Gene Therapy :: IICB scientists achieve breakthrough in gene therapy

In a significant breakthrough in gene therapy, scientists of the Indian Institute of Chemical Biology (IICB) here claimed to have isolated a protein molecule from a parasite that could provide better treatment in some inherited disorders.

The scientists have isolated a protein molecule from the parasite Leischmania and claimed it would ensure better treatment in some forms of inherited disorders.

“What we found is a protein molecule isolated from the parasite. This can be used to correct genetic disorders in a certain kind of genetic disease caused by a change in the DNA sequence. In this, it is a breakthrough, you can say,” Deputy Director Samik Adhya, who led the team of scientists, said.

For their research, the scientists studied the causes of Mitochondrial Epilepsy with Ragged Red Fibres (MERRF) that is characterised by progressive degeneration of tissues.

Mutating functions of mitochondric deficiency leads to a variety of effects on cells and tissues, including nerve and muscle tissues, Adhya said.

“Cells containing such defective mitochondria were treated with the protein isolated from the parasite. What we have observed is that the protein entered the cell and corrected mytochondrial defects leading to increase in respiration,” he pointed out.

The implication of this observation was that it opened an alternative to the conventional gene therapy that used DNA to correct genetic difficulty.

A similar approach can be taken to correct mitochondrial effects in some other diseases such as a certain form of Alzheimer’s or maternally inherited diabetes, Adhya said.

According to the Deputy Director, the team would now work towards validation of the finding on animal models, besides studying its psychotoxic and pharmacological parameters and side effects of the findings.

However, Adhya said there was a problem in conducting trials on animal models, as model with MERRF were not available.

“Our immediate priority, however, is to work on a method to find out how the protein enters the cell. So we will have to do some studies in basic biology,” he said.

Adhya, who began the study with his team nearly 15 years ago, said the first results were reported in 2003.

Funds amounting to about Rs 10 lakh annually had been provided by IICB’s parent body Council for Scientific and Industrial Research (CSIR) and the Department of Science and Technology, Government of India, he added.

Spirit India