Category Archives: Gene Therapy

PUBLIC RELEASE DATE: 19-Aug-2014 Contact: Derek Thompson thompsonder@health.missouri.edu 573-882-3323 University of Missouri-Columbia COLUMBIA, Mo. A new gene therapy developed by researchers at the University of Missouri School of Medicine has been shown to protect mice from a life-threatening heart condition caused by muscular dystrophy. "This is a new therapeutic avenue," said Yi Lai, Ph.D., the leading author of the study and assistant research professor in the MU School of Medicine's Department of Molecular Microbiology and Immunology. "This is just a first step, but we hope this could lead to a treatment for people with this devastating heart condition, which is a leading cause of death for people with Duchenne muscular dystrophy." About one in 3,500 children, mostly boys, are born with Duchenne muscular dystrophy (DMD). They experience a progressive wasting away of muscles, starting in the legs and pelvis. Children with DMD have difficulty walking, and most need wheelchairs by age 12. As DMD depletes the skeletal muscles, it also causes the heart to decay. A weakened heart kills up to 40 percent of people with DMD, usually by their 20s or early 30s. DMD originates with mutations in a single gene. For more than two decades, researchers have explored … Continue reading →

(DGAP-Media / 19.08.2014 / 10:21) Tissue specific optimized AAV Plasmids are now available to make research & therapy more targeted Gene Therapy development progresses with tissue specific AAV Plasmids developed by Europe's leading commercial supplier of viral vectors, SIRION Biotech in Munich. Munich, 19 August 2014, viral vectors are a new class of biologics that help treat diseases caused by defective gene function / proteins ("gene therapy"). More than 20 companies worldwide the majority of which originate in the United States are applying viral vectors to conduct clinical studies. A key hurdle when applying viral vectors is to limit the transduction (gene transfer) to the appropriate cells of a specific tissue without affecting their surrounding environment. But, how can an expression system differentiate between tissue or even cell type? SIRION Biotech, in cooperation with U of Munich (LMU) and U of Cologne, has developed a line of viral vectors with specific promotors that are only active in a specific set of targetetd cells to initiate the desired gene expression. Using this method, the gene of interest is only being expressed in the targeted tissue which is relevant for the desired therapy. This improves the effectiveness of the therapy and also … Continue reading →

Ben Hirschler Tuesday, August 19, 2014 US biotech firm Celladon hopes that its Mydicar treatment can help Lee Adams and patients like him who have advanced heart failure and rely on devices to keep them alive until a donor heart becomes available. The treatment works by inserting a gene called SERCA2a - the lack of which makes hearts pump weakly - directly into heart cells via a catheter to repair them. The trial will evaluate how much of the gene is getting to the heart muscle and how well it is working. Adams is the first of 24 patients who will be given either the gene therapy or a placebo as part of a clinical study partially funded by the British Heart Foundation and sponsored by Imperial College London. "Advanced heart failure is a progressive condition that results in a poor quality of life and shortened life expectancy," said Nick Banner, the consultant cardiologist at Harefield Hospital who carried out the first infusion. "The best treatment currently available is a heart transplant but the shortage of donor organs means that many patients will die on the waiting list." Adams has been living for more than 2 years with a Left … Continue reading →

Gene therapy can offer an effective treatment for drug-resistant radio-insensitive cancer. However, progress has been hampered by the difficulties in developing an appropriate delivery mechanism. Now researchers have demonstrated for the first time that magnetic nanoparticles provide safe, effective and targeted "suicide-gene" delivery to cells of a particularly prevalent and highly resilient type of liver cancer. Because the nanoparticles are magnetic they can also be used for hyperthermia treatments, where magnetic energy is converted into heat to elevate the temperature of the surrounding cancerous tissue, increasing the overall therapeutic effect of the gene therapy. "Our in vivo and in vitro experiments showed that the gene therapy combined with the heating treatment was very effective," explains Chenyan Yuan, a researcher from Southeast University in China. "In mice, we saw that the tumour growth rate, volume and mass were significantly less in the combined treatment group compared to gene therapy and hyperthermia therapy alone." Yuan and colleagues from the Affiliated Zhong Da Hospital of Southeast University and Jiangsu Key Laboratory for Biomaterials and Devices in China equipped the magnetic nanoparticles with a tumour-specific promoter gene to specifically tackle hepatocellular carcinoma, which is the most common form of liver cancer and causes more … Continue reading →

