GENE THERAPY

Treatment of one eye affects both

Sci. Transl. Med. 12, eaaz7423 (2020)

The injection of a gene-therapy treatment for Leber hereditary optic neuropathy (LHON) into only the right eye of patients with LHON resulted in the unexpected improvement of sight in both eyes.

LHON is a monogenic hereditary mitochondrial DNA neuropathy that results in bilateral vision loss for which there is limited treatment. However, gene-therapy strategies are being developed in which the therapeutic protein product is translocated into the mitochondria through a mitochondrial targeting sequence.

Sahel and colleagues carried out a randomized double-masked, sham-controlled phase 3 trial of one such therapy (AAV2/2-ND4) in 37 patients with LHON. For those who were given the treatment, the authors injected the therapy into the right eye only and treated the left with a sham therapy. They found that this therapy induced an improvement in a least one eye in 25 participants and that 29 participants had improvement in both eyes. This was surprising; however, the authors found a plausible mechanistic explanation in a further nonhuman primate study.

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Correspondence to Hannah Stower.

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Stower, H. Treatment of one eye affects both. Nat Med 27, 21 (2021). https://doi.org/10.1038/s41591-020-01218-0

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