Primary Research Focus
The Weimer Lab focuses on understanding mechanisms of polarized signaling involved in development of the cerebral cortex. These processes are known to regulate the proliferation and placement of neurons, formation of axons in differentiating cells and long term maintenance and trafficking within these same processes.
The Weimer Lab draws on expertise in cell and molecular biology, genetics and behavioral neuroscience to answer questions that bridge from basic mechanisms to translational and clinical approaches to treat human disease. These questions answer how and why various scaffolding or “signaling complex” form in neurons, how disruption in these complexes can contribute to human disease and determine if these complexes may serve as druggable targets.
Specifically, the team explores a number of mechanisms involved in neural stem cell proliferation, neuronal polarity and axonal outgrowth and trafficking with the hope of advancing our knowledge of neurobiology and aiding in the future treatment of rare neural developmental disorders. This includes projects focused on neuropediatric disorders including Batten disease, cortical malformations and neurofibromatosis type I.
About the Weimer Lab
Lab Projects and News
Engineering Superior Gene Therapies for Neurological Disease
In collaboration with Amicus Therapeutics, the Weimer lab screens several second-generation gene therapies that improve viral vector transduction and secretion in several lysosomal storage and metabolic disorders. With these improvements in technology, gene therapies can become safer, more therapeutic to the patient, and more cost-effective.
Discovering Novel and Shared Interactors of Batten Disease Proteins
It remains unclear what role Batten disease proteins have in neurons and whether these proteins share pathways with one another. This project uses in vitro and in vivo BioID proximity labeling to identify novel protein interactors of CLN3, CLN6, and CLN8, which will inform understanding of CLN biology and potential therapeutic targets.
Identifying Molecular Therapies for Multiple Forms of Batten Disease
This project seeks to identify small molecule therapies for CLN1, CLN2, CLN3, CLN6, and CLN8 disease using a cutting edge high-content screening pipeline. After identifying new lead drugs in cell models, we perform comprehensive screens in mouse models using a combination of in-life biomarkers, histopathology, neurobehavior, and kinematic gait analysis.
Novel Mechanisms for Distal Transport in Developing and Mature Neurons
This study will expand our understanding of CLN6’s contribution to crucial cellular processes and start to unravel the biological significance of the CCK complex in developing and mature neurons, as well as its role and the role of intracellular trafficking in neurological disorders such as the NCLs.
The NF-1 Porcine Model Collaborative Research ProgramIn collaboration with NF1 investigators. we are studying a newly developed porcine model of NF1 to better understand the neurobiology of neurofibromin loss in the CNS. A longitudinal pig study examining histopathology, neurobehavior, and neurotransmitters will allow a better understanding of the molecular mechanisms of NF1.
Batten Disease and Gene Therapy
Jill Weimer, PhD, discussed gene therapy and its potential as a treatment for Batten disease with the American Society of Gene & Cell Therapy. Watch the video to learn more.
Searching for an Effective Treatment for CLN6-Batten Disease
This project brings together a team exploring small molecules/repurposed FDA approved drugs, gene therapy, cellular therapy and gene editing as possibly treatments for CLN6-Batten disease. The Weimer Lab serves as the central screening and phenotyping facility for all human patient cell line and mouse model studies.
Characterizing Large Animal Models of Neurological Disease
In Collaboration with Precigen Exemplar, we have established porcine models of CLN2 disease, CLN3 disease, and neurofibromatosis type 1 (NF1). These pigs are being characterized with histopathology, neuroimaging, and neurobehavior metrics, and are being used to test various therapeutics in preclinical drug testing.
Developing Translatable Biomarkers for Batten Disease
Finding sensitive outcome measures to use in clinical trials for Batten Disease has been challenging. We are performing deep profiling of blood samples, biopotential readouts, and neuroimaging from several Batten disease mouse and pig models to identify new markers that can be used to track disease progression and responses to experimental therapies.
Meet the Weimer Team
Jacob Cain, PhD
Dr. Jacob Cain joined the Weimer Lab in 2014 as a postdoctoral fellow. He works on testing the efficacy of AAV9 mediated gene therapies to treat mouse models of Batten disease.
Dr. Cain holds a Ph.D. in biology from the University of North Dakota.
Vicki Swier, PhD
Dr. Vicki Swier joined the Weimer lab in 2017. She holds a Ph.D. from Texas Tech University and completed a postdoctoral fellowship at Creighton University.
Clarissa Booth, MS
Clarissa Booth joined the Weimer lab in 2018 to research the efficacy of various treatments for different pediatric diseases. Her primary focus is with in vivo work. Booth holds a master’s degree in biology with a neuroscience specialization from the University of South Dakota.
