In a recent article, both researchers and scientists from Sangamo BioSciences, Inc., have come up with a way to treat hemophilia in animals by using a process of genome editing. Hemophilia is a blood clotting disorder where blood does not clot normally, but now it can be treated by using gene therapy to specifically pinpoint and repair a genetic defect. This new process uses two versions of viruses that are genetically engineered, which carry a replacement gene to be copied into the DNA sequence and enzymes that cut the DNA in the affected area. This study was done using this process by injecting several mice with the gene therapy vector, which was genetically engineered to transport directly to the liver. In the end result, the mice that had received the gene combination, produced a successful amount of clotting agent to reduce the harmful level of clotting to normal levels. This article is both positive and amazing if it will actually cure many of our populations blood clotting disorders throughout the world known as hemophilia.
The zinc finger nucleases are fairly incredible enzymes and there manner of editing mutated sequences of DNA is very cool. As compared to traditional methods which may lead to incorrect delivery (leading to other awful ramifications), one could see the possible effect this process my have with human application in the future.
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