The population of mitochondria in the oocyte provides the template for all mitochondria that end up populating every cell in the resulting organism. As such anything that compromises the function of this mitochondrial population puts embryo development and organismal health at risk. Functional mitochondria are built primarily by the nuclear genome, but a small number of critical genes are also encoded by the mitochondrial genome. As such, mutations in relevant nuclear genes or in mitochondrial genes can severely compromise mitochondrial function, leading to severe disease and death.

Mitochondrial diseases have no cure, but the IVF-based technique known as mitochondrial donation can prevent the inheritance of mitochondrial DNA disease in subsequent generations. Mitochondrial Donation involves using micromanipulation techniques to transfer the nuclear genome from the egg or of an affected individual to an enucleated donor egg with healthy mitochondria. The resultant egg contains the nuclear DNA of the intending parent and the mitochondrial DNA of the egg donor.

This intervention has the potential to transform the reproductive options for women at risk of transmitting mitochondrial DNA disease, thereby relieving future generations from disease. It’s introduction to the clinic was pioneered in the UK and a recent publication reported the birth of 8 healthy babies (Hyslop et al, 2025). Now, with Maeve’s Law passed in Australia in 2022, the MRFF-funded mitoHOPE Pilot Program, including an underpinning research program and clinical trial, is progressing through the regulatory pathway. This program will focus on improving the efficacy and safety of mitochondrial donation as it progresses toward introduction to clinical practice.