Category Archives: Gene Therapy

In one of the biggest advances against leukemia and other blood cancers in many years, doctors are reporting unprecedented success by using gene therapy to transform patients blood cells into soldiers that seek and destroy cancer. A few patients with one type of leukemia were given this one-time, experimental therapy several years ago, and some remain cancer-free today. Now, at least six research groups have treated more than 120 patients with many types of blood and bone marrow cancers, with stunning results. Its really exciting, said Dr. Janis Abkowitz, blood diseases chief at the University of Washington in Seattle and president of the American Society of Hematology. You can take a cell that belongs to a patient and engineer it to be an attack cell. In one study, all five adults and 19 of 22 children with acute lymphocytic leukemia, commonly known as ALL, had a complete remission, meaning no cancer could be found after treatment, although a few have relapsed since then. These were gravely ill patients out of options. Some had tried multiple bone marrow transplants and up to 10 types of chemotherapy or other treatments. Cancer was so advanced in Emily Whitehead, now 8, of Philipsburg, Pa., … Continue reading →

Albany, New York (PRWEB) May 22, 2014 Gene therapy involves use of DNA as a pharmaceutical agent to treat diseases. It is one of the most important developments in the field of medicine that has potential to treat various lethal diseases such as HIV, cancer and cystic fibrosis. In the long run, biotechnology and clinical trial industries will benefit from developments in gene therapy and provide potential treatment solutions for various incurable diseases. Browse the full report - http://www.transparencymarketresearch.com/gene-therapy-market.html. In the present scenario, various pharmaceutical companies are using clinical data to validate the concept of gene therapy. Moreover, many venture capital investors are also showing their interest in gene therapy, and are investing heavily in its development. However, gene therapy is highly dependent on the regulatory approvals and most of the products are currently in clinical trial phase. Most of these gene therapy products are for cancer and cardiovascular diseases, and are in Phase III/ Phase II of clinical trials. In addition, growing popularity of DNA vaccines boost advances in gene therapy and is likely to be practiced in clinics in the near future, with a number of therapy programs now in phase II/III trials, showing promising results. Get report … Continue reading →

PUBLIC RELEASE DATE: 21-May-2014 Contact: Kathryn Ruehle kruehle@liebertpub.com 914-740-2100 Mary Ann Liebert, Inc./Genetic Engineering News New Rochelle, NY, May 21, 2014A targeted gene silencing strategy blocks production of the dysfunctional huntingtin (Htt) protein, the cause of Huntington's disease, a fatal, inherited neurodegenerative disorder. The effectiveness of this RNA interference (RNAi) approach in reducing levels of mutant Htt protein and disease symptoms in a mouse model of the disease is described in Human Gene Therapy, a peer-reviewed journal from Mary Ann Liebert, Inc., publishers. The article is available free on the Human Gene Therapy website. Lisa Stanek and coauthors from Genzyme (Framingham, MA) used an adeno-associated viral (AAV) vector to deliver a targeted nucleic acid sequence called a small interfering RNA (siRNA) into the cells of affected mice. The siRNA selectively binds to the mutated gene, blocking disease-causing Htt production. The authors present data demonstrating the ability to deliver the therapeutic RNAi into the cells, reduce mutant Htt levels, and impact behavioral deficits in the mice without causing any noticeable neurotoxicity, in their article "Silencing Mutant Huntingtin by Adeno-Associated Virus-Mediated RNA Interference Ameliorates Disease Manifestations in the YAC128 Mouse Model of Huntington's Disease." "The Genzyme group uses state-of-the-art delivery technology and … Continue reading →

PUBLIC RELEASE DATE: 22-May-2014 Contact: Kathryn Ruehle kruehle@liebertpub.com 914-740-2100 Mary Ann Liebert, Inc./Genetic Engineering News New Rochelle, NY, May 22, 2014To make up for insufficient amounts of SMN protein, the cause of the inherited neuromuscular disease spinal muscular atrophy (SMA), researchers have successfully delivered a replacement SMN1 gene directly to the spinal cords of animal models of SMA. A new study demonstrating that enough copies of the SMN1 gene can be delivered to the spinal cord motor neurons to extend the survival of the treated animals is published in Human Gene Therapy, a peer-reviewed journal from Mary Ann Liebert, Inc., publishers. The article is available free on the Human Gene Therapy website. Marco Passini and coauthors from Genzyme (Framingham, MA), University of California San Francisco, Emory University School of Medicine (Atlanta, GA), and Georgetown University Medical Center (Washington, DC) used an adeno-associated viral vector as the delivery vehicle to transport copies of the SMN1 gene into motor neurons in the spinal cord via intrathecal delivery. They report on the effectiveness of restoring the levels of functional SMN protein in normal pig and non-human primate SMA models that would predict efficacy based on gene transfer with the same vector in an … Continue reading →

Gene Therapy Biology Project for Swavely A project for my biology class. By: TeddyBearPower7 … Continue reading →

Germline Gene Therapy By: Farhana Zahari … Continue reading →

Gene Therapy (Using Biolistic gene gun for gene delivery) By: Kang Weiling … Continue reading →

Gene therapy for RTT syndrome benefits and drawbacks By: osama ashmawy … Continue reading →

At the forefront of medicine, Gene Therapy brings you the latest research into genetic and cell-based technologies to treat disease. It also publishes Progress & Prospects reviews and News and Commentary articles, which highlight the cutting edge of the field. Volume 21, No 5 May 2014 ISSN: 0969-7128 EISSN: 1476-5462 2012 Impact Factor 4.321* 70/290 Biochemistry & Molecular Biology 22/159 Biotechnology & Applied Microbiology 33/161 Genetics & Heredity 25/121 Medicine, Research & Experimental Editors: J Glorioso, USA N Lemoine, UK *2012 Journal Citation Reports Science Edition (Thomson Reuters, 2013) Download the presentation from our How to Get Published Session. (281 kB) Open Gene Therapy now offers authors the option to publish their articles with immediate open access upon publication. Open access articles will also be deposited on PubMed Central at the time of publication and will be freely available immediately. Find out more from our FAQs page. Reviews by top researchers in the field. See the recent Progress and Prospects articles. Essential topics explored in depth in Gene Therapy Special Issues. See the original post: Gene Therapy - Nature … Continue reading →

Spark Therapeutics, a gene therapy medical company, this week signed an agreement for a headquarters in West Philadelphia. Spark Therapeutics, spun out of Children's Hospital of Philadelphia in October with $50 million in capital, will build out a 28,000-square-foot facility at 3737 Market St. to house its business operations, clinical research and development, and manufacturing. Jeffrey D. Marrazzo, cofounder and chief executive, said the new facility "will support the continued expansion of our team and expand our manufacturing capacity to support our clinical development and commercial plans." Spark anticipates moving into its new headquarters and expanding to 50 full-time employees by the end of 2014. Spark is preparing to complete clinical development of its lead, Phase 3 clinical program to address inherited retinal dystrophies caused by mutations in the RPE65 gene. - Erin Arvedlund Excerpt from: Spark Therapeutics to open headquarters in West Philadelphia … Continue reading →