Katie White, BS
Katie White joined the Weimer Lab in 2014 to assist in the characterization of novel miniswine models. She has since worked on several projects involving gene and small molecule therapies for Batten disease, contributing to preclinical data for 2 clinical trials and 15 publications during her time in the lab. White holds a BS in Animal Sciences from the University of Missouri and is pursuing her MPH in Administration and Policy from the University of Minnesota.
Joelle Anderson joined the Weimer Lab in 2020. She is working on the mouse behavior and cell culture teams on a number of drug discovery projects.
Anderson holds a bachelor's degree in biomedical sciences from MSU Mankato.
Jon Brudvig, PhD
Dr. Jon Brudvig has worked with the Weimer Lab since 2014. Jon leads a variety of basic bio and preclinical initiatives in the lab, with the overarching goal of developing a better understanding of Batten Disease biology leading to novel therapeutic strategies. Jon holds a PhD in Basic Biomedical Sciences from the University of South Dakota.
Brandon Meyerink, BS
Brandon Meyerink joined the Weimer Lab in 2017. He studies efficacy screening of gene therapy in mouse models of Batten disease and works on in vivo screening of small molecule therapies for lysosomal storage disorder.
Meyerink holds a bachelor’s degree from the University of South Dakota.
Melissa Pratt, BS
Research Project Manager
Melissa Pratt joined the Weimer Lab in 2018, where she oversees work on a variety of projects, including in vivo small molecule drug studies for Batten disease. Pratt holds a bachelor’s degree in biology from the University of South Dakota.
Kaylie Timm, BS
Senior Research Specialist
Kaylie Timm joined the Weimer Lab in 2018 to provide experimental support to several of the team’s projects.
Timm holds a bachelor’s degree in criminal justice from the University of South Dakota.
Tyler Johnson, PhD
Dr. Tyler Johnson joined the Weimer team as a postdoctoral fellow in September 2016 after completing his Ph.D. at University of Nebraska-Lincoln. Dr. Johnson works on preclinical therapeutic development for the multiple subtypes of Batten disease, characterizing our porcine models of CLN3 and CLN2 Batten disease and investigating/developing comprehensive Batten disease biomarker panels for tracking disease state and progression.
Steven Ortmeier, MS
Senior Research Specialist
After a dozen years of part-time lab work at Sanford Research while teaching high school Biology as a career, Steve joined the Weimer Lab full-time in 2021 (retiring from education). He currently focuses his skills on immunohistochemistry, image analysis, and data summarization for various CLN2 projects. Steve holds a master’s degree in Biology from South Dakota State University.
Taylor is a current student at Augustana University working towards a Bachelor’s degree in Biology, with an Allied Health emphasis. She joined the Weimer lab in the beginning of 2021 and primarily assists with immunohistochemistry work for a variety of Batten disease projects.
Lauren Kelly joined the Weimer Lab as an undergraduate research assistant in 2020. She is currently pursuing a degree in biochemistry at Augustana University. In the lab, she does behavior and IHC support.
Associate Research Specialist
Martee joined the Weimer Lab in 2020. In the lab, she helps with the mouse colony management and drug deliveries to the study animals.
Larson holds a bachelor's degree in Microbiology from SDSU.
Hannah joined the Weimer Lab in 2020. She helps oversee much of the lab's biochemistry work and Batten disease drug studies.
Leppert holds a bachelor's degree in Chemistry and Applied Biological Sciences from South Dakota School of Mines and Technology.
Lauren joined the Weimer Lab in 2020. She works as part of the lab's animal phenotyping team, helping with drug deliveries, small animal surgeries and tissue processing.
Lindsey holds a bachelor's degree in human biology from SDSU.
Associate Research Specialist
Kelsey joined the Weimer Lab in 2020. In the lab, she helps with studies that model disease phenotypes in small animal models of Batten Disease and with general animal colony husbandry and maintenance.
Schecher holds a bachelor's degree in biology from South Dakota School of Mines and Technology.
Senior Research Specialist
Mitchell joined the Weimer Lab in 2019. In the lab, he assists with molecular work and Batten disease drug studies.
Rechtzigel holds a bachelor's degree in biology from Augustana University.
Associate Research SpecialistEmily graduated from Black Hills State University with degrees in Biology and Psychology. During her undergraduate years, she did research in bacterial characterization and was involved in molecular genetics laboratory work as well. Emily joined the Weimer lab in August 2021. She works with our animal phenotyping team in collecting and processing tissue samples and other mouse colony work.
Associate Research SpecialistTesla graduated from USF in 2020 with her Bachelors of Science in Biology and Chemistry. While there, she did undergraduate research on molecularly imprinted polymers for dopamine, as well as blood platelet interactions with ovarian cancer. Tesla joined the Weimer lab in October 2021 and assists with genotyping and mouse colony work.