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Archive - Nov 30, 2011

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Research Suggests Way to Improve Efficiency and Safety of Gene Therapy

A combination of two techniques promises to improve the efficiency and effectiveness of experimental gene therapies, while also reducing potential side effects, says a new research report published in the December 2011 issue of the FASEB Journal. The report describes how scientists from Germany combined two techniques involving the use of site-specific recombinases, or enzymes that facilitate the exchange of genetic material between DNA strands, to help guide exactly where new genetic material is inserted into a cell's DNA. This experimental approach to gene therapy represents an important advance, as successful gene therapy has the potential to correct the root cause of numerous illnesses and health conditions. "The central outcome of these and related techniques is the predictability and safety of a therapeutic regimen," said Dr. Juergen Bode, a researcher involved in the work from the Institute of Experimental Hematology at Hanover Medical School in Hanover, Germany. "These novel strategies will obviate the majority of animal experiments that are presently needed; it will enhance the effectiveness and shorten the timeline." To make this discovery, Bode and colleagues identified two types of site-specific recombinases (SSR), one from yeast (Flp recombinase) and one from phages (PhiC31 recombinase), which are capable of tagging and targeting specific areas in a DNA strand. Specifically, the tagging process involves mounting a distinct address within a genome, whereas the targeting process covers the delivery of genetic material to this address. PhiC31was identified as an ideal enzyme for tagging because it recognizes just a limited number of pre-existent genomic addresses with well-known and mostly beneficial characteristics, allows for only a one-way transfer of genetic material, and is basically irreversible.

Simple Blood Test Detects Parkinson’s Disease Before Symptoms Appear

A new research report appearing in the December 2011 issue of the FASEB Journal shows how scientists from the United Kingdom have developed a simple blood test to detect Parkinson's disease even at the earliest stages. The test is possible because scientists found a substance in the blood, called "phosphorylated alpha-synuclein," which is common in people with Parkinson's disease, and then developed a way to identify its presence in blood. "A blood test for Parkinson's disease would mean you could find out if a person was in danger of getting the disease, before the symptoms started," said Dr. David Allsop, a researcher involved in the work from the Division of Biomedical and Life Sciences and the School of Health and Medicine at the University of Lancaster, in Lancaster, UK. "This would help the development of medicines that could protect the brain, which would be better for the quality of life and future health of older people." To develop the blood test for Parkinson's disease, Dr. Allsop and colleagues studied a group of people diagnosed with the disease and a second group of healthy people of a similar age. Blood samples from each group were analyzed to determine the levels of phosphorylated alpha-synuclein present. They found those with Parkinson's disease had increased levels of the substance. Based upon these findings, researchers developed a blood test that detects the presence of phosphorylated alpha-synuclein, which could allow for diagnosis of the disease well before symptoms appear, but when brain damage has already begun to occur. "When most people think of Parkinson's disease, they think of the outward symptoms, such as involuntary movements," said Dr.