino M. Genetically Corrected iPSC-Derived Neural Stem Cell Grafts Deliver Enzyme Replacement to Affect CNS Disease in Sanfilippo B Mice. Mol Ther Methods Clin Dev. 2018;10:113-127. PMCID:PMC6076361  PMID:30101150 
25. Jones L, Wells K, Lin HJ, Wang C, Alo AK, Williams P, Jones F, Dickson PI, Han S, Pardo D, Norris K, Jones A, Wright A, Young K, Rotter JI. Community Partnership in Precision Medicine: Themes from a Community Engagement Conference. Ethn Dis. 2018;28(Suppl 2):503-510. PMCID:PMC6128349  PMID:30202204 
26. Marcogliese PC, Shashi V, Spillmann RC, Stong N, Rosenfeld JA, Koenig MK, Martínez-Agosto JA, Herzog M, Chen AH, Dickson PI, Lin HJ, Vera MU, Salamon N, Graham JM Jr, Ortiz D, Infante E, Steyaert W, Dermaut B, Poppe B, Chung HL, Zuo Z, Lee PT, Kanca O, Xia F, Yang Y, Smith EC, Jasien J, Kansagra S, Spiridigliozzi G, El-Dairi M, Lark R, Riley K, Koeberl DD, Golden-Grant K, Program for Undiagnosed Diseases (UD-PrOZA)., Undiagnosed Diseases Network., Yamamoto S, Wangler MF, Mirzaa G, Hemelsoet D, Lee B, Nelson SF, Goldstein DB, Bellen HJ, Pena LDM. IRF2BPL Is Associated with Neurological Phenotypes. Am J Hum Genet. 2018;103(3):456. PMCID:PMC6128320  PMID:30193138 
27. Marcogliese PC, Shashi V, Spillmann RC, Stong N, Rosenfeld JA, Koenig MK, Martínez-Agosto JA, Herzog M, Chen AH, Dickson PI, Lin HJ, Vera MU, Salamon N, Graham JM Jr, Ortiz D, Infante E, Steyaert W, Dermaut B, Poppe B, Chung HL, Zuo Z, Lee PT, Kanca O, Xia F, Yang Y, Smith EC, Jasien J, Kansagra S, Spiridigliozzi G, El-Dairi M, Lark R, Riley K, Koeberl DD, Golden-Grant K, Program for Undiagnosed Diseases (UD-PrOZA)., Undiagnosed Diseases Network., Yamamoto S, Wangler MF, Mirzaa G, Hemelsoet D, Lee B, Nelson SF, Goldstein DB, Bellen HJ, Pena LDM. IRF2BPL Is Associated with Neurological Phenotypes. Am J Hum Genet. 2018;103(2):245-260. PMCID:PMC6081494  PMID:30057031 
28. Le SQ, Kan SH, Clarke D, Sanghez V, Egeland M, Vondrak KN, Doherty TM, Vera MU, Iacovino M, Cooper JD, Sands MS, Dickson PI. 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 
29. Middleton DM, Li JY, Chen SD, White LE, Dickson P, Ellinwood NM, Provenzale JM. 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 
30. Holley RJ, Ellison SM, Fil D, O'Leary C, McDermott J, Senthivel N, Langford-Smith AWW, Wilkinson FL, D'Souza Z, Parker H, Liao A, Rowlston S, Gleitz HFE, Kan SH, Dickson PI, Bigger BW. Macrophage enzyme and reduced inflammation drive brain correction of mucopolysaccharidosis IIIB by stem cell gene therapy. Brain. 2018;141(1):99-116. PMID:29186350 
31. Scarpa M, Orchard PJ, Schulz A, Dickson PI, Haskins ME, Escolar ML, Giugliani R. Treatment of brain disease in the mucopolysaccharidoses. Mol Genet Metab. 2017;122S:25-34. PMID:29153844 
32. Middleton DM, Li JY, Chen SD, White LE, Dickson PI, Matthew Ellinwood N, Provenzale JM. 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 
33. Middleton DM, Li JY, Lee HJ, Chen S, Dickson PI, Ellinwood NM, White LE, Provenzale JM. Diffusion tensor imaging tensor shape analysis for assessment of regional white matter differences. Neuroradiol J. 2017;30(4):324-329. PMCID:PMC5524281  PMID:28631949 
