June 23, 2016 – 11:51 am
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.
June 20, 2016 – 11:57 am
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.
June 8, 2016 – 4:50 pm
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.
May 20, 2016 – 9:52 am
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.
May 19, 2016 – 2:11 pm
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."
May 17, 2016 – 11:11 am
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.
May 17, 2016 – 10:53 am
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.
May 10, 2016 – 2:23 pm
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.
May 4, 2016 – 9:38 am
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.