Alzheimer’s Disease: Gene Therapy May Promote Neuronal Growth

A gene therapy that boosts nerve growth factor (NGF) production in cholinergic neurons was safe and appeared to promote neuronal growth in a small study of patients with early Alzheimer's disease, researchers reported. All 10 patients in the trial, which started in 2001, showed a trophic response to the growth factor delivered via gene therapy, Mark Tuszynski, MD, PhD, of the University of California San Diego, and colleagues reported online in JAMA Neurology.
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Synthetic DNA–based MERS Vaccine Shows Promise

A synthetic DNA–based vaccine targeting the Middle East respiratory syndrome coronavirus (MERS-CoV) protects rhesus macaques from developing symptoms of the disease. The vaccine also elicits antibodies against the spike protein of the virus in camels, which are thought to facilitate the spread of the pathogen to humans. These preclinical results were published today (August 19) in Science Translational Medicine. David Weiner, a professor of pathology at the University of Pennsylvania who develops gene-based vaccines, and his colleagues took cues from previous vaccine studies of the related severe acute respiratory syndrome coronavirus (SARS-CoV), which showed that the spike glycoprotein is immunogenic and induces neutralizing, infection-preventing antibodies. The researchers designed an artificial consensus sequence of the MERS-CoV glycoprotein based on sequences of isolates from past and ongoing MERS outbreaks. “[The sequence is changed slightly to emphasize conserved regions of the protein that provoke an immune response,” said Weiner.
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Recorded Webinar: ‘DNA Vaccines: Immunology, History and Ways Forward’ is Now Available!

DNA Vaccines: Immunology, History and Ways Forward: Recording of DNAvaccine.com July 28, 2015 webinar presented by Jeremy Houser, a student at Concordia College (MN).
The online recording of Jeremy Houser’s presentation: ‘DNA Vaccines: Immunology, History and Ways Forward’ can be viewed in the webinar tab found on our homepage. To view this and other archived webinars simply click on the following link: http://www.dnavaccine.com/resources/
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Redirecting Gene Therapy Restores Sight

A team of researchers at the University of Manchester enabled blind mice with advanced retinal degeneration to see by delivering the gene for a light-sensing protein into nervous cells within their retinas, according to a study published last month in Current Biology. “This is the most effective example yet of the use of genetic therapy to treat advanced retinal degeneration,” Robin Ali, who studies gene therapy at University College London and was not involved in the work told New Scientist.
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Inovio Pharmaceuticals Enters Into Strategic Cancer Vaccine Collaboration and License Agreement With MedImmune

Inovio Pharmaceuticals today announced that it has entered into a license agreement and collaboration with MedImmune, the global biologics research and development arm of AstraZeneca. Under the agreement, MedImmune will acquire exclusive rights to Inovio's INO-3112 immunotherapy, which targets cancers caused by human papillomavirus (HPV) types 16 and 18. INO-3112, which is in phase I/II clinical trials for cervical and head and neck cancers, works by generating killer T-cell responses that are able to destroy HPV 16- and 18-driven tumors. These HPV types are responsible for more than 70 percent of cervical pre-cancers and cancers. MedImmune intends to study INO-3112 in combination with selected immunotherapy molecules within its pipeline in HPV-driven cancers. Emerging evidence suggests that the benefits from immuno-oncology molecules, such as those in MedImmune's portfolio, can be enhanced when they are used in combination with cancer vaccines that generate tumor-specific T-cells.
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Newly Discovered Gene Therapy Could Help Treat Cystic Fibrosis

An increasing number of studies testing gene therapies are showing promise for treating a wide range of conditions, including cystic fibrosis. Normally in these studies, genes are injected into cells using a virus, however, scientists recently tested a new method of delivering DNA to the faulty CFTR gene in CF through what is called a cationic liposome, a fatty container that can deliver DNA directly to a cell. Results from a Phase 2 efficacy study testing this non-viral CFTR gene therapy for cystic fibrosis appeared in the July 3rd issue of the Lancet Respiratory Medicine, showing promise as a future, novel therapeutic approach for CF.
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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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