34. Li JY, Middleton DM, Chen S, White L, Ellinwood NM, Dickson P, Vite C, Bradbury A, Provenzale JM. 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 
35. Polgreen LE, Kunin-Batson A, Rudser K, Vehe RK, Utz JJ, Whitley CB, Dickson P. 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 
36. Nestrasil I, Shapiro E, Svatkova A, Dickson P, Chen A, Wakumoto A, Ahmed A, Stehel E, McNeil S, Gravance C, Maher E. 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 
37. Raymond GV, Pasquali M, Polgreen LE, Dickson PI, Miller WP, Orchard PJ, Lund TC. Elevated cerebral spinal fluid biomarkers in children with mucopolysaccharidosis I-H. Sci Rep. 2016;6:38305. PMCID:PMC5133554  PMID:27910891 
38. Kan SH, Le SQ, Bui QD, Benedict B, Cushman J, Sands MS, Dickson PI. Behavioral deficits and cholinergic pathway abnormalities in male Sanfilippo B mice. Behav Brain Res. 2016;312:265-71. PMCID:PMC4970944  PMID:27340089 
39. Polgreen LE, Vehe RK, Rudser K, Kunin-Batson A, Utz JJ, Dickson P, Shapiro E, Whitley CB. 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 
40. Khalid O, Vera MU, Gordts PL, Ellinwood NM, Schwartz PH, Dickson PI, Esko JD, Wang RY. 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 
41. Kishnani PS, Dickson PI, Muldowney L, Lee JJ, Rosenberg A, Abichandani R, Bluestone JA, Burton BK, Dewey M, Freitas A, Gavin D, Griebel D, Hogan M, Holland S, Tanpaiboon P, Turka LA, Utz JJ, Wang YM, Whitley CB, Kazi ZB, Pariser AR. 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 
42. Choi J, Dickson P, Calabrese E, Chen S, White L, Ellingwood M, Provenzale JM. 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 
43. Dickson PI, Kaitila I, Harmatz P, Mlikotic A, Chen AH, Victoroff A, Passage MB, Madden J, Le SQ, Naylor DE, Mucopolysaccharidosis I Intrathecal Research Collaborative.. 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 
44. Provenzale JM, Nestrasil I, Chen S, Kan SH, Le SQ, Jens JK, Snella EM, Vondrak KN, Yee JK, Vite CH, Elashoff D, Duan L, Wang RY, Ellinwood NM, Guzman MA, Shapiro EG, Dickson PI. 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 
45. Dickson PI, Kaitila I, Harmatz P, Mlikotic A, Chen AH, Victoroff A, Passage MB, Madden J, Le SQ, Naylor DE, Mucopolysaccharidosis I Intrathecal Research Collaborative.. 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 
46. Dickson PI, Tolar J. The individual (single patient) IND for inborn errors of metabolism. Mol Genet Metab. 2015;116(1-2):1-3. PMID:25456747 
47. Maarup TJ, Chen AH, Porter FD, Farhat NY, Ory DS, Sidhu R, Jiang X, Dickson PI. 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 
48. Mendez DC, Stover AE, Rangel AD, Brick DJ, Nethercott HE, Torres MA, Khalid O, Wong AM, Cooper JD, Jester JV, Monuki ES, McGuire C, Le SQ, Kan SH, Dickson PI, Schwartz PH. 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 
49. Shah A, Miller CJ, Nast CC, Adams MD, Truitt B, Tayek JA, Tong L, Mehtani P, Monteon F, Sedor JR, Clinkenbeard EL, White K, Mehrotra R, LaPage J, Dickson P, Adler SG, Iyengar SK. 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 
50. Calias P, Banks WA, Begley D, Scarpa M, Dickson P. Intrathecal delivery of protein therapeutics to the brain: a critical reassessment. Pharmacol Ther. 2014;144(2):114-22. PMID:24854599 
