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Modifying the Human Genome with Ease

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Researchers have developed a simple and versatile tool for effecting the human genome.

humanGenetics is becoming a bigger and bigger part of modern medicine as our knowledge increases. From diagnostics, to research, and even potential treatments, advanced biotechnologies are becoming more common.

Each of these medical fields requires precise analysis and often manipulation of human DNA. Diagnostics may require mutating certain genes to see what the effect is, in the hopes of identifying disease risk. For research and drug testing scientists need to have cell cultures that mimic the genetic characteristics of various diseases. And for many conditions with a genetic component, gene therapy is being researched as a potential cure. The issue was that scientists didn’t have a standard tool for manipulating DNA in this way, until now.

Researchers from Duke University have found a protein that can be used to precisely and easily manipulate the human genome. After years of tinkering with genes using specially engineered proteins, they took a look at one found in nature. Called Cas9 and found in a streptococcus bacteria, its original function was splicing in virus DNA as part of the bacteria’s adaptive immune system. Previous research showed that these proteins could function in a human cell, so the Duke scientists changed the proteins target. Because Cas9 is guided by RNA it’s easy to manipulate and the research have already achieved promising results.

They demonstrated this tool could activate specific human genes, in particular those related to reducing inflammation, and creating neurons, muscle cells, and stem cells. They were also able to activate a gene known to alleviate the symptoms of sickle cell anemia, in tissue samples. With this protein’s versatility and relative simplicity, they will be making this tool freely available to other research groups. Potentially starting a new wave of genetic discoveries and therapies.

Pablo Perez-Pinera et al. (2013). RNA-guided gene activation by CRISPR-Cas9–based transcription factors NATURE METHODS DOI: 10.1038/nmeth.2600

dna manipulation in humans,cas9 protein

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