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James Wilson, M.D., Ph.D. receives Pioneer Award
James M. Wilson, M.D., Ph.D. (University of Pennsylvania Perelman School of Medicine, Philadelphia) has dedicated his research and medical career to developing gene therapy and the vectors needed to deliver genes into cells for the treatment and cure of inherited diseases. (2014-04-30)

How a fish can fry: Scientists uncover evolutionary clues behind electric fish
Take a muscle cell, modify it over millions of years, and you end up with an exciting and literally shocking evolutionary result: the electric fish.The authors speculate that the down-regulation of the Scn4aa gene leads to quicker evolution and adaptation. (2014-04-29)

Non-uniform genetic mutations identified in lung cancers could lead to targeted treatment
Victorian researchers have extensively studied three of the more common genetic mutations and their distribution across individual lung cancers to see if they matched up to regions of different tumor architecture under the microscope. (2014-04-23)

Loss of memory in Alzheimer's mice models reversed through gene therapy
For the first time, researchers from the Universitat Autonoma de Barcelona have been able to use gene therapy to reverse memory loss in its initial stages in model mice with Alzheimer's disease. The research is featured in this week's issue of the Journal of Neuroscience. (2014-04-23)

Hearing quality restored with bionic ear technology used for gene therapy
Researchers at UNSW Australia have for the first time used electrical pulses delivered from a cochlear implant to deliver gene therapy, thereby successfully regrowing auditory nerves. (2014-04-23)

Progressive neurodegenerative disorder linked to R-loop formation
Researchers at UC Davis have identified a new feature of the genetic mutation responsible for the progressive neurodegenerative disorder, fragile X-associated tremor/ataxia syndrome -- the formation of 'R-loops,' which they believe may be associated with the disorder's neurological symptoms, such as tremors, lack of balance, features of Parkinsonism, and cognitive decline. (2014-04-17)

Ferns borrowed genes to flourish in low light
During the age of the dinosaurs, the arrival of flowering plants as competitors could have spelled doom for primitive ferns. Instead, ferns diversified and flourished under the new canopy -- using a mysterious gene that helped them adapt to low-light environments. A team led by Duke University scientists has pinpointed the curious origins of this gene and determined that it was transferred to ferns from a group of unassuming, mossy plants called hornworts. (2014-04-14)

Seeing double: New study explains evolution of duplicate genes
From time to time, living cells will accidentally make an extra copy of a gene during the normal replication process. Throughout the history of life, evolution has molded some of these seemingly superfluous genes into a source of genetic novelty, adaptation and diversity. A new study shows one way that some duplicate genes could have long-ago escaped elimination from the genome, leading to the genetic innovation seen in modern life. (2014-04-07)

Friedreich's ataxia -- an effective gene therapy in an animal model
The team led by Helene Puccio, director of research for Inserm at the Institute of Genetics and Molecular and Cellular Biology in close collaboration with Patrick Aubourg's team has demonstrated, in the mice, the efficacy of gene therapy for treating the heart disease associated with Friedreich's ataxia, a rare hereditary neurodegenerative disorder. (2014-04-06)

New research initiative investigates gene regulation in evolution and development
Johannes Gutenberg University Mainz has received EUR 900,000 for three years to investigate, jointly with the Institute of Molecular Biology, how gene regulation affects evolution and development. (2014-04-02)

Male extinction prevented by promiscuous females
Female fruit flies with a large number of sexual partners are playing an invaluable role in preventing the extinction of males, research at the University of Liverpool has shown. (2014-04-01)

One gene, many tissues
A map of how genes vary in biological tissues: a huge project that required the collaboration of dozens of laboratories worldwide, including the Neurogenomics Laboratory of the International School for Advanced Studies in Trieste. This is the result of a study just published in Nature by the FANTOM consortium -- and signed by over two hundred authors. It is only the first of a series of more specific papers that will focus on how the single genes work in each type of tissue. (2014-03-27)

Certain genetic variants may put bladder cancer patients at increased risk of recurrence
In the Western world, bladder cancer is the fourth most common cancer in men and the eighth most common in women, with many patients experiencing recurrence after treatment. A new study published in BJU International indicates that inheriting certain DNA sequences can affect a patient's prognosis. (2014-03-26)

