Idebenone vs. rAAV2-ND4 gene therapy in the treatment of Leber's hereditary optic neuropathy: An indirect comparison meta-analysis.

Although not showing the desired effect at the primary endpoint of visual acuity recovery at 24 weeks, the RHODOS results still generated enough of a positive trend in the secondary endpoints of change from baseline in best visual acuity that it warranted further trials assessing the effect of idebenone on LHON.[ 5 6 ] Gene therapy has also been employed in the treatment of LHON by using adeno-associated viruses (AAV), which are nonpathogenic viruses that can infect nondividing human cells.

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