Patricia Dickson

2022

Brain transplantation of genetically corrected Sanfilippo type B neural stem cells induces partial cross-correction of the disease

Pearse, Y., Clarke, D., Kan, S. H., Le, S. Q., Sanghez, V., Luzzi, A., Pham, I., Nih, L. R., Cooper, J., Dickson, P. I. & Iacovino, M., Dec 8 2022, In: Molecular Therapy - Methods and Clinical Development. 27, p. 452-463 12 p.

Research output: Contribution to journal › Article › peer-review

Open Access

1 Scopus citations

Cross-species efficacy of enzyme replacement therapy for CLN1 disease in mice and sheep

Nelvagal, H. R., Eaton, S. L., Wang, S. H., Eultgen, E. M., Takahashi, K., Le, S. Q., Nesbitt, R., Dearborn, J. T., Siano, N., Puhl, A. C., Dickson, P. I., Thompson, G., Murdoch, F., Brennan, P. M., Gray, M., Greenhalgh, S. N., Tennant, P., Gregson, R., Clutton, E., Nixon, J., & 10 othersProudfoot, C., Guido, S., Lillico, S. G., Whitelaw, C. A. B., Lu, J. Y., Hofmann, S. L., Ekins, S., Sands, M. S., Wishart, T. M. & Cooper, J. D., Oct 17 2022, In: Journal of Clinical Investigation. 132, 20, e163107.

Research output: Contribution to journal › Article › peer-review

Open Access

Diagnostic yield of exome sequencing in congenital vertical talus

Tayebi, N., Charng, W. L., Dickson, P. I., Dobbs, M. B. & Gurnett, C. A., Jun 2022, In: European Journal of Medical Genetics. 65, 6, 104514.

Research output: Contribution to journal › Article › peer-review

Open Access

1 Scopus citations

From karyotypes to precision genomics in 9p deletion and duplication syndromes

Sams, E. I., Ng, J. K., Tate, V., Claire Hou, Y. C., Cao, Y., Antonacci-Fulton, L., Belhassan, K., Neidich, J., Mitra, R. D., Cole, F. S., Dickson, P., Milbrandt, J. & Turner, T. N., Jan 13 2022, In: Human Genetics and Genomics Advances. 3, 1, 100081.

Research output: Contribution to journal › Article › peer-review

Open Access

3 Scopus citations

Impaired mitophagy in Sanfilippo a mice causes hypertriglyceridemia and brown adipose tissue activation

Tillo, M., Lamanna, W. C., Dwyer, C. A., Sandoval, D. R., Pessentheiner, A. R., Al-Azzam, N., Sarrazin, S., Gonzales, J. C., Kan, S. H., Andreyev, A. Y., Schultheis, N., Thacker, B. E., Glass, C. A., Dickson, P. I., Wang, R. Y., Selleck, S. B., Esko, J. D. & Gordts, P. L. S. M., Aug 2022, In: Journal of Biological Chemistry. 298, 8, 102159.

Research output: Contribution to journal › Article › peer-review

Open Access

1 Scopus citations

Infantile-onset Pompe disease complicated by sickle cell anemia: Case report and management considerations

Starosta, R. T., Hou, Y. C. C., Leestma, K., Singh, P., Viehl, L., Manwaring, L., Granadillo, J. L., Schroeder, M. C., Colombo, J. N., Whitehead, H., Dickson, P. I., Hulbert, M. L. & Nguyen, H. T., Sep 28 2022, In: Frontiers in Pediatrics. 10, 944178.

Research output: Contribution to journal › Article › peer-review

Open Access

1 Scopus citations

Whole-Genome and Long-Read Sequencing Identify a Novel Mechanism in RFC1 Resulting in CANVAS Syndrome

King, K. A., Wegner, D. J., Bucelli, R. C., Shapiro, J., Paul, A. J., Dickson, P. I. & Wambach, J. A., Dec 1 2022, In: Neurology: Genetics. 8, 6, e200036.

Research output: Contribution to journal › Article › peer-review

Open Access

2021

A GH89 human α-N-acetylglucosaminidase (hNAGLU) homologue from gut microbe Bacteroides thetaiotaomicron capable of hydrolyzing heparosan oligosaccharides

Yang, X., Yang, X., Yu, H., Na, L., Ghosh, T., McArthur, J. B., Chou, T. F., Dickson, P. & Chen, X., Dec 2021, In: AMB Express. 11, 1, 94.