MRI reveals genetic activity
New MIT technique could help decipher genes' roles in learning and memory. (2014-03-25)

Pioneer Award recipients Marina Cavazzana and Adrian Thrasher recognized for advancing gene therapy to the clinic for immunodeficiency disorders
Marina Cavazzana, M.D., Ph.D., Paris Descartes University, France and Adrian J. Thrasher, M.D., Ph.D., University College London Institute of Child Health, United Kingdom, have been honored with the Pioneer Award for basic and clinical gene therapy for immunodeficiency disorders. (2014-03-24)

Genetic factor contributes to forgetfulness
Misplaced your keys? Can't remember someone's name? Didn't notice the stop sign? Those who frequently experience such cognitive lapses now have an explanation. Psychologists from the University of Bonn have found a connection between such everyday lapses and the DRD2 gene. Those who have a certain variant of this gene are more easily distracted and experience a significantly higher incidence of lapses due to a lack of attention. The scientific team reports their results in Neuroscience Letters. (2014-03-21)

Electroacupuncture effect on depression and variation of polygenes expression
Preliminary basic research and clinical findings have demonstrated that electroacupuncture therapy exhibits positive effects in ameliorating depression. Using a rat genomic gene-chip, Dr. Dongmei Duan and co-workers from General PLA Hospital in China profiled hippocampal gene expression changes in rats after electroacupuncture therapy. (2014-03-21)

New discoveries place lack of energy at the basis of Parkinson's disease
Neuroscientists Vanessa Morais and Bart De Strooper from VIB and KU Leuven have demonstrated how a defect in the gene Pink1 results in Parkinson's disease. By mapping this process at a molecular level, they have provided the ultimate proof that a deficient energy production process in cells can result in Parkinson's disease. These insights are so revolutionary that they have been published in the leading journal Science. (2014-03-20)

Gene variants protect against relapse after treatment for hepatitis C
Researchers at the Sahlgrenska Academy, University of Gothenburg, have identified a gene, which explains why certain patients with chronic hepatitis C do not experience relapse after treatment. The discovery may contribute to more effective treatment. (2014-03-13)

Gene therapy for lysosomal storage disease shown to be safe and well tolerated
Several young children suffering from a severe degenerative genetic disease received injections of therapeutic genes packaged within a noninfectious viral delivery vector. (2014-03-11)

Exclusive David Gancberg article in Human Gene Therapy
Over the past three funding stages, the European Commission has invested nearly $475 million in 100 projects in the gene transfer and gene therapy field. (2014-02-24)

Like mom or dad? Some cells randomly express one parent's version of a gene over the other
Both of our parents contribute one copy of a gene to our genetic makeup. Generally, both copies are switched on or off together. Occasionally, a cell will begin to use of one copy over the other. Today, a team of researchers at CSHL shows that this random phenomenon is far more likely to be found in mature, developed cell types than in their stem cell precursors, offering an unexpected glimpse of variability in gene expression. (2014-02-24)

Detailed measurements in living cells challenges classic model for gene regulation
In all living organisms, genes are regulated by proteins called transcription factors. The established model states that a gene is switched off as long as a repressing transcription factor is bound to the DNA. For the first time ever, Swedish researchers have been able to study the process in living cells, showing that it may be more complex than previously thought. (2014-02-23)

Joseph Glorioso, Ph.D., receives Pioneer Award
Joseph C. Glorioso, III, Ph.D., devoted much of his research career to developing herpes viruses as efficient vectors for delivering therapeutic genes into cells, and has received a Pioneer Award from Human Gene Therapy. (2014-02-19)

Gene for dissected leaves
Arabidopsis thaliana lost the RCO gene over the course of evolution and thus forms simple leaves. (2014-02-14)

A promising new approach for treating leukemia discovered
A group of researchers at the Institute for Research in Immunology and Cancer (IRIC) of Université de Montréal discovered a promising new approach to treating leukemia by disarming a gene that is responsible for tumor progression. (2014-02-13)

Prostate cancer advance could improve treatment options
Researchers at the University of East Anglia have made an important advance in understanding genetic changes associated with terminal prostate cancer. The research highlights why relapses could happen in some men following hormone therapy. And it could help identify those patients that will develop fatal prostate cancer much earlier for life-extending therapy. (2014-02-12)

