Gene Therapy Unveils New Target for Treating Depression

Researchers at Northwestern Medicine say they have manipulated a novel target in the brain using gene therapy that could lead to new treatments for depression. The investigators showed decreasing hyperpolarization-activated cyclic nucleotide-gated (HCN) channel proteins reduced depression-like behavior in mice. If replicated in humans, the findings could inform fresh therapies for millions of patients who do not respond to existing treatments for depression. "Drugs currently available for treating depression help most patients, but they stop working for some patients and don't work from the get-go for others," said senior author Dane Chetkovich, M.D., Ph.D., a professor of neurology and physiology at Northwestern University Feinberg School of Medicine and a Northwestern Medicine neurologist. "There is a real need for new therapies to help patients desperate for alternatives to the available therapeutic options."
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New Targeted Gene Therapy Could Lead to Improved Treatment for Emphysema

Researchers have developed a new strategy using lung-targeted gene therapy that may lead to improved treatments for inherited diseases including emphysema. Alpha-1-antitrypsin deficiency (AATD) is the most common genetic cause of emphysema, a chronic lung disease that leads to significant morbidity and mortality. AATD, which affects up to 100,000 Americans, is caused by inheritance of a single gene mutation. The current treatment for patients affected by AATD involves weekly intravenous infusion of the normal AAT protein -- an inconvenient, invasive and expensive option. Delivery of a normal copy of the gene, known as gene therapy, is an experimental approach to treating some genetic conditions including AATD. Because patients with AATD have low levels of AAT protein in their lungs, researchers at the Center for Regenerative Medicine (CReM) of Boston University and Boston Medical Center have focused their recent efforts on evaluating whether targeting gene therapy directly to the lungs may have promise as a treatment for AATD.
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Cancer Vaccines Conference in London

SMi Group is thrilled to present the 5th annual Cancer Vaccines conference, taking place on 21st & 22nd September 2016 in Central London, UK. Aimed at an audience of senior scientists and oncology specialists involved in targeted vaccines and drug research, Cancer Vaccines 2016 will provide a perfect platform to discuss pioneering clinical developments and the next generation of cancer immunotherapy. The 5th annual show will capture expert insight by honing in on new pathways with novel vaccines and immunotherapies, drug formulations, personalised treatment, immune check-point inhibitors, plus more. Join us this September for strategic direction on vaccine commercialisation and arm yourself with the key requirements and tools for successful vaccine development through informed guidance delivered by a top panel of speakers.
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Medical Breakthrough: First Zika Vaccine Is Ready For Human Trial

Scientists around the world have been long trying to design a vaccine that could help control the menace spread of the Zika virus. The efforts made have been particularly substantial after the World Health Organization (WHO) declared Zika outbreak as the public health emergency in February. So far, the researchers have been able to decode the structure of the virus and actually figure out how Zika infected the fetus of the Zika-infected pregnant mother. Now, for the first time, a Zika vaccine has been finally approved for human trials. According to reports, South Korea's GeneOne Life Sciences and Pennsylvania-based vaccine maker Inovio Pharmaceuticals have received approval from the regulatory agency in the United States to test their DNA vaccine on humans.
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Gene Therapy Is Curing Hemophilia

Maurits has hemophilia B, which means his body doesn’t produce enough factor IX, a protein that clots blood. He’s at risk for bleeding and his joints are damaged from all the bruises. Since he was 10, he’s depended on injections of “ridiculously expensive” replacement protein. Lately, his left ankle has been killing him. In April Maurits, an engineering designer, joined a study in which he was dosed with viruses packed with a correct version of the gene that codes for factor IX. Today at the European Hematology Association’s meeting in Copenhagen, the Philadelphia company that ran the gene-therapy study, Spark Therapeutics, is presenting results on four patients, him included. In all four, factor IX activity has reached about 30 percent of average. That’s enough to prevent bleeding when you get hit by a baseball or twist your ankle. It’s also been enough to let Maurits go without factor IX replacements since April. “There’s no other explanation than ‘It worked,’” says Maurits.
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How a Liver-Damaging Drug May Give a Boost to Gene Therapy