Research output: Contribution to journal › Article › peer-review

Open Access

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

Reynolds, M., Linneman, L. A., Luna, S., Warner, B. B., Turmelle, Y. P., Kulkarni, S. S., Jiang, X., Khanna, G., Shinawi, M., Porter, F. D., Ory, D. S., Cole, F. S. & Dickson, P. I., Sep 2021, In: Molecular Genetics and Metabolism Reports. 28, 100772.

Research output: Contribution to journal › Article › peer-review

Open Access

8 Scopus citations

Biochemical evaluation of intracerebroventricular rhNAGLU-IGF2 enzyme replacement therapy in neonatal mice with Sanfilippo B syndrome

Kan, S. H., Elsharkawi, I., Le, S. Q., Prill, H., Mangini, L., Cooper, J. D., Lawrence, R., Sands, M. S., Crawford, B. E. & Dickson, P. I., Jun 2021, In: Molecular genetics and metabolism. 133, 2, p. 185-192 8 p.

Research output: Contribution to journal › Article › peer-review

1 Scopus citations

Correction: Phenotypic expansion of CACNA1C-associated disorders to include isolated neurological manifestations (Genetics in Medicine, (2021), 23, 10, (1922-1932), 10.1038/s41436-021-01232-8)

Undiagnosed Diseases Network, Oct 2021, In: Genetics in Medicine. 23, 10, p. 2016 1 p.

Research output: Contribution to journal › Comment/debate

Open Access

Enzyme Replacement Therapy for Mucopolysaccharidosis IIID using Recombinant Human α- N-Acetylglucosamine-6-Sulfatase in Neonatal Mice

Wang, F., Moen, D. R., Sauni, C., Kan, S. H., Li, S., Le, S. Q., Lomenick, B., Zhang, X., Ekins, S., Singamsetty, S., Wood, J., Dickson, P. I. & Chou, T. F., Jan 4 2021, In: Molecular Pharmaceutics. 18, 1, p. 214-227 14 p.

Research output: Contribution to journal › Article › peer-review

5 Scopus citations

Evaluation of artificial signal peptides for secretion of two lysosomal enzymes in CHO cells

Cheng, K. W., Wang, F., Lopez, G. A., Singamsetty, S., Wood, J., Dickson, P. I. & Chou, T. F., Jun 2021, In: Biochemical Journal. 478, 12, p. 2309-2319 11 p.

Research output: Contribution to journal › Article › peer-review

Open Access

2 Scopus citations

Genetic/familial high-risk assessment: Breast, ovarian, and pancreatic, version 2.2021

Daly, M. B., Pal, T., Berry, M. P., Buys, S. S., Dickson, P., Domchek, S. M., Elkhanany, A., Friedman, S., Goggins, M., Hutton, M. L., Karlan, B. Y., Khan, S., Klein, C., Kohlmann, W., Kurian, A. W., Laronga, C., Litton, J. K., Mak, J. S., Menendez, C. S., Merajver, S. D., & 14 othersNorquist, B. S., Offit, K., Pederson, H. J., Reiser, G., Senter-Jamieson, L., Shannon, K. M., Shatsky, R., Visvanathan, K., Weitzel, J. N., Wick, M. J., Wisinski, K. B., Yurgelun, M. B., Darlow, S. D. & Dwyer, M. A., Jan 2021, In: JNCCN Journal of the National Comprehensive Cancer Network. 19, 1, p. 77-102 26 p.

Research output: Contribution to journal › Review article › peer-review

Open Access

343 Scopus citations

Mucopolysaccharidoses type I gene therapy

Hurt, S. C., Dickson, P. I. & Curiel, D. T., Sep 2021, In: Journal of Inherited Metabolic Disease. 44, 5, p. 1088-1098 11 p.

Research output: Contribution to journal › Review article › peer-review

Open Access

6 Scopus citations

Neuropathology of murine Sanfilippo D syndrome

Takahashi, K., Le, S. Q., Kan, S. H., Jansen, M. J., Dickson, P. I. & Cooper, J. D., Dec 2021, In: Molecular genetics and metabolism. 134, 4, p. 323-329 7 p.

Research output: Contribution to journal › Article › peer-review

2 Scopus citations

Phenotypic expansion of CACNA1C-associated disorders to include isolated neurological manifestations

Undiagnosed Diseases Network, Oct 2021, In: Genetics in Medicine. 23, 10, p. 1922-1932 11 p.