British scientists have launched a pioneering trial to see whether gene therapy can potentially replace heart transplants. Lee Adams, a 37-year-old carpenter from Hertfordshire, is the first of 24 patients with advanced heart failure to be recruited. Like all the other participants, he has been fitted with a mechanical pump to keep his blood flowing while waiting for a suitable donor heart. SEE ALSO: Bears: Wise Teachers In The Fight Against Diabetes And Obesity How Smelling Farts Can Save Your Life (This Is Not A Prank) The study is the first in the world to investigate the use of gene therapy in heart failure patients kept alive by a Left Ventricular Assist Device (LVAD). Sixteen randomly chosen patients will be treated with a corrective gene to help their hearts beat more strongly. Eight others are to receive a "dummy" placebo therapy. Mr Adams, who has had an LVAD for more than two and a half years, does not know which group he is in because the trial is "blinded". He said: "Of course the best thing that could happen would be for my heart function to show signs of improvement and for the gene therapy to prove to be a … Continue reading →

The therapy involves injecting a harmless altered virus into the blood stream [GETTY/PIC POSED BY MODEL] Lee Adams, a 37-year-old carpenter, is the first patient in the world to take part in a gene therapy trial while wired up to a mechanical heart pump. The study is to investigate the use of the therapy on 24 patients with advanced heart failure, recruited from Harefield Hospital, London, and Papworth Hospital, Cambridgeshire. All the participants are kept alive by a Left Ventricular Assist Device while they wait for heart transplants. Many such patients endure agonising delays in finding a suitable donor. Sixteen randomly chosen patients will be treated with a corrective gene to help their hearts beat more strongly. Eight others will receive a dummy placebo therapy. Lee, from Rickmansworth, Herts does not know which group he is in. He said: Of course the best thing that could happen would be for my heart function to show signs of improvement and for the gene therapy to prove to be a miracle cure. No matter what the cause of the heart failure, the therapy should be equally beneficial for patients Read the original: Gene therapy could replace heart transplant operations … Continue reading →

Stamford, CT (PRWEB) August 11, 2014 Alliance for Cancer Gene Therapy, Inc. (ACGT) the nations only nonprofit dedicated exclusively to cell and gene therapies for cancer has announced that David Darst and Alexandra Landes have joined the foundations Board of Directors. Both Darst and Landes lost their mothers to cancer and share a commitment to uncovering innovative methods for treating cancer with reduced side-effects. ACGT is based in Stamford, CT and funds top physicians and researchers at medical institutions in the US and Canada. The foundation supports a number of gene therapy treatments including immunotherapy, which activates patients own immune systems to battle cancerous cells. Recently, the FDA granted fast-track status to an immunotherapy treatment for leukemia for which ACGT provided early funding. ACGT has given almost $25 million in grants to researchers since its founding in 2001 by Barbara Netter and her late husband, Edward. David Darst, a 32-year-old New York City resident, is a graduate of Harvard College and Harvard Business School. Darst has held positions with Pfizer, Orbimed Advisors, and a slate of other companies that are working to develop and finance new drugs in the biotech industry. Darst is currently the CEO at Rgenix, Inc., where … Continue reading →

PUBLIC RELEASE DATE: 11-Aug-2014 Contact: Franca Davenport f.davenport@imperial.ac.uk 44-207-594-3415 Imperial College London For the first time in the world, a patient with a mechanical heart pump has taken part in a new gene therapy trial for heart failure at Harefield Hospital, London. This is the start of a new clinical trial that will assess gene therapy for patients with heart pumps and provide detailed insight on its impact on the heart muscle. Heart failure occurs when the heart no longer pumps blood effectively and it affects hundreds of thousands of people in the UK. Some individuals with an advanced heart failure can be fitted with a Left Ventricular Assist Device (LVAD), which supports the failing heart and aims to restore normal blood flow. The LVAD is an electrically driven pump, moving the blood from the left ventricle into the main artery (aorta) so it can circulate the oxygen-rich blood to the rest of the body. Individuals with advanced heart failure who require a transplant may be fitted with an LVAD to keep them alive until a suitable donor heart becomes available. Currently there are around 100 to 150 people in the UK living with an LVAD. The new trial, led … Continue reading →

If successful the heart muscle would be able to pump blood around the body without any help. Mr Adams, of Hertfordshire, is the first person in the world to take part in the trial. "It took some getting used to, living with a (heart pump), he said. You can't just jump in the bath or the shower and it's difficult sleeping whilst being attached to it. Everywhere I go I have to carry the power supply and spare batteries in a backpack. "Of course the best thing that could happen would be for my heart function to show signs of improvement and for the gene therapy to prove to be a 'miracle cure' for myself and other patients. If it does prove to be successful it would be exciting for patients who need a transplant but end up on the waiting list for a long time because of the shortage of donors." Heart failure occurs when the heart no longer pumps blood effectively and it affects hundreds of thousands of people in the UK. Some patients with advanced heart failure are fitted with a Left Ventricular Assist Device (LVAD), a mechanical pump which supports the failing heart and aims to … Continue reading →

Gene Therapy, Trials in Down Syndrome by Siham Al-Lawati Gene Therapy, Trials in Down Syndrome. By: National Genetic Centre / … Continue reading →