Researchers Develop First Step Toward Controlled Gene TherapyJuly 20, 2016
The ability to switch disease-causing genes on and off remains a fantasy. However, a team of researchers from Charité – Universitätsmedizin Berlin and the Max Planck Institute for Medical Research in Heidelberg may have just turned this into reality. Led by Dr. Mazahir T. Hasan, the team has programmed a virus to transport the necessary genetic material to affected tissues and deliver instructions to the host without becoming part of it.
"We use attenuated, non-replicating viruses known as recombinant adeno-associated viruses (rAAV). We use them to transport genetically encoded material into live organisms affected by disease," explains Dr. Hasan. "This approach opens up a whole range of options which, in the future, may allow us to treat and heal various diseases."
RAAVs can transport genetically-encoded material into any type of cell and tissue and are capable of repeatedly switching gene therapy applications on and off again. This on/off switch is controlled chemically, via food intake or drinking water. RAAV-infected cells also do not trigger any immune response and their genetic material remains intact.
For more information, read the article in Nature.
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