51. Kan SH, Aoyagi-Scharber M, Le SQ, Vincelette J, Ohmi K, Bullens S, Wendt DJ, Christianson TM, Tiger PM, Brown JR, Lawrence R, Yip BK, Holtzinger J, Bagri A, Crippen-Harmon D, Vondrak KN, Chen Z, Hague CM, Woloszynek JC, Cheung DS, Webster KA, Adintori EG, Lo MJ, Wong W, Fitzpatrick PA, LeBowitz JH, Crawford BE, Bunting S, Dickson PI, Neufeld EF. 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 
52. Wang RY, Aminian A, McEntee MF, Kan SH, Simonaro CM, Lamanna WC, Lawrence R, Ellinwood NM, Guerra C, Le SQ, Dickson PI, Esko JD. 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 
53. Kan SH, Troitskaya LA, Sinow CS, Haitz K, Todd AK, Di Stefano A, Le SQ, Dickson PI, Tippin BL. 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 
54. Lawrence R, Brown JR, Lorey F, Dickson PI, Crawford BE, Esko JD. Glycan-based biomarkers for mucopolysaccharidoses. Mol Genet Metab. 2014;111(2):73-83. PMCID:PMC3769472  PMID:23958290 
55. Vera M, Le S, Kan SH, Garban H, Naylor D, Mlikotic A, Kaitila I, Harmatz P, Chen A, Dickson P. Immune response to intrathecal enzyme replacement therapy in mucopolysaccharidosis I patients. Pediatr Res. 2013;74(6):712-20. PMCID:PMC3855632  PMID:24002329 
56. Vite CH, Nestrasil I, Mlikotic A, Jens JK, Snella EM, Gross W, Shapiro EG, Kovac V, Provenzale JM, Chen S, Le SQ, Kan SH, Banakar S, Wang RY, Haskins ME, Ellinwood NM, Dickson PI. Features of brain MRI in dogs with treated and untreated mucopolysaccharidosis type I. Comp Med. 2013;63(2):163-73. PMCID:PMC3625057  PMID:23582423 
57. Vera M, Lester T, Zhao B, Tiger P, Clarke S, Tippin BL, Passage MB, Le SQ, Femenia J, Lemontt JF, Kakkis ED, Dickson PI. 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 
58. Dickson PI. Delivering drugs to the central nervous system: an overview. Drug Deliv Transl Res. 2012;2(3):145-51. PMID:25786863 
59. 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;106(1):68-72. PMCID:PMC3336016  PMID:22402327 
60. 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;58(6):391-6. PMCID:PMC3293367  PMID:22172101 
61. 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;119(8):513-21. PMCID:PMC3137268  PMID:21749451 
62. Newkirk KM, Atkins RM, Dickson PI, Rohrbach BW, McEntee MF. Ocular lesions in canine mucopolysaccharidosis I and response to enzyme replacement therapy. Invest Ophthalmol Vis Sci. 2011;52(8):5130-5. PMID:21436264 
63. Dickson PI, Chen AH. Intrathecal enzyme replacement therapy for mucopolysaccharidosis I: translating success in animal models to patients. Curr Pharm Biotechnol. 2011;12(6):946-55. PMID:21506913 
64. Lyons JA, Dickson PI, Wall JS, Passage MB, Ellinwood NM, Kakkis ED, McEntee MF. Arterial pathology in canine mucopolysaccharidosis-I and response to therapy. Lab Invest. 2011;91(5):665-74. PMCID:PMC3084338  PMID:21383673 
65. Dickson PI, Pariser AR, Groft SC, Ishihara RW, McNeil DE, Tagle D, Griebel DJ, Kaler SG, Mink JW, Shapiro EG, Bjoraker KJ, Krivitzky L, Provenzale JM, Gropman A, Orchard P, Raymond G, Cohen BH, Steiner RD, Goldkind SF, Nelson RM, Kakkis E, Patterson MC. Research challenges in central nervous system manifestations of inborn errors of metabolism. Mol Genet Metab. 2011;102(3):326-38. PMCID:PMC3040279  PMID:21176882 
