Gene breakthrough could banish inherited diseases

Published on Wednesday 26 August 2009 21:29

A HOST of crippling inherited diseases could be banished forever after a dramatic IVF breakthrough was announced by scientists.

The landmark research raises the prospect of wiping out diseases caused by defective genes in the mitochondria – tiny energy-generating power plants in cells. Experts said the "exciting" genetic research had the potential to be used in human patients after successful tests in monkeys.

Professor Ian Wilmut, of Edinburgh University, said the work took scientists a step closer to preventing children being born with life-threatening diseases.

The study, outlined in the journal Nature, focused on work in macaque monkeys in the United States. By switching around genetic material in monkey eggs, scientists effectively swapped one set of mitochondria for another. The altered eggs were used to produce four healthy monkey infants through in-vitro fertilisation (IVF).

Read Professor Ian Wilmut's analysis of this story here

In future, the same technique could be used to rid human embryos of "bad" mitochondria, and the diseases they cause if used in women with a family link to such illnesses.

Future generations would also be spared the disorders once the fix was in place, marking a controversial step forward from conventional gene therapy.

The scientists believe it will only be a few years before they are ready to try the technique on human patients, assuming they get the funding and ethical go-ahead.

Such research is banned in the UK. But the Human Fertilisation and Embryology Act, which becomes law on 1 October, could open the door to similar trials with the approval of parliament. Further primary legislation would not be needed.

Prof Wilmut, director of the Medical Research Council Centre for Regenerative Medicine in Edinburgh,

said: "The authors are to be congratulated for being the first to demonstrate a new route to therapy. This brings us an important step nearer to being able to prevent the birth of children with a particular type of inherited disease."

Prof Wilmut, part of the team that cloned Dolly the sheep, said the technique could provide an answer to many diseases.

Mitochondrial mutations are known to cause about 150 disorders, some of which cause terrible disabilities or shorten life.

They include muscle-wasting mitochondrial myopathies, Leber's hereditary optic neuropathy which leads to blindness, nerve illnesses similar to multiple sclerosis, and conditions that damage the heart.

Mitochondrial DNA is also believed to play a role in neurodegenerative diseases such as Alzheimer's, Parkinson's and Huntington's, as well as cancers, diabetes and infertility.