Research
Publications & Contributions
Ongoing Research
Elucidating translational aberrations resulting from IGHMBP2 mutations
Sarah Hurt, Gangadhar Vadla, Christian Lorson, and Monique Lorson
Target: Molecular Therapy
Understanding the function of the IGHMBP2-ABT1 interaction and its role in SMARD1/CMT2S
Sarah Hurt, Gangadhar Vadla, Christian Lorson, and Monique Lorson
Target: Cell
Publications
Subtle cellular phenotypes inform pathological and benign genetic mutants in the Iduronidase-2-Sulfatase Gene
BioRxiv, April 2025
Authors: Viswanathan A, Elia S, Le SQ, Hurt S, Balraj Doray, Jason Waligorski, Kylan Kelley, William Buchser, Patricia Dickson
As with most enzyme-storage disorders, DNA testing at an early age is critical for identifying genetic variants and their impact on disease burden. Still, most variants in genes such as Iduronate-2-sulfatase (IDS) are Variants of Uncertain Significance (VUS). In patients presenting with Hunter Syndrome, clinical testing for IDS enzyme activity has been the mainstay to determine whether a variant is likely damaging. This study developed an image-based assay using genome-engineered cells with IDS mutations to identify if a specific mutation causes lysosomal and membrane disruptions that characterize the disease. Overall, these cell-based lysosomal and membrane phenotypes may be key to quickly and accurately profiling clinical variants in the IDS gene.
Specific antibodies to recombinant human alpha-L-iduronidase prevent disease correction in cortical bone in mucopolysaccharidosis I mice
Molecular Therapy: Methods & Clinical Development, March 2025
Authors: Hurt SC, Le SQ, Kan S, Bui QD, Brodt MD, Dickson PI
Mucopolysaccharidosis I (MPS I) is a lysosomal storage disorder resulting from a deficiency in the enzyme α-L-iduronidase (IDUA). The study hypothesized that the failure of ERT to address skeletal disease in MPS I might be due to the development of anti-IDUA antibodies. The findings indicated that mice receiving ERT along with IRIG exhibited less disease correction in their cortical bone compared to those receiving ERT alone. This suggests that the development of anti-IDUA antibodies can impair the effectiveness of ERT by altering the tissue distribution of the enzyme, particularly in skeletal disease.
Combining angiotensin receptor blockade and enzyme replacement therapy for vascular disease in mucopolysaccharidosis type I
Molecular Genetics and Metabolism Reports, December 2023
Authors: Hurt SC, Vera MU, Le SQ, Kan S, Bui Q, Dickson PI
Vascular involvement in the genetic disorder mucopolysaccharidosis type I (MPS I) has features of atherosclerotic disease near branch points of arterial vasculature, such as intimal thickening with disruption of the internal elastic lamina, and proliferation of macrophages and myofibroblasts. Inflammatory pathways are implicated in the pathogenesis of vascular disease in MPS I animal models, evidenced by cytokines like CD18 and TGF-β within arterial plaques. Here, we combined losartan with the standard therapy for MPS I, enzyme replacement therapy (ERT), to measure effects on cytokines in serum and aortic vasculature. The observations suggest that losartan may impact inflammatory cascades due to MPS I and may also blunt inflammation in combination with ERT.
Mucopolysaccharidoses type I gene therapy
Journal of Inherited Metabolic Disease, September 2021
Authors: Hurt SC, Dickson PI, Curiel DT
Mucopolysaccharidoses type I (MPS I) is an inherited metabolic disease characterized by a malfunction of the α-l-iduronidase (IDUA) enzyme leading to the storage of glycosaminoglycans in the lysosomes. This disease has longtime been studied as a therapeutic target for those studying gene therapy and many studies have been done using various vectors to deliver the IDUA gene for corrective treatment. Additionally, the recent modifications to adenoviral vectors leading them to target the vascular endothelium minimizing the risk of hepatotoxicity could lead to them being a viable option for MPS I gene therapy when coupled with gene editing technologies like CRISPR/Cas9.
Transcriptome and Organellar Genome Sequencing Elucidates the Origin and Diversification of Allotetraploid Brassica napus
Nature Communications, June 2019
Authors: An H, Qi X, Gaynor ML, Hao Y, Gebken SC, et al.
