Gene Editing Prevented NfL Protein Accumulation in CMT2E Motor Neurons

Gene Editing Prevented NfL Protein Accumulation in CMT2E Motor Neurons

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Gene Editing Prevented NfL Protein Accumulation in CMT2E Motor Neurons

The gene-editing technology CRISPR/Cas9 effectively inactivated a specific mutation in the NEFL gene that causes Charcot-Marie-Tooth disease type 2E (CMT2E), a study reports. Correction of this mutation prevented the harmful buildup of neurofilament light chain (NfL) protein in motor neurons — specialized nerve cells that control muscle function — derived from a patient with CMT2E, with no evident adverse effects. The study, “Allele-Specific Gene Editing Rescues Pathology in a Human Model of Charcot-Marie-Tooth Disease Type…

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