Category Archives: Gene Therapy

The Center for Biologics Evaluation and Research (CBER) regulates cellular therapy products, human gene therapy products, and certain devices related to cell and gene therapy. CBER uses both the Public Health Service Act and the Federal Food Drug and Cosmetic Act as enabling statutes for oversight. Cellular therapy products include cellular immunotherapies, and other types of both autologous and allogeneic cells for certain therapeutic indications, including adult and embryonic stem cells. Human gene therapy refers to products that introduce genetic material into a persons DNA to replace faulty or missing genetic material, thus treating a disease or abnormal medical condition. Although some cellular therapy products have been approved, CBER has not yet approved any human gene therapy product for sale. However, the amount of cellular and gene therapy-related research and development occurring in the United States continues to grow at a fast rate. CBER has received many requests from medical researchers and manufacturers to study cellular and gene therapies and to develop cellular and gene therapy products. In addition to regulatory oversight of clinical studies, CBER provides proactive scientific and regulatory advice to medical researchers and manufacturers in the area of novel product development. Read more from the original source: … Continue reading →

Germline Gene Transfer Gene transfer represents a relatively new possibility for the treatment of rare genetic disorders and common multifactorial diseases by changing the expression of a person's genes. Typically gene transfer involves using a vector such as a virus to deliver a therapeutic gene to the appropriate target cells. The technique, which is still in its infancy and is not yet available outside clinical trials, was originally envisaged as a treatment of monogenic disorders, but the majority of trials now involve the treatment of cancer, infectious diseases and vascular disease. Human gene transfer raises several important ethical issues, in particular the potential use of genetic therapies for genetic enhancement and the potential impact of germline gene transfer on future generations. Gene transfer can be targeted to somatic (body) or germ (egg and sperm) cells. In somatic gene transfer the recipient's genome is changed, but the change is not passed on to the next generation. In germline gene transfer, the parents' egg and sperm cells are changed with the goal of passing on the changes to their offspring. Germline gene transfer is not being actively investigated, at least in larger animals and humans, although a great deal of discussion is … Continue reading →

Viral vectors have natural host cell populations that they infect most efficiently. Retroviruses have limited natural host cell ranges, and although adenovirus and adeno-associated virus are able to infect a relatively broader range of cells efficiently, some cell types are refractory to infection by these viruses as well. Attachment to and entry into a susceptible cell is mediated by the protein envelope on the surface of a virus. Retroviruses and adeno-associated viruses have a single protein coating their membrane, while adenoviruses are coated with both an envelope protein and fibers that extend away from the surface of the virus. The envelope proteins on each of these viruses bind to cell-surface molecules such as heparin sulfate, which localizes them upon the surface of the potential host, as well as with the specific protein receptor that either induces entry-promoting structural changes in the viral protein, or localizes the virus in endosomes wherein acidification of the lumen (anatomy) induces this refolding of the viral coat. In either case, entry into potential host cells requires a favorable interaction between a protein on the surface of the virus and a protein on the surface of the cell. For the purposes of gene therapy, one might … Continue reading →

noun, Medicine/Medical. 1. the application of genetic engineering to the transplantation of genes into human cells in order to cure a disease caused by a genetic defect, as a missing enzyme. British Dictionary definitions for gene therapy Expand the replacement or alteration of defective genes in order to prevent the occurrence of such inherited diseases as haemophilia. Effected by genetic engineering techniques, it is still at the experimental stage gene therapy in Medicine Expand gene therapy n. A technique for the treatment of genetic disease in which a gene that is absent or defective is replaced by a healthy gene. gene therapy in Science Expand gene therapy in Culture Expand A promising technology that involves replacing a defective gene in the body with a healthy one. This can be done by removing cells from the body, using genetic engineering techniques to change defective sequences in the DNA, and then reinserting the cells. This technique has been carried out successfully, for example, on bone marrow cells, in which defective cells were successfully replaced with healthy, genetically engineered cells. Scientists hope to find an agent, such as a therapeutic virus, that will be able to correct defective DNA in situ. (See cloning … Continue reading →

Cystic fibrosis (CF) is the most common, classic mendelian autosomal recessive, life-limiting disease among the white population.1,2 It is a multisystem disease that results from loss of function in the CF transmembrane conductance regulator (CFTR) gene, classically leading to respiratory tract, gastrointestinal (GI), pancreatic, and reproductive abnormalities.2 CF was recognized as a distinct clinical entity in 1938 and was believed to be invariably fatal during infancy.3 Since the 1970s, the life spans of CF patients have been prolonged, with advances in early diagnosis, care, and disease therapy. Early diagnosis has been improved by newborn screening. Advances in care include management of meconium ileus and improved methods of sputum clearance and managing respiratory failure. Improvements in disease therapy include better antibiotics, especially macrolides, and better pancreatic enzymes. With current management, almost 80% of patients with CF will reach adulthood; thus, CF is no longer a purely pediatric disease.4-6 For patients born in the 1990s, the median survival is predicted to be greater than 40 years.5 As more CF patients are surviving longer, adult issues including careers, relationships, and family are becoming important.6 A range of comorbid conditions that are more prevalent in adult CF patients are also being encountered with increasing … Continue reading →