Brassica napus, an allotetraploid crop, is hypothesized to be a hybrid from unknown varieties of Brassica rapa and Brassica oleracea. Despite the economic importance of B. napus, much is unresolved regarding its phylogenomic relationships, genetic structure, and diversification. Here we conduct a comprehensive study among diverse accessions from 183 B. napus, 112 B. rapa, and 62 B. oleracea and its wild relatives. Using RNA-seq of B. napus accessions, we define the genetic diversity and sub-genome variance of six genetic clusters. This study highlights the complex origin and evolution of B. napus providing insights that can further facilitate B. napus breeding and germplasm preservation.
Topological Data Analysis as a Morphometric Method: Using Persistent Homology to Demarcate a Leaf Morphospace
Frontiers in Plant Science, April 2018
Authors: Li M, An H, Angelovici R... Gebken S, et al.
Current morphometric methods that comprehensively measure shape cannot compare the disparate leaf shapes found in seed plants and are sensitive to processing artifacts. We explore the use of persistent homology, a topological method applied as a filtration across simplicial complexes, to overcome these limitations. We apply the method to the analysis of 182,707 leaves, both published and unpublished, representing 141 plant families collected from 75 sites throughout the world. The application of a persistent homology method, using topological features, to measure leaf shape allows for a unified morphometric framework to measure plant form, including shapes, textures, patterns, and branching architectures.
Fellowships & Grants
Trainee Professional Development Award
November 2025
Society for Neuroscience
Award recognizes undergraduate and graduate students and post baccalaureate and postdoctoral scholars who demonstrate scientific merit and excellence in research. Covers registration and travel stipend.
Spinal Cord Injury/Disease Research Program Grant
April 2025
University of Missouri System
Amount: $100,000
One year of support for research and salary to study translational aberrations because of IGHMBP2 mutations.
Young Investigator Award
February 2024
WORLD Symposium
Chosen as one of 10 investigators to be honored. Covers registration and boarding.
Ruth L. Kirschstein National Research Service Award Individual Predoctoral Fellowship (F31)
April 2023 – May 2024
National Institute of Health: NHLBI
Amount: $27,000
Competitive grant funding two years of research and living expenses.
Precision Medicine Graduate Pathway
July 2021 – May 2023
Washington University in St. Louis
Emphasis in precision medicine upon completion of coursework and clinical partnerships.
Graduate Research Fellowship Program (GRFP)
April 2019 – May 2022
National Science Foundation
Amount: $37,000/year
Competitive fellowship funding three years of financial support.
Travel Grant to Botany 2018
July 2018
American Society of Plant Taxonomists
Chosen as one of 45 students to receive travel support.
Grant in Aid of Research
May 2017 – May 2018
Sigma Xi
Amount: $750
Competitive grant to support research projects.
Ronald E. McNair Scholars
May 2017 – May 2018
University of Missouri – Columbia
Competitive program encouraging minority and first-generation undergraduate students to pursue graduate studies.
Health Careers Opportunity Program (HCOP) Summer Fellowship
May 2017 – August 2017
Johns Hopkins University
Competitive summer fellowship for economically or educationally disadvantaged undergraduate students.
Research Experience for Undergraduates (REU)
May 2016 – August 2016
National Science Foundation
Competitive summer program pairing biological and computational students.
Missouri EPSCoR Summer Fellowship
May 2016 – August 2016
University of Missouri – Columbia
Initiative to support plant centric research in Missouri.
Freshman Research in Plant Science
September 2015 – May 2015
University of Missouri – Columbia
Chosen as one of ten freshmen to be financially supported to conduct research.
