JAX-NYSCF Stem Cell Toolkit for Parkinson’s Disease
Backed by $6.4M in grants, the team will create and validate more than 20 patient-derived stem cell lines, then share the cells, protocols and data publicly.
Key Points
- JAX-NYSCF is developing a stem cell toolkit to study the genetic basis of Parkinson’s disease, with a focus on the GBA1 gene.
- The work is backed by $6.4 million in grant funding from The Michael J. Fox Foundation for Parkinson’s Research and the Aligning Science Across Parkinson Collaborative Research Network.
- Researchers plan to create and validate more than 20 patient-derived human-induced pluripotent stem cell (iPSC) lines and share the cell lines, protocols, and data through public repositories.
A collaboration of JAX scientists in Connecticut and New York is working to better understand the genetic roots of Parkinson’s disease by developing and sharing scalable human stem cell models. Through a new stem cell toolkit, researchers at The Jackson Laboratory New York Stem Cell Foundation Collaborative (JAX-NYSCF) aim to create, validate, and openly distribute stem cell models and data related to Parkinson’s disease.
The research is funded by a $1.8 million grant from The Michael J. Fox Foundation for Parkinson’s Research, which will also administer a $4.6 million grant from the Aligning Science Across Parkinson (ASAP) Collaborative Research Network.
“Parkinson’s disease is a terrible neurodegenerative disease that is actually the fastest growing in our society,” said Valentina Fossati, JAX-NYSCF co-director.
Parkinson’s disease affects more than 8 million people worldwide and causes progressive symptoms including difficulty with walking and speaking. Current treatments can temporarily help with symptoms, but there is no cure.
“We really haven’t had any major breakthrough on Parkinson treatments for decades,” Fossati said. “It definitely is a disease that we need to understand better.”
Genetic focus
Many genetic variants have been associated with Parkinson’s disease, and JAX researchers are seeking to better understand their role. A major focus is the gene GBA1, which is one of the most important genetic risk factors linked to Parkinson’s disease.
“If you have a mutation, a variant in this very specific gene, you have a higher chance to get Parkinson’s disease,” said Stefan Semrau, JAX-NYSCF co-director. “It’s not guaranteed that you will get Parkinson’s, but there’s a higher chance.”
Semrau said that this type of genetic mutation can also lead to earlier disease onset and more severe progression.
Using stem cells gathered from Parkinson’s patients, JAX-NYSCF is studying brain cells to better understand the early-onset causes of the disease.
Shared research tools
Researchers will generate more than 20 new human-induced pluripotent stem cell (iPSC) lines. These cell lines are intended to provide a shared foundation for researchers to model genetic changes, study their effects in cells, and evaluate potential therapies.
“Researchers have identified many promising Parkinson’s disease targets, but the lack of standardized tools has made them difficult to study and compare across laboratories,” said Justin McDonough, a principal investigator of the project and associate director of the Cellular Engineering service lab at JAX.
Validated cell lines will be made available through the JAX iPSC Repository. Associated protocols and data resources will also be shared through the ASAP network and public repositories.
“What we need to understand is, very early, how the disease starts,” Fossati said. “So that we can intervene as early as possible.”
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