February 3, 2016 – 11:53 am
Hematology researchers have used a single injection of gene therapy to correct a rare bleeding disorder, factor VII deficiency, in dogs. This success in large animals holds considerable potential for a safe, effective and long-lasting new treatment in humans with the same bleeding disorder. "Our finding has great clinical relevance for patients with factor VII deficiency," said study leader Paris Margaritis, D. Phil., a hematology researcher at the Raymond G. Perelman Center for Cellular and Molecular Therapeutics (CCMT) at The Children's Hospital of Philadelphia (CHOP). "These dogs have the type of mutation found in the majority of patients with this disorder, so this approach could lead to a sustained gene therapy in people." The study appeared online Dec. 23 in Blood.
February 2, 2016 – 9:46 am
SEOUL, South Korea, Jan. 22, 2016 (GLOBE NEWSWIRE) -- GeneOne Life Science today announced the initiation of a collaborative research program with Inovio Pharmaceuticals to test and advance a DNA-based vaccine for preventing and treating the emerging and virulent Zika virus infection. Inovio and its collaborators are leveraging their past experience in designing and testing novel DNA-based vaccines for related viruses including the West Nile, dengue, and chikungunya viruses. Moreover, Inovio and GeneOne are currently collaborating on two phase I stage vaccine candidates for severe infectious diseases: INO-4212, a vaccine for Ebola infection and GLS-5300, a vaccine for MERS infection. The Zika virus vaccine candidate is currently undergoing preclinical animal studies to evaluate its immunogenicity.
January 26, 2016 – 10:02 am
A technique that combines gene therapy and magnets could someday provide a new tool for treating cardiovascular disease, which puts millions of lives at risk every year. Researchers have produced cells that carry magnetic nanoparticles linked to a therapeutic gene. With an external magnet to direct the cells, the researchers used them to repair damaged arteries in mice (ACS Nano 2015, DOI: 10.1021/acsnano.5b04996).
January 22, 2016 – 10:02 am
LogicBio Therapeutics, a gene therapy startup backed quietly by OrbiMed Advisor’s Israeli arm, is in the midst of an early capital raise: It’s brought in $4 million of a proposed $5.4 million round, according to a regulatory filing. Looks like LogicBio uses recombinant adeno-associated virus-mediated gene targeting (rAAV) to target inherited diseases like hemophilia. The applications of this technology in association with CRISPR gene editing seem promising.
January 13, 2016 – 9:41 am
HIV affects over 30 million people worldwide (over 1.2 million in the U.S.), and while there is no cure yet, there are various drug treatments to contain the virus, which attacks the immune system. Antiretroviral treatments consist of so-called cocktails of chemicals that mainly attack the virus’s ability to replicate in the bloodstream. They have to be taken daily and can have short term and long term side effects.But what if there were a means of rendering the virus harmless by altering the accessibility of the patient’s own white blood cells? That’s the goal of Calimmune’s treatment, called Cal-1. HIV attacks the immune system by destroying CD4+ T-lymphocytes, subsets of white blood cells that are crucial to the health of the immune system. A key landing point for the virus is the CCR5 receptor on the outer surface of these white blood cells.Cal-1 is designed to block the virus by preventing it from binding to CCR5. It achieves this by replacing the CCR5 receptor with a natural variant of the protein found only in the very small percentage of people that are born resistant to HIV.
January 7, 2016 – 11:06 am
UniQure this morning presented results from an early study of AMT-060, a gene therapy it’s developing for patients with hemophilia B. To be clear, these are numbers from just the first handful of patients in UniQure’s study, treated with a low dose of the gene therapy. And they’re early—the patient treated the longest is now 20 weeks post-treatment. For gene therapy to become a viable option for hemophilia, the effects will have to hold up for a long period of time, stop the dangerous, spontaneous bleeds that patients can suffer, and eliminate the need for frequent infusions of the recombinant factors that patients have to take to clot their blood. But the early results are the kind that can make a meaningful impact if they continue to hold up. The first two hemophilia patients treated with AMT-060, about 12 and 20 weeks after treatment, are now producing 4.5 percent and 5.5 percent, respectively, of normal Factor IX. To put that in context, these patients have severe or moderately severe hemophilia, meaning they typically produce less than 1 to 2 percent of these levels, and rely on frequent infusions to get those numbers up.
January 5, 2016 – 9:27 am
Gene therapy has long struggled with a delivery problem: How do you distribute an edited gene to all of the parts of the body where it needs to go? Now scientists have used the CRISPR genome editing tool in mice to solve that problem, a promising finding on the long path to a human therapy. Three papers, published Thursday in the journal Science, show that scientists can use CRISPR’s molecular scissors to treat mice that have been genetically modified to have Duchenne muscular dystrophy, a rare and fatal disease. A similar feat had been accomplished previously in a Petri dish with human cells or mouse embryos, but for the new work, scientists injected the therapy into juvenile and adult mice.
December 28, 2015 – 9:33 am
Oxford University researchers across the globe are working to beat Malaria. Now, a team of Oxford scientists in the UK and Kenya, working with colleagues in three Swiss institutes, have found two people who could reveal a new approach to targeting the malaria parasite. Malaria still claims over 400,000 lives every year, mostly due to infection by a single species of malaria parasite called Plasmodium falciparum. However, people living in areas where malaria is common naturally acquire immunity that protects them during their adult lives. So one route to finding new vaccines against malaria is by getting down to the molecular level to identify the processes behind this natural immune response.
December 28, 2015 – 9:24 am
Physics researchers at The University of Texas at Arlington have developed a new platform that uses ultrafast near-infrared lasers to deliver gene therapy to damaged areas of the retina to enable vision restoration in patients with photo-degenerative diseases. "Most therapies focus on slowing down or halting degeneration but cannot target already-damaged areas of the retina," said Samarenda Mohanty, assistant professor of physics and head of UTA's Biophysics and Physiology Group, who led the research. "Our capacity to specifically target these damaged areas cell by cell opens up a new world of possibilities for vision restoration."
December 16, 2015 – 9:18 am
A combination of immune-modulatory in situ gene therapy (GT) and intensity-modulated radiotherapy (IMRT) is highly effective in treating prostate cancer, according to a study published online in the Journal of Radiation Oncology. The GT was comprised of intraprostatic injection of adenoviral vector containing herpes simplex thymidine kinase followed by valacyclovir. The study included just over 60 patients who were divided into 2 groups. One group, who had cancer cells confined to the prostate, received radiation treatment. The second group, who had more aggressive prostate cancer, received both radiation and hormone treatments. The first group received the experimental gene therapy twice, and the second group received it 3 times during a phase II clinical trial conducted between 1999 and 2003.