Dickson Lab

The Dickson laboratory studies the mucopolysaccharidosis (MPS) disorders, which are lysosomal enzyme deficiencies affecting the catabolism of glycosaminoglycans. Central nervous system manifestations include progressive intellectual disability, communicating hydrocephalus, dysmyelination, spinal cord compression, and cortical atrophy. Our lab studies cerebrospinal fluid delivery of recombinant enzymes to treat central nervous disease due to MPS, and has demonstrated biodistribution of intrathecally-delivered recombinant enzymes throughout the neuroaxis of MPS models, with correction of lysosomal storage. The laboratory also studies neuroimaging and neuropathology of white matter in MPS brain and the humoral immune responses to therapeutic enzymes. Projects range from bench to bedside including clinical trials.

Keywords: mucopolysaccharidosis, intrathecal, glycosaminoglycan, heparan sulfate, dermatan sulfate, Hurler, Scheie, Sanfilippo, CNS therapeutics, lysosomal storage

Principal Investigator

Featured publications

Impaired Mitophagy in Sanfilippo A mice Causes Hypertriglyceridemia and Brown Adipose Tissue Activation

June 21, 2022

Tillo M, Lamanna WC, Dwyer CA, Sandoval DR, Pessentheiner AR, Al-Azzam N, Sarrazin S, Gonzales JC, Kan SH, Andreyev AY, Schultheis N, Thacker BE, Glass CA, Dickson PI, Wang RY, Selleck SB, Esko JD, Gordts PLSM. Impaired Mitophagy in Sanfilippo A mice Causes Hypertriglyceridemia and Brown Adipose Tissue Activation. J Biol Chem. 2022 Jun 21:102159. doi: 10.1016/j.jbc.2022.102159. Epub ahead of print. PMID: 35750212.

Diagnostic yield of exome sequencing in congenital vertical talus

June 1, 2022

Tayebi N, Charng WL, Dickson PI, Dobbs MB, Gurnett CA. Diagnostic yield of exome sequencing in congenital vertical talus. Eur J Med Genet. 2022 Jun;65(6):104514. doi: 10.1016/j.ejmg.2022.104514. Epub 2022 Apr 27. PMID: 35487415.

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

December 24, 2021

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. 2021 Dec 24;3(1):100081. doi: 10.1016/j.xhgg.2021.100081. PMID: 35047865; PMCID: PMC8756500.

Neuropathology of murine Sanfilippo D syndrome

December 1, 2021

Takahashi K, Le SQ, Kan SH, Jansen MJ, Dickson PI, Cooper JD. Neuropathology of murine Sanfilippo D syndrome. Mol Genet Metab. 2021 Dec;134(4):323-329. doi: 10.1016/j.ymgme.2021.11.010. Epub 2021 Nov 24. PMID: 34844863.

Neuropathology of murine Sanfilippo D syndrome

September 1, 2021

Hurt SC, Dickson PI, Curiel DT. Mucopolysaccharidoses type I gene therapy. J Inherit Metab Dis. 2021 Sep;44(5):1088-1098. doi: 10.1002/jimd.12414. Epub 2021 Jul 9. PMID: 34189746; PMCID: PMC8525653.

Recent publications

• Characterization of early markers of disease in the mouse model of mucopolysaccharidosis IIIB
  December 1, 2024
  Characterization of early markers of disease in the mouse model of mucopolysaccharidosis IIIBMcCullough, K. B., Titus, A., Reardon, K., Conyers, S., Dougherty, J. D., Ge, X., Garbow, J. R., Dickson, P., Yuede, C. M. & Maloney, S. E., Dec 2024, In: Journal of neurodevelopmental disorders. 16, 1, 16.Research output: Contribution to journal › Article › peer-review
• LUSTR: a new customizable tool for calling genome-wide germline and somatic short tandem repeat variants
  December 1, 2024
  LUSTR: a new customizable tool for calling genome-wide germline and somatic short tandem repeat variantsUndiagnosed Diseases Network, Lu, J., Toro, C., Adams, D. R., Moreno, C. A. M., Lee, W. P., Leung, Y. Y., Harms, M. B., Vardarajan, B., Heinzen, E. L., Acosta, M. T., Adam, M., Izumi, K., Alvarez, R. L., Alvey, J., Amendola, L., Andrews, A., Ashley, E. A., Bacino, C. A. & Bademci, G. & 181 others, Balasubramanyam, A., Baldridge, D., Bale, J., Bamshad, M., Barbouth, D., Bayrak-Toydemir, P., Beck, A., Beggs, A. H., Behrens, E., Bejerano, G., Bellen, H. J., Bennett, J., Berg-Rood, B., Bernstein, J. […]
• Using a new analytic approach for genotyping and phenotyping chromosome 9p deletion syndrome
  September 1, 2024
  Using a new analytic approach for genotyping and phenotyping chromosome 9p deletion syndromeStarosta, R. T., Jensen, N., Couteranis, S., Slaugh, R., Easterlin, D., Tate, V., Sams, E. I., Valle, K., Akinwe, T., Hou, Y. C. C., Turner, T. N., Cole, F. S., Milbrandt, J. & Dickson, P., Sep 2024, In: European Journal of Human Genetics. 32, 9, p. 1095-1105 11 p.Research output: Contribution to journal › Article › peer-review
• Community consensus for Heparan sulfate as a biomarker to support accelerated approval in Neuronopathic Mucopolysaccharidoses
  August 1, 2024
  Community consensus for Heparan sulfate as a biomarker to support accelerated approval in Neuronopathic MucopolysaccharidosesMuenzer, J., Ho, C., Lau, H., Dant, M., Fuller, M., Boulos, N., Dickson, P., Ellinwood, N. M., Jones, S. A., Zanelli, E. & O'Neill, C., Aug 2024, In: Molecular genetics and metabolism. 142, 4, 108535.Research output: Contribution to journal › Review article › peer-review
• D-mannose as a new therapy for fucokinase deficiency-related congenital disorder of glycosylation (FCSK-CDG)
  June 1, 2024
  D-mannose as a new therapy for fucokinase deficiency-related congenital disorder of glycosylation (FCSK-CDG)Starosta, R. T., Lee, A. J., Toolan, E. R., He, M., Wongkittichote, P., Daniel, E. J. P., Radenkovic, S., Budhraja, R., Pandey, A., Sharma, J., Morava, E., Nguyen, H. & Dickson, P. I., Jun 2024, In: Molecular genetics and metabolism. 142, 2, 108488.Research output: Contribution to journal › Review article › peer-review

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