Gene therapy: Donor DNA may protect babies from certain disorders – Medical News Today

A novel new technique combining DNA from three different people has reportedly been used as a way to prevent the generational transmission of certain rare genetic diseases.

The process, called mitochondrial donation, uses genetic material from a mother and father and a third donor, in an attempt to drastically reduce or eliminate the exchange of mitochondrial diseases such as muscular dystrophy, hearing and vision disorders, epilepsy, heart conditions, learning disabilities, and even potentially neurodegenerative diseases.

Mitochondria are often called the powerhouses of the cell, and in the cases of mitochondrial diseases, they stop being able to power certain functions as well as they would if they were healthy. That includes the most energy-intensive cells, such as nerves and heart muscle cells.

These babies have genomes derived from their biological father and mother just like any other babies but only had their mitochondria replaced with the ones coming from the donor, said Dr. Steven Kim, a researcher in aging and cancer at the Coriell Institute of Medical Research in New Jersey and a medical content advisor at Breakout.

The practitioners did so by transferring the nucleus of the original egg (mother) to a new, unfertilized egg (donor), he explained to Medical News Today. This will theoretically eliminate the mitochondrial disorders but not without limitations since some residual mitochondria can still be present in the egg and later develop problems.

The process has been approved for use in the United Kingdom under the auspices of the countrys Human Fertilization and Embryology Authority (HFEA), which regulates fertility clinics and their operations.

Hopeful parents are eligible for this procedure only if they are at a very high risk of passing a serious mitochondrial disease onto their children, according to the authoritys website.

Mitochondrial diseases can be quite severe, Dr. Shvetha Murthy Zarek, a reproductive endocrinologist and the Medical and Practice IVF Director at Oma Fertility in St. Louis, told Medical News Today. Curative therapies for mitochondrial diseases have proven to be challenging and this technique is promising.

So far, fewer than five babies have been born using this procedure, although 32 have been approved to do so, the HFEA says.

Mitochondrial donation treatment offers families with severe inherited mitochondrial illness the possibility of a healthy child. The HFEA oversees a robust framework which ensures that mitochondrial donation is provided in a safe and ethical manner, they wrote in a statement. These are still early days for mitochondrial donation treatment and the HFEA continues to review clinical and scientific developments.

While the program is an experimental procedure, its success has raised concerns about using genetic techniques to alter babies before birth in ways outside of the scope of rare genetic diseases.

Before its widespread adoption, there will inevitably be some form of ethical scrutiny and debate in the scientific and healthcare communities, and the regulatory authorities will intervene and provide guidance in the near future, Kim said.

But in the case of this particular method, experts say the potential for it to be exploited for other means is low.

This may be seen as a form of genetic engineering, but this form of Artificial Reproductive Technology (ART) does not lead to intentional modifications in a childs physical features, Dr. Karenne Fru, a fertility specialist at Oma Fertility in Atlanta, told Medical News Today. There is limited utility for this form of ART in individuals without mitochondrial disease. As we gather more data on feasibility and long-term effects, it is possible that more couples choose to proceed with this route.

Fru said there was the potential for all genetic mitochondrial disorders to be cured in this fashion since all of a babys mitochondria are passed down through the mother.

Beyond that, she said the ethics were similar to those involved with any in vitro fertilizations involving donors.

There needs to be clear counseling and communication [to kids] about their origins in a supportive manner to avoid any crisis of identity later on, Fru said. Simply put, it took both parents and a generous, healthy donor for them to exist.

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Gene therapy: Donor DNA may protect babies from certain disorders - Medical News Today