NeurosciencebioRxiv
Patient-Specific Midbrain Organoids with CRISPR Correction Recapitulate Neuronopathic Gaucher Disease Phenotypes and Enable Evaluation of Novel Therapies
Yi Lin, Benjamin Liou, Venette Fannin…·Ying Sun
This paper develops patient-specific midbrain organoids with CRISPR correction to model neuronopathic Gaucher disease and test therapies.
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