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[ Adults With Hemophilia B Benefit From Gene Therapy ]

Adults With Hemophilia B Benefit From Gene Therapy

Symptoms improved significantly in adults with the bleeding disorder hemophilia B following a single treatment with gene therapy developed by researchers at St. Jude Children's Research Hospital in Memphis and demonstrated to be safe in a clinical trial conducted at the University College London (UCL) in the U.K. The findings of the six-person study mark the first proof that gene therapy can reduce disabling, painful bleeding episodes in patients with the inherited blood disorder. Results of the Phase I study appear in the online edition of the New England Journal of Medicine. The research was also scheduled to be presented December 11 at the 53rd annual meeting of the American Society of Hematology in San Diego. Four study participants stopped receiving protein injections to prevent bleeding episodes after undergoing the therapy and have not suffered spontaneous bleeding.

The Tale Of An Outbreak's Evolution Told By Bacterial Genes

Researchers at Harvard Medical School and Children's Hospital Boston have retraced the evolution of an unusual bacterial infection as it spread among cystic fibrosis patients by sequencing scores of samples collected during the outbreak, since contained. A significant achievement in genetic pathology, the work also suggests a new way to recognize adaptive mutations - to see evolution as it happens - and sheds new light on how our bodies resist infection. The results are to be published online November 13 in Nature Genetics. Cystic fibrosis (CF) is a hereditary disease that renders the lungs susceptible to bacterial infection. Though there is no cure for CF, it is managed with antibiotics and therapies that remove mucous from the lungs. An infection that resists antibiotics can overwhelm the body's defenses and lead eventually to respiratory failure and death, but advances in care have increased the median life expectancy for Americans born with CF from six months in 1959 to nearly 40 years today.

New Medication Effectively Treats Underlying Cause Of Cystic Fibrosis

A new study has confirmed that the drug, ivacaftor (VX-770), significantly improves lung function in some people with cystic fibrosis (CF). The results of the phase III clinical trial study, "A CFTR Potentiator in Patients with Cystic Fibrosis and the G551D Mutation, " led by Bonnie W. Ramsey, MD of Seattle Children's Research Institute and the University of Washington, were published in the New England Journal of Medicine. Ivacaftor, also known as VX-770, was developed by Vertex Pharmaceuticals with financial support from the Cystic Fibrosis Foundation. The oral medicine targets the defective protein produced by the gene mutation called G551D that causes CF. Researchers found that patients carrying G551D - approximately four per cent of all CF patients - who were treated with VX-770 showed a 17 per cent relative improvement in lung function that was sustained over the course of 48 weeks.

New Therapy Marks A Milestone In Fight Against Cystic Fibrosis

Results of the pivotal Phase 3 clinical trial published in the Nov. 3, 2011 New England Journal of Medicine, find that the oral medication ivacaftor (VX-770) provides major, sustained improvement in lung function, growth and other signs and symptoms for cystic fibrosis (CF) patients. The multi-site study marks the first time a drug has been shown to work on the disease process rather than on the symptoms. CF, which has no cure, is the most common lethal genetic disease in Caucasians. "Ivacaftor represents a significant advancement in the treatment of CF. This study shows that the therapy can safely provide long-term benefits to patients with a specific type of cystic fibrosis, " said Michael W. Konstan, MD, one of the study's co-authors and Chairman of the Department of Pediatrics at Case Western Reserve University School of Medicine and UH Rainbow Babies and Children's Hospital.

'Vampire' Bacteria Has Potential As Living Antibiotic

A vampire-like bacteria that leeches onto specific other bacteria - including certain human pathogens - has the potential to serve as a living antibiotic for a range of infectious diseases, a new study indicates. The bacterium, Micavibrio aeruginosavorus, was discovered to inhabit wastewater nearly 30 years ago, but has not been extensively studied because it is difficult to culture and investigate using traditional microbiology techniques. However, biologists in the University of Virginia's College of Arts & Sciences, Martin Wu and graduate student Zhang Wang, have decoded its genome and are learning "how it makes its living, " Wu said. The bacterium "makes its living" by seeking out prey - certain other bacteria - and then attaching itself to its victim's cell wall and essentially sucking out nutrients.

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