Researchers who want to treat diseases by ferrying a gene into cells often face the hurdle of safely introducing the DNA into enough of them to make a difference. Now, scientists have come up with a novel way to make gene-modified cells in the liver take over much of that organ: They cripple the unmodified cells. This seemingly risky strategy, which relies on the liver’s exceptional regenerative skills, has passed its first test in mice. If equally successful in people, it could be a boon for treating many inherited diseases involving the liver. “This is very much proof of concept. The authors [of the new study] would be first to admit it still has a way to go, but it’s a very exciting step in my view,” says Ian Alexander of the University of Sydney in Australia, who works on gene therapy for childhood liver diseases. “This is very much proof of concept. The authors [of the new study] would be first to admit it still has a way to go, but it’s a very exciting step in my view,” says Ian Alexander of the University of Sydney in Australia, who works on gene therapy for childhood liver diseases.
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The First Gene Therapy for Children has Just Been Approved in Europe

In a landmark moment for scientific research, the world's first gene therapy treatment for children has been given the green light by the European Commission. It's called Strimvelis, and it treats severe combined immunodeficiency (ADA-SCID) – a rare disorder that can be fatal in a very short space of time for those affected. The disease leaves newborns with almost no defence against viruses and bacteria, and is closely linked to X-linked SCID or 'bubble boy' disease, so-called because of a young patient in the US who lived inside a protective plastic shield. What makes the new treatment special is that it's the first time a commercial treatment has used genetic repair techniques to cure a disorder, and thanks to success in clinical trials, it's now approved for marketing within Europe.
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The Virus That Could Cure Alzheimer’s, Parkinson’s, and More

In 2004, the British chemist Chris Dobson speculated that there might be a universal elixir out there that could combat not just alpha-synuclein for Parkinson’s but the amyloids caused by many protein-misfolding diseases at once. Remarkably, in that same year an Israeli scientist named Beka Solomon discovered an unlikely candidate for this elixir, a naturally occurring microorganism called a phage. Solomon, a professor at Tel Aviv University, made a serendipitous discovery one day when she was testing a new class of agents against Alzheimer’s disease. If it pans out, it might mark the beginning of the end of Alzheimer’s, Parkinson’s, and many other neurodegenerative diseases. It’s a remarkable story, and the main character isn’t Solomon or any other scientist but a humble virus that scientists refer to as M13.
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Nanoparticle-Based Gene Therapy May Hold Promise for Patients with Asthma

A new study has demonstrated a way to deliver a nanoparticle-based gene therapy, in order to repair lungs damaged by chronic allergic asthma and to reduce inflammation that causes asthma attacks. The potential therapy, tested in mice, may hold promise for asthma patients whose disease is not controlled by the most commonly used treatments. The study was presented at the ATS 2016 International Conference. "We found that a single dose of highly compacted DNA nanoparticle thymulin gene therapy effectively reduces the inflammatory and remodeling process in asthmatic lungs," said lead author Adriana Lopes da Silva, PhD, from the Federal University of Rio de Janeiro, Brazil. "This is especially important because some patients do not benefit from long-lasting beta2-agonists and inhaled corticosteroids, which are the most widely used asthma therapies."
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HIV Vaccine Design Should Adapt as HIV Mutates

Human immunodeficiency virus is known to be a highly variable virus that adapts to a person's immune response during the lifetime infection, and a new study published in Nature Medicine shows that viral adaptation in HIV can predict a person's current disease status, as well as the degree to which newly transmitted HIV-1 is adapted to their new host. By using a novel method to measure the extent of adaptation of a virus to a person's cellular immune response, the investigators were able to predict how rapidly the disease will progress in that person. The cellular arm of the immune response, through CD8+ T-cells, eliminates HIV-infected cells. These T-cells are activated by pieces of the virus, peptide epitopes, presented on the human leukocyte antigen proteins on the surface of antigen-presenting cells. HLA is a cell surface protein that is one of the most polymorphic variable parts of the human genome, as unique as each person's DNA.
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