Gene Therapy for Botulism

Gene therapy could be more effective than existing treatments for botulism, a rare paralytic illness caused by a nerve toxin, according to an infectious disease researcher at Cummings School. The new approach might also be able to be used to treat other more common infections, such as E. coli food poisoning and the hospital-acquired superbug known as Clostridium difficile. Botulism is caused by a toxin produced by the soil-borne bacterium C. botulinum. People typically get sick when they eat improperly preserved or canned food. Despite the relatively small number of botulism poisoning cases in the U.S., globally there is a serious concern that the toxin—the most potent one known—can be produced easily and inexpensively as a bioterrorism weapon.
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Universal Flu Vaccine in the Works

Each year, scientists create an influenza (flu) vaccine that protects against a few specific influenza strains that researchers predict are going to be the most common during that year. Now, a new study shows that scientists may be able to create a 'universal' vaccine that can provide broad protection against numerous influenza strains, including those that could cause future pandemics. The study appears in mBio, the online open-access journal of the American Society for Microbiology.
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Gene Therapy Restores Hearing in Mice

Researchers have restored hearing in mice with two versions of a genetic mutation that, in humans, causes gradual hearing loss during childhood. The successful work with mice is focused on a gene critical to hearing, TMC1, which encodes a protein that allows physical stimulation of "hairs" in the ear to be translated into sounds understood by the brain. Two types of genetic mutations, one recessive, with the gene deleted, and one dominant, with an altered amino acid, lead to what accounts for 4 to 8 percent of genetic deafness cases. The recessive mutation, which is more common, causes hearing loss by the time a child is 2 years old. The dominant version progressively causes children to lose their hearing starting around 10 or 15 years old.
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Gene Therapy for Cystic Fibrosis Shows Encouraging Results

The study was carried out by the UK Cystic Fibrosis Gene Therapy Consortium, a group of scientists and clinical teams from Imperial College London, the Universities of Oxford and Edinburgh. It involved 116 patients aged 12 and over who received monthly doses of either the therapy or the placebo for one year. Patients who received therapy showed a significant but modest benefit in lung function compared with those receiving a placebo. The trial is the first to show that repeated doses of gene therapy can have a meaningful effect on the disease, and change the lung function of patients. However, the team say more research is needed to improve the effectiveness before the therapy will be suitable for clinical use.
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Creative Diagnostics Launches DNA Immunization Antibody Production Service

Creative Diagnostics, the global diagnostics service and product supplier, is pleased to announce its launch of custom antibody production service based on DNA immunization technology. This DNA immunization technology allows high success rate in generation of high-affinity antibodies recognizing difficult-to-express proteins in their native confirmation, such as GPCRs, ion channels and other multiple membrane spanning proteins. Genetic immunization is an attractive approach to generate antibodies because native proteins are expressed in vivo with normal post-transcriptional modifications, avoiding time-consuming and costly antigen isolation or synthesis. And DNA Immunization technology is a powerful tool to aid in custom antibody production against membrane proteins, other problematic antigens, as well as early DNA vaccine development studies. The unique antibody development approach involves direct immunization of host animals with plasmid DNA encoding the target protein of interest.
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First Canadian Gene Therapy Trial for Blindness Sees Positive Results

The first four patients in Canada’s only gene therapy trial for blindness say their vision has improved. All four of the Edmonton-area men have choroideremia, an inherited disorder that usually leads to legal blindness by the age of 40. Ken Ross, 43, underwent the procedure on his right eye on May 25, after what he describes as decades of “looking through a pinhole.” He says when doctors removed the bandage two days later the room seemed instantly brighter. And the view outside was better than ever.
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DNA-Based Animal Vaccines Providing Growth Impetus for Overall Global Vaccines Market

The DNA vaccine segment of the animal vaccines market accounted for the highest growth in veterinary vaccine technology during 2014. Continued current innovation in the field seems likely to continue to drive this trend and thus diversify the animal vaccines market in general. DNA vaccines, particularly in the current animal vaccine market, offer various advantages over more traditional vaccination approaches. It enhances the immune systems response via an uptake in the overall quality and extent from the body, as well as carrying a stronger safety record than other applied veterinary approaches. It has been proven to combat both infectious diseases and malignant cancerous forms, breaking the tolerance these cancerous cells hold in the animal's body, which is a key focus for the vaccination development companies moving forward. With Dr Niranjan Sardesai of Inovio Pharmaceuticals declaring that the past ten years has been potentially the 'decade of DNA vaccines', perhaps exploitation of their cost-effectiveness will also contribute to a continued growth in CAGR.
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Pioneering Gene Therapy May Save Babies’ Lives

SMA, the most common childhood disease of its type, arises from a recessive defect, meaning both mom and dad have to carry it and that each pregnancy brings a 25 percent risk of a baby born with a mutation in a gene called SMN1. Researchers have developed a Gene Therapy treatment for the disease which has shown to have significantly improved the condition of mice used in the experiments. In a small scale test on 8 infants the treatment has shown to improve or at least maintain their condition.
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Promising Gene Therapy for Friedreich’s Ataxia Based on Frataxin Expression

A team led by researchers at the Universidad Autónoma de Madrid in Spain recently published new data on a gene therapy for Friedreich’s ataxia based on the expression of the human frataxin gene from artificial systems. The study is entitled “Delivery of the 135 kb human frataxin genomic DNA locus gives rise to different frataxin isoforms” and was published in the journal Genomics. Friedreich’s ataxia is a rare inherited neurodegenerative disease characterized by progressive damage of the nervous system with degeneration of the spinal cord and peripheral nerves that leads to muscle weakness, sensory loss, balance deficits and lack of voluntary coordination of muscle movements. The disease is caused by a mutation in a gene called frataxin (FXN) that leads to a defective expression of the frataxin protein. Disease onset is usually during childhood or adolescence and the disorder leads to progressive disability, dependence on a wheelchair and reduced life expectancy. Currently, there is no effective approved treatment for the disease.
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