Register 3 for the price of 2 with the coupon code rcdvb! Genomics & Big Data Summit 2017 June 8-9, 2017 2nd CRISPR & Genome Engineering 4th Cell & Gene Therapy 2nd Big Data Bioinformatics BECOME A SPONSOR GTCbio offers comprehensive sponsorship packages for all our meetings, including presentation opportunities, exhibit and booth space, as well as the event delegate list. All our packages can be customized to meet your company's budget. For information, please contact: Amber Kempf 626-256-6405 x110 amber.kempf [at] gtcbio.com Dear Colleague, We invite you to attend the 3rd Cell & Gene Therapy Conference, which will take place June 8-9, 2017in San Francisco, CA. The conference will bring together an exciting balance of industry and academia, so that delegates have the unique opportunity to network with colleagues and gain fresh perspective on the various research. Treatment of disease using cell and gene therapy is now a reality. With emerging technologies and new clinical trials being developed and launched at a growing rate, this meeting will discuss the evolving regulatory challenges and pre-clinical requirements, emerging technologies, the development and applications of cell and gene therapy, as well as business models and commercial milestones. The 4th Cell & Gene … Continue reading →

In amyotrophic lateral sclerosis (ALS), gene therapy may help if it can deliver a beneficial protein to salvage dying nerve cells. Gene therapy can be simply a means to boost on site production of a helpful factor, at places where nerve cells are in trouble. Researchers can disarm many types of viruses, and put in the genetic instructions to make therapeutic protein. The redesigned viruses are called vectors. They are simply carriers for therapeutic genes. Knowledge of the SOD1 mutations linked to some forms of ALS has produced a vast body of evidence, pointing to a general strategy for the disease that might be successfully implemented through gene therapy. Gene therapy is the use of genetic instructions to produce a protein to treat a disorder or deficiency. It can aid in a disease even if the therapy is not directly targeting a gene defect that causes the disease. In amyotrophic lateral sclerosis (ALS), gene therapy may help if it can deliver a beneficial protein, to salvage dying nerve cells. The gene therapy simply is a means to boost on site production of a trophic (growth enhancing) factor, at places where nerve cells are in trouble. Genes are the molecules in … Continue reading →

What is Gene Therapy? Gene therapy is a technique used to correct defective genes genes that are responsible for disease development. Specifically, according to the American Society of Gene and Cell Therapy- Gene therapy is defined as a set of strategies that modify the expressionof an individuals genes or that correct abnormal genes. Each strategyinvolves the administration of a specific DNA (or RNA). Gene therapy is the manipulation of the expression of specific genes in a persons body, in hopes of treating a disease or disorder. Gene therapy is still considered experimental and only available via clinical trial. Although many successful trials have been documented (see Interesting Links below), gene therapy has a checkered history. In some gene therapy trials, there were cases of leukemia as an unintended side-effect, and even cases of death (see link on Jesse Gelsinger below). Image courtesy of Wikimedia Commons How Does Gene Therapy Work? Although there are several strategies for gene therapy, the most commonly used method involves inserting a therapeutic gene into the genome to replace the abnormal or disease-causing gene. The gene that is inserted is delivered into a target cell via a vector. Usually, this vector is a virus, although non-viral … Continue reading →

Gene Therapy for Diseases Gene Therapy has made important medical advances in less than two decades. Within this short time span, it has moved from the conceptual stage to technology development and laboratory research to clinical translational trials for a variety of deadly diseases. Among the most notable advancements are the following: Severe Combined Immune Deficiency (ADA-SCID) ADA-SCID is also known as the bubble boy disease. Affected children are born without an effective immune system and will succumb to infections outside of the bubble without bone marrow transplantation from matched donors. A landmark study representing a first case of gene therapy "cure," or at least a long-term correction, for patients with deadly genetic disorder was conducted by investigators in Italy. The therapeutic gene called ADA was introduced into the bone marrow cells of such patients in the laboratory, followed by transplantation of the genetically corrected cells back to the same patients. The immune system was reconstituted in all six treated patients without noticeable side effects, who now live normal lives with their families without the need for further treatment. Chronic Granulomatus Disorder (CGD) CGD is a genetic disease in the immune system that leads to the patients' inability to fight … Continue reading →