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Gene therapy for the brain

Gene therapy

Promising gene therapy research could reverse disease process.

22 January 2020

An experimental technique which appears to slow tumour growth and prolong life in mice with brain cancer has been developed by a researcher at Ohio State University in the US. The gene therapy transforms human cells into mass producers of nanoparticles full of genetic materials that could reverse disease processes.

The technique involves a patented technology that prompts donated human cells, such as adult stem cells, to produce millions of exosomes. The technology is said to produce up to 50 times more exosomes than other methods.

In a recent study researchers placed around one million donated cells on a nano-engineered silicon wafer and injected synthetic DNA into them using an electrical stimulus. As a result, the cells were forced to eject unwanted material and repair the holes made in their membranes.

James Lee of Ohio State University who developed the method commented that as the unwanted material within the cell membrane, the exosome, is expelled the cell membrane is repaired. ‘What’s expelled from the cell is our drug,’ Lee said.

Lee added that the advantage of the technique was that there was nothing to provoke an immune response. It is believed that such a drug delivery system could prove useful for treating neurological diseases such as Alzheimer’s and Parkinson’s disease.

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