Allotopic expression

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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,[1] however this view is controversial.[2] While this type of expression has been successfully carried out in yeast, the results in mammals have been conflicting.[3]

Use in Therapy[edit]

Gensight Biologics are currently pursuing a clinical program of expressing the ND4 gene in the nucleus to prevent retinal disease.[4]


The SENS Research Foundation recently reported success in expressing the ATP6 gene allotopically in vitro.[5]

As of 6th September 2016 and as a result of funds raised at SENS Research Foundation showed ATP6 and ATP8 could be successfully expressed and their published research appears in Nucleic Acid Research [6] providing proof of concept for the MitoSENS repair approach for repairing age related damage.