Genetics and Health

Nuclear Transfer to Cure Mitochondrial Diseases

by Hsien Hsien Lei, PhD on September 1st, 2005

Appxoximately 50 metabolic disorders are caused by defects in mitochondrial DNA. Scientists are proposing a controversial form of gene therapy that would cure these diseases by using a fertilized egg and an enucleated donor egg.

1. Pronuclei - the chromosome-containing residues of the egg and sperm - are first removed from a fertilized egg.
2. A donor egg from a healthy woman would have its nuclear DNA removed.
3. The pronuclei from the original fertilized egg are then transplanted into the donor egg.
4. The resulting product would be an embryo with nuclear DNA from the parental egg and sperm but mitochondria - and mitochondrial DNA - from the donor egg.

Nuclear transfer has already successfully cured mitochondrial diseases in mice. But aside from the technological challenges of making it work in humans, there is ethical debate as well such as that regarding the method’s effect on the genetic make-up of future generations.

Among the organizations in support of nuclear transfer is the Children’s Mitochondrial Disease Network and prominent geneticists are demonstrating their support as well.

Prof James Watson, who shared the Nobel Prize for his work on DNA’s double helix, is one enthusiastic proponent: DNA manipulation could be used to make people resistant to the ravages of HIV, or transform slow learners into fast ones, according to Watson, who has often voiced his backing for “simply putting right what chance has put horribly wrong. You should be allowed to improve human life.”

Telegraph, August 31, 2005

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POSTED IN: Genetic Engineering