Gene therapy is an experimental treatment that involves inserting genetic material into your cells to give them a new function or restore a missing function, as cancer may be caused by damaged or missing genes, also known as gene mutations. Although gene therapy may be one way to overcome these changes and treat or prevent cancer, it is currently only available through clinical trials. Cancer is caused by changes in our genes. Genes are inherited from our parents, and determine our traits and characteristics. They are made of biological molecules called deoxyribonucleic acid (DNA) and ribonucleic acid (RNA). DNA and RNA are responsible for making proteins, which have many functions, such as helping a cell to maintain its shape or controlling its growth and division. Changes or mutations in genes can affect the proteins and may sometimes lead to diseases, such as cancer. Gene therapy is designed to modify cancer cells at the molecular level and replace a missing or bad gene with a healthy one. The new gene is delivered to the target cell via a vector, which is usually an inactive virus or liposome, a tiny fat bubble. Gene therapy can be done in two ways: outside (ex … Continue reading →

Editor-in-Chief: Terence R. Flotte, MD Deputy Editor, Europe: Thierry VandenDriessche, PhD Deputy Editor, U.S.: Barry J. Byrne, MD, PhD Human Gene Therapy Editor: Guangping Gao, PhD Methods Editor: Hildegard Bning, PhD Clinical Development Editor: James M. Wilson, MD, PhD Latest Impact Factor* is 3.755 *2014 Journal Citation Reports published by Thomson Reuters, 2015 Human Gene Therapy is the premier, multidisciplinary journal covering all aspects of gene therapy. The Journal publishes in-depth coverage of DNA, RNA, and cell therapies by delivering the latest breakthroughs in research and technologies. Human Gene Therapy provides a central forum for scientific and clinical information, including ethical, legal, regulatory, social, and commercial issues, which enables the advancement and progress of therapeutic procedures leading to improved patient outcomes, and ultimately, to curing diseases. The Journal is divided into three parts. Human Gene Therapy, the flagship, is published 12 times per year. HGT Methods, a bimonthly journal, focuses on the applications of gene therapy to product testing and development. HGT Clinical Development, a quarterly journal, serves as a venue for publishing data relevant to the regulatory review and commercial development of cell and gene therapy products. Human Gene Therapy was voted one of the most influential journals in … Continue reading →

In Lung Cancer, Not All HER2 Alterations Are Created Equal Jan. 28, 2016 Study shows two distinct causes of HER2 activation in lung cancer: mutation of the gene and amplification of the gene. In patient samples of lung adenocarcinoma, 3 percent were found to have HER2 ... read more Dec. 12, 2015 Results from a long-term clinical trial conducted by cancer researchers show that combining radiation treatment with 'suicide gene therapy' provides a safe and effective one-two punch ... read more Gene Therapy Used to Extend Estrogen's Protective Effects on Memory Dec. 8, 2015 The hormone estrogen helps protect memory and promote a healthy brain, but this effect wanes as women age, and even estrogen replacement therapy stops working in humans after age 65. Now researchers ... read more Shrinking Tumors With an RNA Triple-Helix Hydrogel Glue Dec. 7, 2015 An efficient and effective delivery vehicle for gene therapy has been developed by researchers who have used it to shrink tumors by nearly 90 percent in a pre-clinical model of triple-negative breast ... read more Characteristics That May Increase a Breast Cancer Survivor's Risk of Developing Leukemia Following Treatment Identified Dec. 7, 2015 A new analysis indicates that certain characteristics … Continue reading →

A few years ago, a clinical trial began in France in the hope of curing children with a type of genetic immune deficiency called SCID-X1. Children with this disease have a defective gene, called gamma-c, which prevents a subset of the cells of the immune system from forming, and predisposes the children to life-threatening infections. In an attempt to cure the childrenwho would otherwise die at a young agephysicians used gene therapy to provide them with normal gamma-c genes. This particular trial has had striking success as well as tragedy. Eight of the eleven children are currently thriving. However, in two cases the therapy successfully introduced gamma-c genes, but these children have since developed leukemia. In both children, a gamma-c gene inserted next to another gene, called LMO2. The LMO2 gene has previously been linked to leukemia, and scientists speculate that the insertion of the gamma-c gene next to LMO2 may have overstimulated the gene, causing T cells to proliferate in excess. An LMO2 effect, in combination with the proliferation-inducing effects of the gamma-c gene itself, may be the cause of the leukemia in these two patients. Scientists are still investigating other possible causes. From this single trial, it is … Continue reading →

A treatment for Cystic Fibrosis. A cure for AIDS. The end of cancer. That's what the newspapers promised us in the early 1990's. Gene therapy was the answer to what ailed us. Scientists had at last learned how to insert healthy genes into unhealthy people. And those healthy genes would either replace the bad genes causing diseases like CF, sickle-cell anemia and hemophilia or stimulate the body's own immune system to rid itself of HIV and some forms of cancer. A decade later, none of these treatments have come to fruition and research into gene therapy has become politically unpopular, making clinical trials hard to approve and research dollars hard to come by. But some researchers who are taking a different approach to gene therapy could be on the road to more success than ever before. - - - - - - - - - - - - Early Promise Almost as soon as Watson and Crick unwound the double helix in the 1950's, researchers began considering the possibility- and ethics- of gene therapy. The goals were lofty- to fix inherited genetic diseases such as Cystic Fibrosis and hemophilia forever. Gene therapists planned to isolate the relevant gene in question, … Continue reading →