Biomedically engineered AE has been suggested as a possible future tool in gene therapy of certain mitochondria-related diseases,[1] however this view is controversial.
[3] In the cells of extant organisms, the vast majority of the proteins present in the mitochondria are coded for by nuclear DNA.
[4] In 2014, Gensight Biologics began a clinical program of allotopic expressing the MT-ND4 gene in the nucleus as therapies for Leber's hereditary optic neuropathy.
[5][6] In 2020, Gensight released Phase III clinical trial results, which showed a notable improvements against the normal progression of the disease but statistical insignificance in all areas.
The company suspected the outcome was due to the transferal of viral vector DNA from the treated eye to the untreated eye of each patient, and thus a full control group trial in which the control group have no exposure to the drug may be needed if requested by the Food and Drug Administration.