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

• Antibodies to recombinant human alpha-L-iduronidase prevent disease correction in cortical bone in MPS I mice
  March 13, 2025
  Antibodies to recombinant human alpha-L-iduronidase prevent disease correction in cortical bone in MPS I miceHurt, S. C., Le, S. Q., Kan, S. H., Bui, Q. D., Brodt, M. D. & Dickson, P. I., Mar 13 2025, In: Molecular Therapy – Methods and Clinical Development. 33, 1, 101405.Research output: Contribution to journal › Article › peer-review
• Gene therapy ameliorates bowel dysmotility and enteric neuron degeneration and extends survival in lysosomal storage disorder mouse models
  January 15, 2025
  Gene therapy ameliorates bowel dysmotility and enteric neuron degeneration and extends survival in lysosomal storage disorder mouse modelsZiółkowska, E. A., Jansen, M. J., Williams, L. L., Wang, S. H., Eultgen, E. M., Takahashi, K., Le, S. Q., Nelvagal, H. R., Sharma, J., Sardiello, M., DeBosch, B. J., Dickson, P. I., Anderson, J. B., Sax, S. E., Wright, C. M., Bradley, R. P., Whiteman, I. T., Makita, T., Grider, J. R. & Sands, M. S. & 2 others, Heuckeroth, R. O. & Cooper, J. D., Jan 15 2025, In: Science translational medicine. 17, 781, eadj1445.Research output: Contribution to journal › Article […]
• Single-Gene Deletion of FGF3 in a Patient With Features of 11q13 Microdeletion Syndrome
  January 1, 2025
  Single-Gene Deletion of FGF3 in a Patient With Features of 11q13 Microdeletion SyndromeRahi, H., Dickson, P. I., Toler, T. L., Corliss, M. M. & Cao, Y., 2025, (Accepted/In press) In: American Journal of Medical Genetics, Part A.Research output: Contribution to journal › Article › peer-review
• Atypical free sialic acid storage disorder associated with tissue specific mosaicism of SLC17A5
  January 1, 2025
  Atypical free sialic acid storage disorder associated with tissue specific mosaicism of SLC17A5Undiagnosed Diseases Network, Jan 2025, In: Molecular genetics and metabolism. 144, 1, 109004.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. […]

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