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A team of researchers, including Dr. Patrick Yu-Wai-Man from the University of Cambridge and Dr. José-Alain Sahel from the University of Pittsburgh and Institut de la Vision, Paris, has successfully treated 37 participants suffering from Leber hereditary optic neuropathy (LHON). A landmark trial for mitochondrial gene therapy The results of this phase 3 clinical trial...
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...