Presentations
Oral Presentations
Adenoviral gene therapy for mucopolysaccharidosis type I using the endothelium as a cellular source
February 2024
Rare Disease Day Symposium, St. Louis, MO
Anti-IDUA IgG alters cortical bone structure of mucopolysaccharidoses type I mice treated with intravenous enzyme replacement therapy
February 2024
WORLD Symposium 2024, San Diego, CA
Sarah Hurt, Steven Le, Shih-hsin Kan, Michael Brodt, Patricia Dickson
Adenoviral gene therapy for mucopolysaccharidosis type I using the endothelium as a cellular source
September 2023
Precision Medicine Pathway Retreat, St. Louis, MO
Adenoviral gene therapy for mucopolysaccharidosis type I using the endothelium as a cellular source
May 2023
Gordon Research Seminar on Lysosomal Diseases, Barcelona, Spain
Sarah Hurt, Steven Le, Samir Mendonça, Al Sorensen, Patricia Dickson, David Curiel
Adenoviral Gene Therapy for Mucopolysaccharidoses Type I Using the Endothelium as a Cellular Source
January 2023
Cardiovascular Trainee Research Seminar, St. Louis, MO
Targeted Genome Engineering Using CRISPR/Cas9 in Brassica oleracea
April 2018
29th Annual MU McNair Conference, Columbia, MO
Sarah Gebken, Makenzie E. Mabry, and J. Chris Pires
Poster Presentations
Understanding the function of the IGHMBP2-ABT1 interaction and its role in SMARD1/CMT2S
November 2025
Society for Neuroscience 2025, San Diego, CA
Sarah Hurt, Gangadhar Vadla, Kamlendra Singh, Christian Lorson, Monique Lorson
Understanding the function of the IGHMBP2-ABT1 interaction and its role in SMARD1/CMT2S
May 2025
CVM Research Day, Columbia, MO
Sarah Hurt, Gangadhar Vadla, Monique Lorson, Christian Lorson
Understanding the function of the IGHMBP2-ABT1 interaction and its role in SMARD1/CMT2S
April 2025
MU Show Me Research Week, Columbia, MO
Sarah Hurt, Gangadhar Vadla, Monique Lorson, Christian Lorson
The contribution and therapeutic implications of IGHMBP2 mutations on IGHMBP2 biochemical activity and ABT1 association
February 2025
Rare Disease Day Symposium, St. Louis, MO
Sarah Hurt, Gangadhar Vadla, Monique Lorson, Christian Lorson
Specific antibodies to recombinant human α-L-Iduronidase prevent disease correction in cortical bone in mucopolysaccharidosis type I
May 2024
Musculoskeletal Research Symposium, St. Louis, MO
Sarah Hurt, Steven Le, Shih-hsin Kan, Michael Brodt, and Patricia Dickson
Anti-IDUA IgG alters cortical bone structure of mucopolysaccharidoses type I mice treated with intravenous enzyme replacement therapy
February 2024
Rare Disease Day Symposium, St. Louis, MO
Sarah Hurt, Steven Le, Shih-hsin Kan, Michael Brodt, and Patricia Dickson
Anti-IDUA IgG alters cortical bone structure of mucopolysaccharidoses type I mice treated with intravenous enzyme replacement therapy
February 2024
WORLD Symposium 2024, San Diego, CA
Sarah Hurt, Steven Le, Shih-hsin Kan, Michael Brodt, and Patricia Dickson
Elucidating expression of a corrective enzyme for Mucopolysaccharidosis type I through administration of an adenoviral vector targeted to endothelial cells
February 2024
WORLD Symposium 2024, San Diego, CA
Sarah Hurt, Steven Le, Samir Mendonca, Al Sorensen, David T. Curiel, and Patricia Dickson
Elucidating expression of a corrective enzyme for Mucopolysaccharidosis type I through administration of an adenoviral vector targeted to endothelial cells
November 2023
Ellis Fischel Cancer Center Basic Science Retreat, Innsbrook, MO
Sarah Hurt, Steven Le, Samir Mendonca, Al Sorensen, Patricia Dickson, and David T. Curiel
Elucidating expression of a corrective enzyme for MPS I through administration of an adenoviral vector targeted to endothelial cells
May 2023
Gordon Research Conference on Lysosomal Diseases, Barcelona, Spain
Sarah Hurt, Steven Le, Samir Mendonça, Al Sorensen, Patricia Dickson, and David T. Curiel
