Allotopic expression (AE) refers to expression of genes in the cell nucleus that normally are expressed only from the mitochondrial genome. Biomedically engineered AE has been suggested as a possible future tool in gene therapy of certain mitochondria-related diseases, however this view is controversial. While this type of expression has been successfully carried out in yeast, the results in mammals have been conflicting.
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Use in Therapy
Gensight Biologics are currently pursuing a clinical program of expressing the ND4 gene in the nucleus to prevent retinal disease.
Research
The SENS Research Foundation recently reported success in expressing the ATP6 gene allotopically in vitro.
As of 6 September 2016 and as a result of funds raised at lifespan.io SENS Research Foundation showed ATP6 and ATP8 could be successfully expressed and their published research appears in Nucleic Acid Research providing proof of concept for the MitoSENS repair approach for repairing age related damage.