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Tag: ND4

Eye
GenSight Biologics has recently released data showing the effectiveness of GS010, the company's gene therapy for Leber Hereditary Optic Neuropathy (LHON), a mitochondrial disease that can lead to blindness. Like in previous studies, this therapy had a bilateral effect. Gene Therapy In LHON, the mitochondrial protein ND4 is poorly expressed through mitochondrial DNA (mtDNA). GS010...