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Catalog
ASAP is committed to accelerating the pace of discovery and informing a path to a cure for Parkinson’s disease through collaboration, research-enabling resources, and data sharing. We’ve created this catalog to showcase the research outputs and tools developed by ASAP-funded programs.
Highly efficient generation of isogenic pluripotent stem cell models using prime editing
Published: Genetically engineered human pluripotent stem cells (hPSCs) containing specific mutations are invaluable for assessing the impact of gene mutations on disease pathology. The authors generated mutated hPSCs through utilization of a novel prime editing-based genome editing platform. Additionally, they show that our system can correct a known Parkinson’s disease-associated mutation. View original preprint here.
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Comparison of alternative pre-mRNA splicing and gene expression patterns in midbrain lineage cells carrying familial Parkinson’s disease mutations
The authors investigated the effects of individual familial PD mutations by developing a medium-throughput platform using genome editing to install individual PD mutations in human pluripotent stem cells (hPSCs) that the authors subsequently differentiated into midbrain lineage cells that include dopaminergic (DA) neurons. Analysis revealed that PD mutations increase pre-mRNA splicing changes.
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iSCORE-PD: an isogenic stem cell collection to research Parkinson’s disease
Genome-edited human pluripotent stem cells (hPSCs) offer the unique potential to advance the understanding of Parksion’s etiology by providing disease-relevant cell types carrying patient mutations along with isogenic control cells. To facilitate this experimental approach, the authors generated a collection of cell lines harboring mutations in genes associated with Parkinson’s.
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