Scientists have a new strategy that will allow them to easily manipulate the DNA of cells to "fix" wayward genes that cause a broad range of diseases including diabetes and cancer.
The tool known as CRISPR (Clustered, Regularly Interspaced, Short Palindromic Repeat), lets scientists edit the human genome, allowing them to cut and paste DNA to create new therapies.
In a study published in Proceedings of the National Academy of Sciences, scientists at the University of California San Francisco disabled a protein on the surface of T-cells which is vulnerable to HIV, and shut down the path the virus uses to infect the cell and cause AIDS.
T-cells are vital for the immune system to function properly, and help protect against invading viruses and bacteria. They are also involved in autoimmune diseases such as diabetes.
"This could be a stepping stone to engineering T-cells that are immune to HIV and then putting them back into the body," Alexander Marson, Ph.D. of the University of California San Francisco told the Telegraph. "Potentially we have the power to engineer proteins that are a target for HIV infection," he said.
In another experiment, the researchers used CRISPR to alter a T-cell protein called PD-1, which helps the immune system fight cancer. Scientists hope to tweak T-cells from cancer patients that would keep their disease at bay and perhaps completely kill tumors. "There is still some work needed to be done ... but I do think this is a powerful tool to add to the arsenal," Marson said.
In theory, the CRISPR system will be easy and inexpensive to use, and can edit genetic information in any organism. Scientists are focused on altering T-cells since they can be easily harvested from patients because they circulate in the blood stream. Scientists hope to edit T-cells with the CRISPR technique, and then return them to the body to treat and cure diseases.
"There is increasing interest in manipulating the genome of T-cells, either by correcting mutations or changing the genome to increase the chances of the cells being able to fight off cancer or infections," Marson told the Telegraph.
Marson and his colleagues used the CRISPR technique to cut and splice fragments of DNA in chromosomes of human T-cells in lab dishes to show that it is precise enough to use eventually as a clinical treatment.
Other researchers have had success as well with the CRISPR technique. It has been used by Harvard researchers to alter genes in mice to lower their cholesterol levels. In addition, scientists at Children's Hospital Boston have used it to correct gene mutations that cause a common genetic disease called beta thalassemia, a blood disorder that reduces the production of hemoglobin.
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