Category Archives: Gene Therapy

Researchers have been working for decades to bring gene therapy to the clinic, yet very few patients have received any effective gene-therapy treatments. But that doesn't mean gene therapy is an impossible dream. Even though gene therapy has been slow to reach patients, its future is very encouraging. Decades of research have taught us a lot about designing safe and effective vectors, targeting different types of cells, and managing and minimizing immune responses in patients. We've also learned a lot about the disease genes themselves. Today, many clinical trials are underway, where researchers are carefully testing treatments to ensure that any gene therapy brought into the clinic is both safe and effective. Below are some gene therapy success stories. Successes represent a variety of approachesdifferent vectors, different target cell populations, and both in vivo and ex vivo approachesto treating a variety of disorders. Sebastian Misztal was a patient in a hemophilia gene therapy trial in 2011. Following the treatment, Misztal no longer had spontaneous bleeding episodes. Credit: UCLH/UCL NIHR Biomedical Research Centre Several inherited immune deficiencies have been treated successfully with gene therapy. Most commonly, blood stem cells are removed from patients, and retroviruses are used to deliver working copies … Continue reading →

An experimental gene therapy has improved eyesight in patients with a rare, inherited eye disease that can cause blindness, according to its developer, Spark Therapeutics Inc. The company said it plans to apply to the Food and Drug Administration next year for approval to market the treatment. If accepted, this would be the first gene therapy to win approval in the United States. "We saw substantial restoration of vision in patients who were progressing toward complete blindness," Dr. Albert M. Maguire, principal investigator in the trial and professor of ophthalmology at the Perelman School of Medicine of the University of Pennsylvania, said in a statement. There were no serious adverse effects associated with the treatment, the company reported. The news comes about a week after a hospital in the U.K. announced it had performed a pioneering human embryonic stem cell operation on a patient with the aim of curing a different, more common cause of blindness known as macular degeneration. Gene therapy involves injecting genetic material into a person's cells to treat or prevent a disease. While research in this area has been going on since the late 1990's, no such treatment has been approved in the U.S. The European … Continue reading →

Researchers are looking at different ways of using gene therapy, including Some types of gene therapy aim to boost the body's natural ability to attack cancer cells. Our immune system has cells that recognise and kill harmful things that can cause disease, such as cancer cells. There are many different types of immune cell. Some of them produce proteins that encourage other immune cells to destroy cancer cells. Some types of therapy add genes to a patient's immune cells to make them better at finding or destroying particular types of cancer. There are a few trials using this type of gene therapy in the UK. Some gene therapies put genes into cancer cells to make the cells more sensitive to particular treatments such as chemotherapy or radiotherapy. This type of gene therapy aims to make the other cancer treatments work better. Some types of gene therapy deliver genes into the cancer cells that allow the cells to change drugs from an inactive form to an active form. The inactive form of the drug is called a pro drug. After giving the carrier containing the gene, the doctor gives the patient the pro drug. The pro drug may be a tablet … Continue reading →

Much of today's cancer research is devoted to finding missing or defective genes that cause cancer or increase an individual's risk for certain types of cancer. Gene research at MDAnderson has resulted in many important discoveries. We identified the mutated multiple advanced cancers gene (MMAC1) involved in some common cancers. We also performed the first successful correction of a defective tumor suppressor gene (p53) in human lung cancer. Current gene therapies are experimental, and many are still tested only on animals. There are some clinical trials involving a very small number of human subjects. The potential benefits of gene therapy are two-fold: The focus of most gene therapy research is the replacement of a missing or defective gene with a functional, healthy copy, which is delivered to target cells with a "vector." Viruses are commonly used as vectors because of their ability to penetrate a cells DNA. These vector viruses are inactivated so they cannot reproduce and cause disease. Gene transfer therapy can be done outside the body (ex vivo) by extracting bone marrow or blood from the patient and growing the cells in a laboratory. The corrected copy of the gene is introduced and allowed to penetrate the cells … Continue reading →

Gene therapy is designed to introduce genetic material into cells to compensate for abnormal genes or to make a beneficial protein. If a mutated gene causes a necessary protein to be faulty or missing, gene therapy may be able to introduce a normal copy of the gene to restore the function of the protein. A gene that is inserted directly into a cell usually does not function. Instead, a carrier called a vector is genetically engineered to deliver the gene. Certain viruses are often used as vectors because they can deliver the new gene by infecting the cell. The viruses are modified so they cant cause disease when used in people. Some types of virus, such as retroviruses, integrate their genetic material (including the new gene) into a chromosome in the human cell. Other viruses, such as adenoviruses, introduce their DNA into the nucleus of the cell, but the DNA is not integrated into a chromosome. The vector can be injected or given intravenously (by IV) directly into a specific tissue in the body, where it is taken up by individual cells. Alternately, a sample of the patients cells can be removed and exposed to the vector in a laboratory … Continue reading →

The mission of the Center for Gene Therapy is to investigate and employ the use of gene and cell based therapeutics for prevention and treatment of human diseases including: neuromuscular and neurodegenerative diseases, lysosomal storage disorders, ischemia and re-perfusion injury, neonatal hypertension, cancer and infectious diseases. Learn about our areas of focus and featured research. The National Institutes of Health has designated the Center for Gene Therapy as a Paul D. Wellstone Muscular Dystrophy Cooperative Research Center (MDCRC). MDCRCs promote basic, translational and clinical research and provide important resources that can be shared within the national muscle biology and neuromuscular research communities. The MDCRC will allow Nationwide Children's researchers to further develop methods to overcome immune barriers to gene correction for Duchenne muscular dystrophy. The Center for Gene Therapy and the Viral Vector Core are home to a Good Manufacturing Practice (GMP) production facility for manufacture of clinical-grade rAAV vectors.View the Viral Vector Core & Clinical Manufacturing Facility site. Investigators with the Center for Gene Therapy currently are conducting numerous clinical research studies, especially for neuromuscular disorders. The OSU and Nationwide Children's Muscle Group brings together investigators with diverse research interests in skeletal muscle, cardiac muscle, and neuromuscular biology. Learn … Continue reading →

Gene therapy that causes people to automatically make more omega 3 DHA benefits babies Gene therapy that improves the function of what researchers describe as Sprechers Shunt causes people to make more Omega 3 lipids physiologically Thus throughout their existence they are... By: beinganangeltreon … Continue reading →

Gene therapy Meaning Video shows what gene therapy means. Any of several therapies involving the insertion of genes into a patient's cells in order to replace defective ones. Gene therapy Meaning. How to pronounce,... By: ADictionary … Continue reading →

Download Non Viral Vectors for Gene Therapy, Volume 89 Physical Methods and Medical Translation PDF Download PDF Here: http://bit.ly/1EbW1Ek. By: Milford Schultz … Continue reading →

Mesothelioma Gene Therapy Treatments for Veterans Weitz Luxenberg, PC | 866-468-1553 | http://www.weitzlux.com What is Mesothelioma? Mesothelioma is caused by inhalation exposure to asbestos. There is no cure for mesothelioma. Many of... By: Weitz Luxenberg … Continue reading →