June 23, 2026

#gene therapy — Tuesday, June 23, 2026 (2 items)

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0		💬 0	Patient-specific midbrain organoids with CRISPR correction recapitulate neuronopathic Gaucher disease phenotypes and enable evaluation of novel therapies. (elifesciences.org)	Lin Y … Sun Y eLife 2026-06-23	#crispr #neurodegeneration #gene therapy #organoid #lysosomal-storage-disorder
0		💬 0	Restoring the interplay between the endoplasmic reticulum and mitochondria by gene therapy improves Charcot-Marie-Tooth type 2A disease. (pnas.org)	Tessier M … Bernard-Marissal N PNAS 2026-06-23	#neurodegeneration #gene therapy #mitochondria #motor neuron #neuropathy