Opening 'the X-files' helped researchers to understand why women and men differ in height
Given its unique nature, the X chromosome has often been neglected when performing large-scale genetic studies. Because women have two copies of this chromosome and men only one, identifying genetic associations with X chromosomal genes can be particularly valuable in helping us to understand why some characteristics differ between sexes. Researchers from the University of Helsinki, Finland, have now identified novel X-chromosomal genetic variants that influence human height. (2014-02-06)

Genetic function discovered that could offer new avenue to cancer therapies
Researchers have discovered a genetic function that helps one of the most important (2014-02-03)

Ronald Crystal, M.D., receives Pioneer Award
In recognition of his seminal work on adenoviral vectors, which accelerated the translation of gene therapy from the research laboratory to the clinic, Ronald G. Crystal, MD (Weill Cornell Medical College, Cornell University, New York City), has received a Pioneer Award from Human Gene Therapy, a peer-reviewed journal from Mary Ann Liebert, Inc., publishers. (2014-01-31)

Precise gene editing in monkeys paves the way for valuable human disease models
Monkeys are important for modeling diseases because of their close similarities to humans, but past efforts to precisely modify genes in primates have failed. In a study published in the journal Cell, researchers achieved precise gene modification in monkeys for the first time using an efficient and reliable approach known as the CRISPR/Cas9 system. The study opens promising new avenues for the development of more effective treatments for a range of human diseases. (2014-01-30)

Single gene separates queen from workers
Scientists have identified how a single gene in honey bees separates the queens from the workers. A team of scientists from Michigan State University and Wayne State University unraveled the gene's inner workings and published the results in the current issue of Biology Letters. The gene, which is responsible for leg and wing development, plays a crucial role in the evolution of bees' ability to carry pollen. (2014-01-29)

Drug to reverse breast cancer spread in development
Researchers at Cardiff University are developing a novel compound known to reverse the spread of malignant breast cancer cells. The vast majority of deaths from cancer result from its progressive spread to vital organs, known as metastasis. In breast cancer up to 12,000 patients a year develop this form of the disease, often several years after initial diagnosis of a breast lump. (2014-01-26)

Gene therapy leads to robust improvements in animal model of fatal muscle disease
Preclinical studies show that gene therapy can strengthen muscles and lengthen lives in animal models of a fatal congenital disease in children, X-linked myotubular myopathy. The findings demonstrate the clinical feasibility of future trials of gene therapy for this devastating disease. Children born with the condition have floppy muscles and breathing difficulty, and may need extensive life support. Most die in childhood. Researchers tested the effectiveness of treatment in mice and dogs with engineered adenovirus vector carrying a gene to replace the mutation. (2014-01-22)

Study: Possible new druggable target in Ewing's Sarcoma
A University of Colorado Cancer Center study, recently published in the journal Oncogene, shows that downstream from the oncogenic fusion of gene EWS with FLI1 is a signaling chain that includes microRNA-22 and the gene KDM3A. By targeting these links, researchers hope to break this cancer-causing chain. (2014-01-21)

Gene therapy improves eyesight in people born with an incurable form of blindness
A new gene therapy has restored some sight in people born with an inherited, progressive form of blindness. The technique replaces a defective gene in the eye with a normal working copy of the gene using a single injection. (2014-01-15)

Mitochondrial genes matter!
Contrary to common belief, mitochondrial genes seem to matter for how well individuals survive and reproduce. These new results are reported by researchers at Uppsala University, Sweden, who studied the genes of a common beetle species. (2014-01-14)

Does the body's immune response to viral vector delivery systems affect the safety or efficacy of gene therapy?
Packaging replacement genes in viruses is an effective method to deliver them to target tissues, but the human body mounts an immune response against the virus. (2014-01-08)

How fat might be controlled through the body clock
Australian researchers have shed more light on an underexplored aspect of the important brain-signaling system that controls appetite, body composition and energy use. Their findings suggest that a specific gene regulating our body clock may play a central role in determining how fat we become. (2014-01-07)

Loss of function of a single gene linked to diabetes in mice
Researchers from the University of Illinois at Chicago College of Medicine have found that dysfunction in a single gene in mice causes fasting hyperglycemia, one of the major symptoms of Type 2 diabetes. Their findings were reported online in the journal Diabetes. (2014-01-03)

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