66. Wu SJ, Tetteh B, Dickson P. Index of suspicion, case 3: Hyperimmunoglobulin D syndrome Pediatr Rev. 2011;32:299-305. 
67. Dierenfeld AD, McEntee MF, Vogler CA, Vite CH, Chen AH, Passage M, Le S, Shah S, Jens JK, Snella EM, Kline KL, Parkes JD, Ware WA, Moran LE, Fales-Williams AJ, Wengert JA, Whitley RD, Betts DM, Boal AM, Riedesel EA, Gross W, Ellinwood NM, Dickson PI. Replacing the enzyme alpha-L-iduronidase at birth ameliorates symptoms in the brain and periphery of dogs with mucopolysaccharidosis type I. Sci Transl Med. 2010;2(60):60ra89. PMCID:PMC3075726  PMID:21123810 
68. Dickson PI, Hanson S, McEntee MF, Vite CH, Vogler CA, Mlikotic A, Chen AH, Ponder KP, Haskins ME, Tippin BL, Le SQ, Passage MB, Guerra C, Dierenfeld A, Jens J, Snella E, Kan SH, Ellinwood NM. 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(2-3):115-22. PMCID:PMC2950221  PMID:20655780 
69. Ezra N, Tetteh B, Diament M, Jonas AJ, Dickson P. Hereditary multiple exostoses with spine involvement in a 4-year-old boy. Am J Med Genet A. 2010;152A(5):1264-7. PMID:20425833 
70. Chen A, Dickson P. Intrathecal enzyme replacement therapy to treat spinal cord compression in mucopolysaccharidosis: Overview and rationale. J Pediatr Rehabil Med. 2010;3(1):7-11. PMID:21791827 
71. Passage MB, Krieger AW, Peinovich MC, Lester T, Le SQ, Dickson PI, Kakkis ED. Continuous infusion of enzyme replacement therapy is inferior to weekly infusions in MPS I dogs. J Inherit Metab Dis. 2009;32 Suppl 1:S253-8. PMCID:PMC2889197  PMID:19562502 
72. Carlstrom LP, Jens JK, Dobyns ME, Passage M, Dickson PI, Ellinwood NM. Inadvertent propagation of factor VII deficiency in a canine mucopolysaccharidosis type I research breeding colony. Comp Med. 2009;59(4):378-82. PMCID:PMC2779214  PMID:19712579 
73. Dickson PI. Novel treatments and future perspectives: outcomes of intrathecal drug delivery. Int J Clin Pharmacol Ther. 2009;47 Suppl 1:S124-7. PMID:20040323 
74. Muñoz-Rojas MV, Costa R, Fagondes Canani S, Jardim L, Vedolin L, Kakkis E, Dickson P, Vieira T, John AB, Raymundo M, Giugliani R.. Intrathecal enzyme replacement therapy in a patient with mucopolysaccharidosis type I and symptomatic spinal cord compression Am J Med Genet A. 2008;epub. 
75. Dickson P, Peinovich M, McEntee M, Lester T, Le S, Krieger A, Manuel H, Jabagat C, Passage M, Kakkis E. 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. 
76. Dickson P, McEntee M, Vogler C, Le S, Levy B, Peinovich M, Hanson S, Passage M, Kakkis E. Intrathecal enzyme replacement therapy: Successful treatment of brain disease via the cerebrospinal fluid. Mol Genet Metab. 2007;91:61-68. 
77. Tiranti V, Briem E, Lamantea E, Mineri R, Papaleo E, Degioia L, Forlani F, Rinaldo P, Dickson P, Abu-Libdeh B, Cindro-Heberle L, Owaidha M, Jack RM, Christensen E, Burlina A, Zeviani M. ETHE1 mutations are specific to Ethylmalonic Encephalopathy. J Med Genet. 2006;43:340-6. 
78. Belichenko PV, Dickson P, Passage M, Jungles S, Mobley WC, Kakkis E. 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. 
79. Dickson PI, Briones NY, Baylen BG, Jonas AJ, French SW, Lin HJ. Costello syndrome with pancreatic islet cell hyperplasia. Am J Med Genet A. 2004;130A(4):402-5. PMID:15384101 
80. Joyce JJ, Dickson PI, Qi N, Noble JE, Raj JU, Baylen BG. Normal right and left ventricular mass development during early infancy. Am J Cardiol. 2004;93(6):797-801. PMID:15019899 
81. 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. ol Genet Metab. 2004;83:163-174. 
82. Joyce JJ, Dickson PI, Qi N, Noble JE, Raj JU, Baylen BG. Normal right and left ventricular mass development during early infancy. Am J Cardiology. 2004;93:797-801. 
83. Dayan P, Ahmad F, Urtecho J, Novick M, Dixon P (sic), Levine D, Miller S. 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.