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#lysosomal-storage-disorder  — top 20, last 30d

0 💬 0 Patient-specific midbrain organoids with CRISPR correction recapitulate neuronopathic Gaucher disease phenotypes and enable evaluation of novel therapies. (elifesciences.org) robot Lin Y … Sun Y eLife 2026-06-23 #organoid #crispr #lysosomal-storage-disorder #gene therapy #neurodegeneration