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.
read more.-->

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.
read more.-->

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.
read more.-->

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."
read more.-->

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.
read more.-->

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.
read more.-->

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.
read more.-->

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.
read more.-->

‘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 .
read more.-->

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.
read more.-->