Research output: Contribution to journal › Article › peer-review

Open Access

11 Scopus citations

2020

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

Zhang, H., Dickson, P. I., Stiles, A. R., Chen, A. H., Le, S. Q., McCaw, P., Beasley, J., Millington, D. S. & Young, S. P., Sep 2020, In: Clinica Chimica Acta. 508, p. 179-184 6 p.

Research output: Contribution to journal › Article › peer-review

4 Scopus citations

Evaluation of non-reducing end pathologic glycosaminoglycan detection method for monitoring therapeutic response to enzyme replacement therapy in human mucopolysaccharidosis I

Vera, M. U., Le, S. Q., Victoroff, A., Passage, M. B., Brown, J. R., Crawford, B. E., Polgreen, L. E., Chen, A. H. & Dickson, P. I., Feb 2020, In: Molecular genetics and metabolism. 129, 2, p. 91-97 7 p.

Research output: Contribution to journal › Article › peer-review

3 Scopus citations

Fatal COVID-19 infection in a patient with long-chain 3-hydroxyacyl-CoA dehydrogenase deficiency: A case report

Wongkittichote, P., Watson, J. R., Leonard, J. M., Toolan, E. R., Dickson, P. I. & Grange, D. K., Nov 1 2020, In: JIMD Reports. 56, 1, p. 40-45 6 p.

Research output: Contribution to journal › Article › peer-review

Open Access

8 Scopus citations

Genetic/familial high-risk assessment: Breast, ovarian, and pancreatic, version 1.2020 featured updates to the NCCN guidelines

Daly, M. B., Pilarski, R., Yurgelun, M. B., Berry, M. P., Buys, S. S., Dickson, P., Domchek, S. M., Elkhanany, A., Friedman, S., Garber, J. E., Goggins, M., Hutton, M. L., Khan, S., Klein, C., Kohlmann, W., Kurian, A. W., Laronga, C., Litton, J. K., Mak, J. S., Menendez, C. S., & 13 othersMerajver, S. D., Norquist, B. S., Offit, K., Pal, T., Pederson, H. J., Reiser, G., Shannon, K. M., Visvanathan, K., Weitzel, J. N., Wick, M. J., Wisinski, K. B., Dwyer, M. A. & Darlow, S. D., Apr 2020, In: JNCCN Journal of the National Comprehensive Cancer Network. 18, 4, p. 380-391 12 p.

Research output: Contribution to journal › Article › peer-review

Open Access

244 Scopus citations

Intrathecal enzyme replacement for cognitive decline in mucopolysaccharidosis type I, a randomized, open-label, controlled pilot study

Chen, A. H., Harmatz, P., Nestrasil, I., Eisengart, J. B., King, K. E., Rudser, K., Kaizer, A. M., Svatkova, A., Wakumoto, A., Le, S. Q., Madden, J., Young, S., Zhang, H., Polgreen, L. E. & Dickson, P. I., Feb 2020, In: Molecular genetics and metabolism. 129, 2, p. 80-90 11 p.

Research output: Contribution to journal › Article › peer-review

13 Scopus citations

Myelin and Lipid Composition of the Corpus Callosum in Mucopolysaccharidosis Type I Mice

Le, S. Q., Nestrasil, I., Kan, S. H., Egeland, M., Cooper, J. D., Elashoff, D., Guo, R., Tolar, J., Yee, J. K. & Dickson, P. I., Nov 2020, In: Lipids. 55, 6, p. 627-637 11 p.

Research output: Contribution to journal › Article › peer-review

3 Scopus citations

Newborn Screening for Mucopolysaccharidosis I: Moving Forward Learning from Experience

Clarke, L. A., Dickson, P., Ellinwood, N. M. & Klein, T. L., Dec 2020, In: International Journal of Neonatal Screening. 6, 4, 91.

Research output: Contribution to journal › Article › peer-review

Open Access

8 Scopus citations

Phenotypic expansion of KMT2D-related disorder: Beyond Kabuki syndrome

Baldridge, D., Spillmann, R. C., Wegner, D. J., Wambach, J. A., White, F. V., Sisco, K., Toler, T. L., Dickson, P. I., Cole, F. S., Shashi, V. & Grange, D. K., May 1 2020, In: American Journal of Medical Genetics, Part A. 182, 5, p. 1053-1065 13 p.

