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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Gene Therapy Against Brain Cancer

Applying gene therapy to glioblastoma appears to be a very promising treatment, say researchers. Tests in vitro and in vivo on mice provided very clear-cut results, and modelling demonstrates that the treatment targets at least six different points of tumor metabolism. Gene therapy, a technique that selectively attacks a tumor, might provide hope in the fight against this type of deadly cancer, for which surgery is practically impossible and chemo- and radiotherapy are ineffective against very aggressive recurrences.
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Gene Therapy Might be the Best, and Perhaps Only, Chance at Curing Brain Diseases

There is no treatment and no cure for Canavan disease. Yet standing in a room at the University of Massachusetts Medical School, Epstein holds a mouse that once had the same symptoms as Rachel—but now runs effortlessly over her outstretched fingers. Weeks before, the mouse was treated with a single intravenous injection of a gene therapy drug developed in the lab of Gessler’s doctoral adviser, microbiologist Guangping Gao. The drug is the fruit of Gao’s 23-year career in gene therapy. Gene therapy is well-suited for treating inherited brain diseases. First, most drugs can’t get through the brain’s formidable blood-brain barrier, but something small, like a virus with a healthy gene tucked inside, can do it. Additionally, the brain is a closed compartment, so the risks of gene therapy are minimized—other parts of the body, the liver or the lungs, say, are undisturbed.
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Aerosol-Delivered Gene Therapy Seen to Stop Vascular Remodeling in PH Study in Pigs

A new, aerosol-delivered gene therapy has shown promising effects in a pig model of pulmonary hypertension (PH), according to a study published in the Journal of The American College of Cardiology (JACC), titled “Intratracheal Gene Delivery of SERCA2a Ameliorates Chronic Post-Capillary Pulmonary Hypertension“. PH is characterized by pulmonary arterial remodeling that results in increased pressure in the vessels that take blood from the heart to the lungs. The thickening of pulmonary vessels is triggered by abnormal calcium levels within the vascular cells, which leads to hyper-proliferation of the muscle cells that line the blood vessels. One protein involved in the regulation of intracellular calcium levels is the sarcoplasmic reticulum calcium ATPase pump (SERCA2a), which may be a possible therapeutic target for the treatment of PH.
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‘Cure for Blindness’ Gene Therapy to be Tested on Humans Within 3 Years

Groundbreaking gene therapy that could cure blind people is to be tested on humans within three years. Scientists who are currently testing the treatment on mice have revealed they plan to be ready to test on human subjects soon. The development is to be applied to those suffering from an inherited retinal condition called Retinitis Pigmentosa (RP). Although the issue affects one in 4,000 people, there is currently no effective treatment. The research conducted by the University of Manchester, was licensed by a commercial concern, the US clinical-stage biotechnology company Acucela .
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FIT Biotech Oy: Finnish FIT Biotech and US Ichor to Collaborate on New Cost Effective Infectious Disease Treatment

FIT Biotech Oy (“FIT Biotech”) (Nasdaq: FITBIO) and Ichor Medical Systems (“Ichor”) of San Diego, California announced today that they have entered into a Research Collaboration Agreement. The purpose of the collaboration is to test FIT Biotech’s proprietary GTU® technology in combination with Ichor’s TriGrid® electroporation system in order to elicit the endogenous production of protective antibodies for passive immunoprophylaxis. By offering the prospect for rapid and sustained production of highly active antibodies from a recipient’s own cells, this approach could provide a new potential avenue to prevent the onset and progression of a wide range of infectious diseases. The ultimate goal of the research is the development of a highly adaptable and cost effective infectious disease product platform capable not only of addressing currently circulating infectious pathogens but also emerging diseases with the potential to cause worldwide pandemias.
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Inovio Pharma Announces Publication of Positive CHIKV-Targeting DNA Vaccine Data in Mice

Inovio Pharmaceuticals, Inc. (Nasdaq: INO) announced that its novel dMAb antibody and DNA vaccine targeting the chikungunya virus (CHIKV) provided 100% protection against a lethal virus challenge in mice. This breakthrough data was published in the latest issue of The Journal of Infectious Diseases in a paper, “Rapid and long-term immunity elicited by DNA encoded antibody prophylaxis and DNA vaccination against Chikungunya virus,” prepared by Inovio authors and their academic collaborators. While conventional vaccine and marketed monoclonal antibody technologies have shown limited ability to provide an effective solution to CHIKV to date, Inovio’s DNA vaccine and dMAb products show potential, separately and in combination, to offer immediate and long term protection to large populations from CHIKV infection. Over the years, CHIKV outbreaks have occurred in Africa, Asia, Europe, and throughout the Indian and Pacific Oceans, with local transmission in over 43 countries infecting millions of people. In late 2013, CHIKV was found for the first time in the Americas on islands in the Caribbean and spreading to other parts of the western hemisphere, including the United States. Along with a dramatic increase in cases and geographic spread of CHIKV infection and disease there has been a reported increase in morbidity and mortality, suggesting increased virulence. The concern for even greater potential global outbreaks underscores the need for targeted anti-viral interventions.
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Gene Therapy Approach Reported to Block Mutant Huntington’s Gene in Preclinical Studies

uniQure recently published results from preclinical studies of its gene therapy program, AMT-130, indicating that a one-time administration of AAV5-delivered therapy into the central nervous system can block the mutant HTT gene that causes Huntington’s disease (HD). These findings, in the article “Design, Characterization, and Lead Selection of Therapeutic miRNAs Targeting Huntingtin for Development of Gene Therapy for Huntington’s Disease,” were published in the Molecular Therapy-Nucleic Acids journal. The data also formed the basis of a presentation for the 11th Annual CHDI Huntington’s Disease Therapeutics Conference that took place on Feb. 24, 2016, in Palm Springs, California. Huntington’s disease is a neurodegenerative disorder that reduces muscle coordination and leads to behavioral and cognitive decline over time. Though the causes of HD are still not fully understood, a number of studies have shown that genetic mutations leading to the formation of an altered form of a protein called mutant Huntingtin (HTT) leads to HD development.
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