Category Archives: Gene Therapy

Despite bouncing off a 2-year low, biotech is still an unpopular sector and investors are rightfully concerned about its near-term prospects. Recent drug failures, growing pricing pressure and the potential impact of biosimilars all contribute to the negative sentiment, but the main problem is the lack of growth drivers for the remainder of 2016 (and potentially 2017). The biotech industry relies on innovation cycles to create new revenue sources. This was the case in the 2013-2014 biotech bull market, which was driven by a wave of medical breakthroughs (PD-1, HCV, CAR/TCR, oral MS drugs, CF etc.). These waves typically involve new therapeutic approaches coupled with disruptive technologies as their enablers. In oncology, for example, the understanding that cancer is driven by aberrant signaling coupled with advances in medicinal chemistry and antibody engineering led to the development of kinase inhibitors and monoclonal antibodies as blockers of signaling. A decade later, insights around cancer immunology gave rise to the immuno-oncology field and PD-1 inhibitors in particular, which are expected to become the biggest oncology franchise ever. Gene therapy ticks all the boxes While there are several hot areas in biotech such as gene editing and microbiome, most are still early and their … Continue reading →

Virtually all cells in the human body contain genes, making them potential targets for gene therapy. However, these cells can be divided into two major categories: somatic cells (most cells of the body) or cells of the germline (eggs or sperm). In theory it is possible to transform either somatic cells or germ cells. Gene therapy using germ line cells results in permanent changes that are passed down to subsequent generations. If done early in embryologic development, such as during preimplantation diagnosis and in vitro fertilization, the gene transfer could also occur in all cells of the developing embryo. The appeal of germ line gene therapy is its potential for offering a permanent therapeutic effect for all who inherit the target gene. Successful germ line therapies introduce the possibility of eliminating some diseases from a particular family, and ultimately from the population, forever. However, this also raises controversy. Some people view this type of therapy as unnatural, and liken it to "playing God." Others have concerns about the technical aspects. They worry that the genetic change propagated by germ line gene therapy may actually be deleterious and harmful, with the potential for unforeseen negative effects on future generations. Somatic cells … Continue reading →

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 →

Gene therapy is the therapeutic delivery of nucleic acid polymers into a patient's cells as a drug to treat disease.[1] The first attempt at modifying human DNA was performed in 1980 by Martin Cline, but the first successful and approved[by whom?] nuclear gene transfer in humans was performed in May 1989.[2] The first therapeutic use of gene transfer as well as the first direct insertion of human DNA into the nuclear genome was performed by French Anderson in a trial starting in September 1990. Between 1989 and February 2016, over 2,300 clinical trials had been conducted, more than half of them in phase I.[3] It should be noted that not all medical procedures that introduce alterations to a patient's genetic makeup can be considered gene therapy. Bone marrow transplantation and organ transplants in general have been found to introduce foreign DNA into patients.[4] Gene therapy is defined by the precision of the procedure and the intention of direct therapeutic effects. Gene therapy was conceptualized in 1972, by authors who urged caution before commencing human gene therapy studies. The first attempt, an unsuccessful one, at gene therapy (as well as the first case of medical transfer of foreign genes into humans … Continue reading →

What Is Gene Therapy? Explore the what's and why's of gene therapy research, includingan in-depth look at the genetic disorder cystic fibrosis and how gene therapy could potentially be used to treat it. Gene Delivery: Tools of the Trade Explore the methods for delivering genes into cells. Space Doctor You are the doctor! Design and test gene therapy treatments with ailing aliens. Challenges In Gene Therapy Researchers hoping to bring gene therapy to the clinic face unique challenges. Approaches To Gene Therapy Beyond adding a working copy of a broken gene, gene therapy can also repair or eliminate broken genes. Gene Therapy Successes The future of gene therapy is bright. Learn about some of its most encouraging success stories. Gene Therapy Case Study: Cystic Fibrosis APA format: Genetic Science Learning Center. (2012, December 1) Gene Therapy. Retrieved September 02, 2016, from http://learn.genetics.utah.edu/content/genetherapy/ CSE format: Gene Therapy [Internet]. Salt Lake City (UT): Genetic Science Learning Center; 2012 [cited 2016 Sep 2] Available from http://learn.genetics.utah.edu/content/genetherapy/ Chicago format: Genetic Science Learning Center. "Gene Therapy." Learn.Genetics.December 1, 2012. Accessed September 2, 2016. http://learn.genetics.utah.edu/content/genetherapy/. Original post: Gene Therapy - Learn Genetics … Continue reading →

Cystic fibrosis (CF) is a genetic disorder that affects mostly the lungs but also the pancreas, liver, kidneys, and intestine.[1][2] Long-term issues include difficulty breathing and coughing up mucus as a result of frequent lung infections. Other signs and symptoms include sinus infections, poor growth, fatty stool, clubbing of the fingers and toes, and infertility in males, among others. Different people may have different degrees of symptoms.[1] CF is inherited in an autosomal recessive manner. It is caused by the presence of mutations in both copies of the gene for the cystic fibrosis transmembrane conductance regulator (CFTR) protein.[1] Those with a single working copy are carriers and otherwise mostly normal.[3] CFTR is involved in production of sweat, digestive fluids, and mucus.[4] When CFTR is not functional, secretions which are usually thin instead become thick.[5] The condition is diagnosed by a sweat test and genetic testing.[1] Screening of infants at birth takes place in some areas of the world.[1] There is no cure for cystic fibrosis.[3] Lung infections are treated with antibiotics which may be given intravenously, inhaled, or by mouth. Sometimes the antibiotic azithromycin is used long term. Inhaled hypertonic saline and salbutamol may also be useful. Lung transplantation may … Continue reading →

Gene therapy is a way of inserting genes into a patient's cells and replacing the preexisting alleles, or gene variants, to perform some therapeutic function. It has been used thus far primarily to replace mutant defective genes, or alleles, with normal alleles, but could in theory be used to edit the human genome arbitrarily. If gene therapy were applied to reproductive cells in the gonads (the germline), these genetic changes would be heritable. This process has never been performed, but it has a name: germline genetic engineering. Since the early 1980s, gene therapy has been used to produce medicines. Say that a human being needs a certain protein as a medicine. This therapy uses a viral vector, that is, a virus modified to contain the DNA to be introduced. Large quantities of the virus are injected to the target area, or, sometimes tissue is removed, infected with the virus, and then implanted again. The viruses are modified such that the vast majority are not capable of independent self-replication - providing little chance for pathogenic infection. The virus introduced the new DNA into the genome of human cells, much in the same way normal viruses introduce their own genetic material into … Continue reading →