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November 30th

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.

November 26th

Monarch Butterfly Genome May Reveal Secrets of Epic Migration

Each fall millions of monarch butterflies from across the eastern United States use a time-compensated sun compass to direct their navigation south, traveling up to 2,000 miles to an overwintering site in a specific grove of fir trees in central Mexico. Scientists have long been fascinated by the biological mechanisms that allow successive generations of these delicate creatures to traverse such long distances to a small region roughly 300 square miles in size. To unlock the genetic and regulatory elements important for this remarkable journey, neurobiologists at the University of Massachusetts Medical School (UMMS) are the first to sequence and analyze the monarch butterfly genome. "Migratory monarchs are at least two generations removed from those that made the journey the previous fall," said Dr. Steven M. Reppert, professor and chair of neurobiology and senior author of the study. "They have never been to the overwintering sites before, and have no relatives to follow on their way. There must be a genetic program underlying the butterflies' migratory behavior. We want to know what that program is, and how it works." In a paper featured as the cover article of the November 23, 2011 issue of Cell, Dr. Reppert and UMMS colleagues Dr. Shuai Zhan, and Dr. Christine Merlin, along with collaborator Dr. Jeffrey L. Boore, CEO of Genome Project Solutions in Hercules, California, describe how next-generation sequencing technology was used to generate a draft 273 Mb genome of the migratory monarch. Analysis of the combined genetic assembly revealed an estimated set of 16,866 protein-coding genes, comprising several gene families likely involved in major aspects of the monarch's seasonal migration. The novel insights gained by Dr.

November 15th

Gene Variant Impedes Recovery from Alcoholism

People who are alcohol-dependent and who also carry a particular variant of a gene run an increased risk of premature death. This is a recent finding from the interdisciplinary research at the Department of Psychology and the Sahlgrenska Academy at the University of Gothenburg, Sweden. Researchers in the longitudinal project Göteborg Alcohol Research Project (GARP) have been investigating the dopamine D2 receptor gene and found that a variant of this gene is overrepresented in people with severe alcohol dependency, and that it is linked to a number of different negative consequences that can be of vital significance to the person affected. "Our research shows that alcohol-dependent individuals, who are also carriers of this gene variant, run 10 times the risk of dying prematurely, compared with the average population," says Dr. Claudia Fahlke, a representative from the research team. In a study published recently in the journal Alcohol and Alcoholism (issue 46), the research team shows that this gene variant also appears to be associated with a higher tendency among these individuals to suffer a relapse, even if they have undergone treatment for their alcohol dependency. This may provide one explanation as to the higher mortality rate in people suffering from alcohol dependency, who are carriers of this gene variant. "This knowledge emphasises the importance of developing methods for early identifying individuals who are also carriers of this gene variant, since the consequences can be so serious," says Dr. Jan Balldin at the Sahlgrenska Academy, University of Gothenburg. [Press release] [Alcohol and Alcoholism journal]