Research output: Contribution to journal › Article › peer-review

17 Scopus citations

2019

A longitudinal study of neurocognition and behavior in patients with Hurler-Scheie syndrome heterozygous for the L238Q mutation

Ahmed, A., Ou, L., Rudser, K., Shapiro, E., Eisengart, J. B., King, K., Chen, A., Dickson, P. & Whitley, C. B., Sep 2019, In: Molecular Genetics and Metabolism Reports. 20, 100484.

Research output: Contribution to journal › Article › peer-review

Open Access

4 Scopus citations

C-type natriuretic peptide analogue therapy in children with achondroplasia

Savarirayan, R., Irving, M., Bacino, C. A., Bostwick, B., Charrow, J., Cormier-Daire, V., Le Quan Sang, K. H., Dickson, P., Harmatz, P., Phillips, J., Owen, N., Cherukuri, A., Jayaram, K., Jeha, G. S., Larimore, K., Chan, M. L., Labed, A. H., Day, J. & Hoover-fong, J., Jul 4 2019, In: New England Journal of Medicine. 381, 1, p. 25-35 11 p.

Research output: Contribution to journal › Article › peer-review

Open Access

103 Scopus citations

De novo truncating variants in the intronless IRF2BPL are responsible for developmental epileptic encephalopathy

Tran Mau-Them, F., Guibaud, L., Duplomb, L., Keren, B., Lindstrom, K., Marey, I., Mochel, F., van den Boogaard, M. J., Oegema, R., Nava, C., Masurel, A., Jouan, T., Jansen, F. E., Au, M., Chen, A. H., Cho, M., Duffourd, Y., Lozier, E., Konovalov, F., Sharkov, A., & 18 othersKorostelev, S., Urteaga, B., Dickson, P., Vera, M., Martínez-Agosto, J. A., Begemann, A., Zweier, M., Schmitt-Mechelke, T., Rauch, A., Philippe, C., van Gassen, K., Nelson, S., Graham, J. M., Friedman, J., Faivre, L., Lin, H. J., Thauvin-Robinet, C. & Vitobello, A., Apr 1 2019, In: Genetics in Medicine. 21, 4, p. 1008-1014 7 p.

Research output: Contribution to journal › Article › peer-review

Open Access

30 Scopus citations

Intrathecal enzyme replacement for Hurler syndrome: biomarker association with neurocognitive outcomes

Eisengart, J. B., Pierpont, E. I., Kaizer, A. M., Rudser, K. D., King, K. E., Pasquali, M., Polgreen, L. E., Dickson, P. I., Le, S. Q., Miller, W. P., Tolar, J., Orchard, P. J. & Lund, T. C., Nov 1 2019, In: Genetics in Medicine. 21, 11, p. 2552-2560 9 p.

Research output: Contribution to journal › Article › peer-review

Open Access

20 Scopus citations

In-vivo cortical thickness estimation from high-resolution T₁w MRI scans in healthy and mucopolysaccharidosis affected dogs

Labounek, R., Mai, K., Mueller, B., Ellinwood, N. M., DIckson, P. & Nestrasil, I., Jul 2019, 2019 41st Annual International Conference of the IEEE Engineering in Medicine and Biology Society, EMBC 2019. Institute of Electrical and Electronics Engineers Inc., p. 2848-2851 4 p. 8856826. (Proceedings of the Annual International Conference of the IEEE Engineering in Medicine and Biology Society, EMBS).

Research output: Chapter in Book/Report/Conference proceeding › Conference contribution › peer-review

3 Scopus citations

2018

A Humoral Immune Response Alters the Distribution of Enzyme Replacement Therapy in Murine Mucopolysaccharidosis Type I

Le, S. Q., Kan, S. H., Clarke, D., Sanghez, V., Egeland, M., Vondrak, K. N., Doherty, T. M., Vera, M. U., Iacovino, M., Cooper, J. D., Sands, M. S. & Dickson, P. I., Mar 16 2018, In: Molecular Therapy - Methods and Clinical Development. 8, p. 42-51 10 p.

Research output: Contribution to journal › Article › peer-review

Open Access

8 Scopus citations

Community partnership in precision medicine: Themes from a community engagement conference

Jones, L., Wells, K. B., Lin, H. J., Wang, C., Alo, A. K., Williams, P., Jones, F., Dickson, P. I., Han, S., Pardo, D., Norris, K., Jones, A., Wright, A. L., Young, K. & Rotter, J. I., Sep 2018, In: Ethnicity and Disease. 28, p. 503-510 8 p.

