Over the years, Brian Madeux has had 26 surgeries to minimize symptoms of Hunter Syndrome, a serious genetic disorder with a general life expectancy of 15 years or less. But with no known cure available, the 44-year-old has been forced to live with the disease, which gradually damages various areas of the body, including thickening the walls of the heart.

In early November, Madeux was part of a revolutionary experiment in genetic therapy. For the first time, a gene will be inserted into the human body with the express purpose of altering a person’s DNA. The procedure is not guaranteed to work. In fact, the gene must be installed in a very specific part of the body to be effective. If misplaced, the change will be permanent and unfortunately, doctors likely won’t know for a few months whether the gene-editing procedure was successful.

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Genetic manipulation has faced tough criticism from those who are concerned about what might happen when science gets involved in the process of creating humans. But with so many inherited diseases costing human lives, researchers are always interested in ways they can make small adjustments to eradicate genetic issues.

Scientists at Oregon Health & Science University recently confirmed that they’ve been working with human embryos and have successfully modified their genes. The work, which will be published in an upcoming journal, involved using a method called CRISPR that allows scientists to modify a section of genes.

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Thursday, 26 February 2015 00:00

Scientists Extend Human Cell Lifespan

Scientists at the Stanford University School of Medicine have discovered a procedure that can efficiently and quickly extend the length of human telomeres, the protective caps on the tips of chromosomes that have been linked to disease and aging. The scientists found that treated cells behave as if they are a great deal younger than those cells which have not been treated. The cells also have multiplied "with abandon" in the lab dish, as opposed to dying or even stagnating.

“Now we have found a way to lengthen human telomeres by as much as 1,000 nucleotides, turning back the internal clock in these cells by the equivalent of many years of human life,” said Helen Blau, PhD, professor of microbiology and immunology at Stanford and director of the university’s Baxter Laboratory for Stem Cell Biology. “This greatly increases the number of cells available for studies such as drug testing or disease modeling.”

The scientists used modified messenger RNA, which carries orders from the genes in the DNA to the protein-making powerhouses in the cells. One advantage the new technique has over others is that it is temporary. The modified RNA is meant to reduce the cell's immune response to the treatment; however, it only hangs around for about 48 hours before disappearing.

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