Hyderabad lad helps develop novel gene editing technique

May 06, 2015 12:00 am | Updated 09:05 am IST - HYDERABAD:

City boy Pradeep Reddy and his team of researchers at Salk Institute of Biological Studies in California have developed a technique that could help women prevent their children from inheriting severe illnesses including those of heart and brain.

The technique involves editing of DNA in the mitochondria, dubbed as the ‘powerhouse’, as it generates chemical energy for the body. Mitochondria contain genetic material which is inherited only from the mother, unlike DNA in the cell’s nucleus which is inherited from both parents.

Hence, disorders attributed to mitochondrial DNA (mtDNA) mutations can only be inherited from the mother. In families where incidence of maternal inheritance of disease is strong, the eggs can be edited before in vitro fertilization by sperm.

Disorders of mitochondria can affect single organs and could cause fatality at birth or leave individuals with severe illness for a lifetime. Mutations in mtDNA are known to mainly affect organs that are heavily energy dependent like brain and heart. They also cause hormonal disorders like diabetes.

Mr. Reddy’s team showed specific enzymes called ‘restriction endonucleases’ and ‘transcription activator-like effector nucleases’ can be used to target mtDNA mutations and eliminate them. Their technique was demonstrated in preventing a specific type of mtDNA in mice eggs from being passed down.

“In the generated mice, we have done a complete blood analysis and also measured the levels of glucose and other markers for mitochondria dysfunction. All the analysis displayed normal levels in these mice. These results demonstrate the potential of this technology in future to prevent the transmission of mitochondrial diseases,” Mr. Reddy explained via email.

Mr. Reddy had obtained a doctorate from Sweden and completed post-doctorate work at Stanford University before joining Izpisua Belmonte’s lab at the Salk Institute as a research associate. But, the technique has to be tested for efficacy and safety before experiments on human eggs are done.

Should it be clinically approved for human use, the technique can be done in fertility clinics as it only involves administering an injection into eggs, he added.

The technique involves editing of DNA in the mitochondria which will help women prevent their children from inheriting severe illnesses

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