Research output: Contribution to journal › Article › peer-review

Open Access

4 Scopus citations

Diffusion tensor imaging findings suggestive of white matter alterations in a canine model of mucopolysaccharidosis type I

Middleton, D. M., Li, J. Y., Chen, S. D., White, L. E., Dickson, P., Ellinwood, N. M. & Provenzale, J. M., Feb 1 2018, In: Neuroradiology Journal. 31, 1, p. 90-94 5 p.

Research output: Contribution to journal › Article › peer-review

7 Scopus citations

Erratum: IRF2BPL Is Associated with Neurological Phenotypes (The American Journal of Human Genetics (2018) 103(2) (245–260), (S0002929718302337) (10.1016/j.ajhg.2018.07.006))

Program for Undiagnosed Diseases (UD-PrOZA) & Undiagnosed Diseases Network, Sep 6 2018, In: American journal of human genetics. 103, 3, p. 456 1 p.

Research output: Contribution to journal › Comment/debate

Open Access

12 Scopus citations

Genetically Corrected iPSC-Derived Neural Stem Cell Grafts Deliver Enzyme Replacement to Affect CNS Disease in Sanfilippo B Mice

Clarke, D., Pearse, Y., Kan, S. H., Le, S. Q., Sanghez, V., Cooper, J. D., Dickson, P. I. & Iacovino, M., Sep 21 2018, In: Molecular Therapy - Methods and Clinical Development. 10, p. 113-127 15 p.

Research output: Contribution to journal › Article › peer-review

Open Access

9 Scopus citations

IRF2BPL Is Associated with Neurological Phenotypes

Program for Undiagnosed Diseases (UD-PrOZA) & Undiagnosed Diseases Network, Aug 2 2018, In: American journal of human genetics. 103, 2, p. 245-260 16 p.

Research output: Contribution to journal › Article › peer-review

Open Access

54 Scopus citations

Macrophage enzyme and reduced inflammation drive brain correction of mucopolysaccharidosis IIIB by stem cell gene therapy

Holley, R. J., Ellison, S. M., Fil, D., O'Leary, C., McDermott, J., Senthivel, N., Langford-Smith, A. W. W., Wilkinson, F. L., D'Souza, Z., Parker, H., Liao, A., Rowlston, S., Gleitz, H. F. E., Kan, S. H., Dickson, P. I. & Bigger, B. W., Jan 1 2018, In: Brain. 141, 1, p. 99-116 18 p.

Research output: Contribution to journal › Article › peer-review

Open Access

52 Scopus citations

2017

Diffusion tensor imaging tensor shape analysis for assessment of regional white matter differences

Middleton, D. M., Li, J. Y., Lee, H. J., Chen, S., Dickson, P. I., Ellinwood, N. M., White, L. E. & Provenzale, J. M., Aug 1 2017, In: Neuroradiology Journal. 30, 4, p. 324-329 6 p.

Research output: Contribution to journal › Article › peer-review

4 Scopus citations

Intrathecal enzyme replacement therapy reverses cognitive decline in mucopolysaccharidosis type I

Nestrasil, I., Shapiro, E., Svatkova, A., Dickson, P., Chen, A., Wakumoto, A., Ahmed, A., Stehel, E., McNeil, S., Gravance, C. & Maher, E., Mar 1 2017, In: American Journal of Medical Genetics, Part A. 173, 3, p. 780-783 4 p.

Research output: Contribution to journal › Article › peer-review

Open Access

24 Scopus citations

Novel region of interest interrogation technique for diffusion tensor imaging analysis in the canine brain

Li, J. Y., Middleton, D. M., Chen, S., White, L., Ellinwood, N. M., Dickson, P., Vite, C., Bradbury, A. & Provenzale, J. M., Aug 1 2017, In: Neuroradiology Journal. 30, 4, p. 339-346 8 p.

Research output: Contribution to journal › Article › peer-review

2 Scopus citations

Pilot study of the safety and effect of adalimumab on pain, physical function, and musculoskeletal disease in mucopolysaccharidosis types I and II

Polgreen, L. E., Kunin-Batson, A., Rudser, K., Vehe, R. K., Utz, J. J., Whitley, C. B. & Dickson, P., Mar 1 2017, In: Molecular Genetics and Metabolism Reports. 10, p. 75-80 6 p.

