Gene Therapy for Mitochondrial Diseases: Current Status and Future Perspective.

Finally, double administration of AAV2/9-hNDUFS4 by both IV and ICV administration caused a remarkable improvement in the clinical symptoms, such as an amelioration of motor coordination, a gain of body weight, and a highly significant prolongation of the lifespan (82 days) compared with untreated KO mice [ 111 ].

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