Anti-IDUA IgG alters cortical bone structure of mucopolysaccharidosis type I mice treated with intravenous enzyme replacement therapy
May 2023
Musculoskeletal Research Center Symposium 2023, St. Louis, MO
Sarah Hurt, Steven Le, Shih-hsin Kan, Michael Brodt, and Patricia Dickson
Elucidating expression of a corrective enzyme for MPS I through administration of an adenoviral vector targeted to endothelial cells
February 2023
Rare Disease Day Symposium, St. Louis, MO
Sarah Hurt, Steven Le, Samir Mendonça, Al Sorensen, Patricia Dickson, and David T. Curiel
Elucidating expression of a corrective enzyme for MPS I through administration of an adenoviral vector targeted to endothelial cells
February 2023
WORLD Symposium, Orlando, FL
Sarah Hurt, Steven Le, Samir Mendonça, Al Sorensen, Patricia Dickson, and David T. Curiel
Elucidating expression of a corrective enzyme for MPS I through administration of a modified adenoviral vector
September 2022
ISSCR/ASGCT Emerging Therapies, Madison, WI
Sarah Hurt, Steven Le, Samir Mendonça, Al Sorensen, Patricia Dickson, David T. Curiel
An adenoviral mediated gene therapy for mucopolysaccharidosestype I
May 2022
Hope Center for Neurological Disorders Annual Retreat, St. Louis, MO
Sarah Hurt, Steven Le, Samir Mendonça, Paul Boucher, Patricia Dickson, David T. Curiel
An Adenoviral Mediated Gene Therapy for Mucopolysaccharidoses Type I
February 2022
WORLD Symposium, San Diego, CA
Sarah Hurt, Steven Le, Samir Mendonça, Patricia Dickson, and David T. Curiel
Targeted Genome Engineering Using CRISPR/Cas9 in Brassica oleracea
April 2019
MU Undergraduate Research Forum, Columbia, MO
Sarah Gebken, Makenzie E. Mabry, and J. Chris Pires
Targeted Genome Engineering Using CRISPR/Cas9 in Brassica oleracea
April 2019
MU Life Sciences Week, Columbia, MO
Sarah Gebken, Makenzie E. Mabry, and J. Chris Pires
Designing a Detection Device for Viruses
December 2018
Capstone Presentation 2018, Columbia, MO
Ian Heck, Sarah Gebken, Clare Kercher, Robert Rolette and Liqun Gu
Targeted Genome Engineering Using CRISPR/Cas9 in Brassica oleracea
July 2018
Botany 2018, Rochester, MN
Sarah Gebken, Makenzie E. Mabry, and J. Chris Pires
Targeted Genome Engineering Using CRISPR/Cas9 in Brassica oleracea
April 2018
29th Annual MU McNair Conference, Columbia, MO
Sarah Gebken, Makenzie E. Mabry, and J. Chris Pires
Preparing a Genetic Engineering Technique to Bring into the Brassica Crop Plants
April 2018
Council of Undergraduate Research Posters on the Hill, Washington, D.C.
Sarah Gebken, Makenzie E. Mabry, Ruthie Angelovici, and J. Chris Pires
Targeted Genome Engineering Using CRISPR/Cas9 in Brassica oleracea
April 2018
MU Life Sciences Week, Columbia, MO
Sarah Gebken, Makenzie E. Mabry, and J. Chris Pires
Mechanism of Tau Pathological Spreading in a New Mouse Model of Alzheimer’s Disease
September 2017
Beyond the Columns Poster Presentation, Columbia, MO
Sarah Gebken, Kerstin Braunstein, Tong Li, and Philip Wong
Mechanism of Tau Pathological Spreading in a New Mouse Model of Alzheimer’s Disease
July 2017
Johns Hopkins Career, Academic, and Research Experiences for Students (CARES) Symposium, Baltimore, MD
Sarah Gebken, Kerstin Braunstein, Tong Li, and Philip Wong
Implementing CRISPR in Arabidopsis thaliana
May 2017
MU Life Sciences Week, Columbia, MO
Sarah Gebken, Makenzie E. Mabry, J. Chris Pires, Ruthie Angelovici
Preparing a Genetic Engineering Technique to Bring into the Brassica Crop Plants
May 2017
Undergraduate Research Day at the Capitol, Jefferson City, MO
Sarah Gebken, Makenzie E. Mabry, J. Chris Pires, Ruthie Angelovici
Implementing CRISPR in Arabidopsis thaliana
July 2016
Summer Undergraduate Research Forum, Columbia, MO
Sarah Gebken, Makenzie E. Mabry, J. Chris Pires, Ruthie Angelovici