Research output: Contribution to journal › Article › peer-review

Open Access

15 Scopus citations

Quantitative diffusion tensor imaging analysis does not distinguish pediatric canines with mucopolysaccharidosis i from control canines

Middleton, D. M., Li, J. Y., Chen, S. D., White, L. E., Dickson, P. I., Matthew Ellinwood, N. & Provenzale, J. M., Oct 1 2017, In: Neuroradiology Journal. 30, 5, p. 454-460 7 p.

Research output: Contribution to journal › Article › peer-review

Treatment of brain disease in the mucopolysaccharidoses

Scarpa, M., Orchard, P. J., Schulz, A., Dickson, P. I., Haskins, M. E., Escolar, M. L. & Giugliani, R., Dec 2017, In: Molecular genetics and metabolism. 122, p. 25-34 10 p.

Research output: Contribution to journal › Review article › peer-review

Open Access

46 Scopus citations

2016

Behavioral deficits and cholinergic pathway abnormalities in male Sanfilippo B mice

Kan, S. H., Le, S. Q., Bui, Q. D., Benedict, B., Cushman, J., Sands, M. S. & Dickson, P. I., Oct 1 2016, In: Behavioural Brain Research. 312, p. 265-271 7 p.

Research output: Contribution to journal › Article › peer-review

4 Scopus citations

Elevated cerebral spinal fluid biomarkers in children with mucopolysaccharidosis I-H

Raymond, G. V., Pasquali, M., Polgreen, L. E., Dickson, P. I., Miller, W. P., Orchard, P. J. & Lund, T. C., Dec 2 2016, In: Scientific reports. 6, 38305.

Research output: Contribution to journal › Article › peer-review

Open Access

19 Scopus citations

Elevated TNF-α is associated with pain and physical disability in mucopolysaccharidosis types I, II, and VI

Polgreen, L. E., Vehe, R. K., Rudser, K., Kunin-Batson, A., Utz, J. J., Dickson, P., Shapiro, E. & Whitley, C. B., Apr 1 2016, In: Molecular genetics and metabolism. 117, 4, p. 427-430 4 p.

Research output: Contribution to journal › Article › peer-review

34 Scopus citations

Immune-mediated inflammation may contribute to the pathogenesis of cardiovascular disease in mucopolysaccharidosis type I

Khalid, O., Vera, M. U., Gordts, P. L., Ellinwood, N. M., Schwartz, P. H., Dickson, P. I., Esko, J. D. & Wang, R. Y., Mar 2016, In: PloS one. 11, 3, e0150850.

Research output: Contribution to journal › Article › peer-review

Open Access

17 Scopus citations

Immune response to enzyme replacement therapies in lysosomal storage diseases and the role of immune tolerance induction

Kishnani, P. S., Dickson, P. I., Muldowney, L., Lee, J. J., Rosenberg, A., Abichandani, R., Bluestone, J. A., Burton, B. K., Dewey, M., Freitas, A., Gavin, D., Griebel, D., Hogan, M., Holland, S., Tanpaiboon, P., Turka, L. A., Utz, J. J., Wang, Y. M., Whitley, C. B., Kazi, Z. B., & 1 othersPariser, A. R., Feb 1 2016, In: Molecular genetics and metabolism. 117, 2, p. 66-83 18 p.

Research output: Contribution to journal › Review article › peer-review

56 Scopus citations

2015

A novel, long-lived, and highly engraftable immunodeficient mouse model of mucopolysaccharidosis type I

Mendez, D. C., Stover, A. E., Rangel, A. D., Brick, D. J., Nethercott, H. E., Torres, M. A., Khalid, O., Wong, A. MS., Cooper, J. D., Jester, J. V., Monuki, E. S., McGuire, C., Le, S. Q., Kan, S. H., Dickson, P. I. & Schwartz, P. H., Apr 29 2015, In: Molecular Therapy - Methods and Clinical Development. 2, p. 14068 1 p.

Research output: Contribution to journal › Article › peer-review

Open Access

13 Scopus citations

Data from subjects receiving intrathecal laronidase for cervical spinal stenosis due to mucopolysaccharidosis type I

Dickson, P. I., Kaitila, I., Harmatz, P., Mlikotic, A., Chen, A. H., Victoroff, A., Passage, M. B., Madden, J., Le, S. Q. & Naylor, D. E., Dec 1 2015, In: Data in Brief. 5, p. 71-76 6 p.

Research output: Contribution to journal › Article › peer-review

Open Access

7 Scopus citations

Patricia Dickson

Research output

Search results