The Jackson Laboratory Nabs $1.8M Grant from The Michael J. Fox Foundation to Advance Induced Pluripotent Stem Cells for Parkinson’s
JAX will use the funding to study how a genetic variant affects the disease and potentially standardize and characterize human cell models for broader research.
Key Points
- The Jackson Laboratory has received a $1.8 million grant from The Michael J. Fox Foundation to support Parkinson’s disease research through the JAX–NYSCF Collaborative.
- The project will build standardized human cell models using induced pluripotent stem cell (iPSC) technology to study GBA1, a major genetic risk factor for Parkinson’s disease.
- The work includes independent validation by McGill University’s Early Drug Discovery Unit and is intended to generate research methods, model systems, and open datasets for the field.
The Jackson Laboratory has received a $1.8 million grant from The Michael J. Fox Foundation for Parkinson’s Research to study the disease through the JAX–NYSCF Collaborative (a cell therapy partnership between The Jackson Laboratory and the New York Stem Cell Foundation). The project focuses on a persistent issue: the limited availability of standardized, scalable human cell model systems for studying Parkinson’s disease biology and supporting therapeutic research.
The work will center on GBA1, the most commonly known genetic risk factor for Parkinson’s disease. GBA1 encodes an enzyme involved in breaking down certain lipids as part of the cell’s recycling system. People who carry GBA1 variants have a higher risk of developing Parkinson’s disease and may experience earlier onset and faster progression, but the mechanisms linking those variants to disease remain unclear.
“One of MJFF’s goals is to support the development of scalable and reproducible research reagents, models, and services that can help advance understanding of Parkinson’s disease biology across the field,” said Nicole Polinski, director of research resources at MJFF. “GBA1 remains one of the most important genetic risk factors linked to Parkinson’s disease, and efforts like this aim to standardize and characterize accessible human cell modeling systems that may help researchers more consistently investigate disease mechanisms and evaluate potential therapeutic approaches.”
iPSC-derived brain cells
Researchers will use induced pluripotent stem cell (iPSC) models carrying GBA1 variants and develop methods to study how the gene functions in cells. The team plans to differentiate those stem cells into brain cell types relevant to Parkinson’s disease, including:
- Microglia, the brain’s resident immune cells
- A specific population of neurons that is preferentially lost as Parkinson’s disease progresses
The group will then apply biochemical and microscopy assays to identify disrupted GBA1 function and examine effects on the cell’s recycling system. The project is expected to produce standardized research methods, cell modeling systems, and open datasets for the broader research community.
“Many genetic variants associated with Parkinson’s disease have been identified, but for most of them we still don’t understand their functional roles or how they contribute to disease risk,” said Stefan Semrau, co-director of the JAX–NYSCF Collaborative, and principal investigator for the project. “Our goal is to standardize and characterize human cell modeling systems that directly link genetic changes to what happens in the cell, and to do so in a way that researchers and industry partners can readily use to develop new approaches for studying Parkinson’s disease and evaluating potential therapies.”
More on the JAX-NYSCF partnership
The JAX–NYSCF Collaborative combines JAX’s genetics and genomics capabilities with NYSCF’s stem cell and automation technologies. The integrated platform is intended to support biologically relevant models that are also standardized, scalable, and broadly useful to Parkinson’s disease researchers.
The project will also involve the Early Drug Discovery Unit at the Montreal Neurological Institute at McGill University. That group will independently validate the models and assays to test reproducibility across laboratories.
More information about The Jackson Laboratory is available at jax.org.
(Image credit The Jackson Laboratory)
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