Category Archives: Gene Therapy

LOS ANGELES (KABC) -- In bright daylight, 10-year-old Mark Devoe has no trouble seeing his friends. But inside or even in the shade, Mark's eyes sometimes don't work. "I have trouble seeing like, trees, when the road ends, and when there's like a drop there," said Mark. At age 6, Mark's doctors diagnosed him with the genetic condition choroideremia, which causes people to progressively lose vision until they are completely blind. "I don't know what it's like to live in darkness, but I've seen it," said Susan Devoe, Mark's mother. Susan is a carrier of the blindness gene. Mark's grandfather has the condition. "Watching my father go blind was devastating," said Susan. "I was a little girl. You know, you count on daddy to do things, and daddy couldn't do them." Dr. Jean Bennett is one of two U.S. researchers preparing to test a gene therapy for choroideremia in humans. "I think gene therapy holds a huge promise for developing treatments for blinding diseases," said Bennett. Researchers will use a virus carrying a small choroideremia gene and inject the virus just under the retina. The gene should begin to work in a few weeks. Go here to see the original: … Continue reading →

Gene Therapy: Hemophilia Twenty-thousand Americans are living with hemophilia, a condition that prevents the blood from clotting easily after a cut or injury. By: NewsChannel 5 … Continue reading →

Sickle Cell Anemia Gene Therapy By: Candace Kesselring … Continue reading →

Ruben Hernandez: "Recent developments in the gene therapy of solid tumours" Educational Day* at ESGCT Conference in Madrid. Rubn Hernndez, PI in Cancer Gene Therapy at Universita de Nvarra (University of Navarra, Spain) gives a le... By: European Society for Gene and Cell Therapy … Continue reading →

Odile Cohen: "Ethic issues in cell and gene therapy" Dr Odile COHEN HAGUENAUER, MCU-PH at Hpital Saint-Louis and School of Medicine at Denis Diderot University (Paris 7), talks about "Ethical Issues in Cell an... By: European Society for Gene and Cell Therapy … Continue reading →

Personalized Gene Therapy Reducing HIV In Some Patients For the first-time ever, doctors at the University of Pennsylvania have successfully used personalized gene therapy in a dozen patients with HIV, knocking the virus down to the point where... By: NewsChannel 5 … Continue reading →

Contact Information Available for logged-in reporters only Newswise UCLA stem cell researchers have pioneered a stem cell gene therapy cure for children born with adenosine deaminase (ADA)-deficient severe combined immunodeficiency (SCID), often called Bubble Baby disease, a life-threatening condition that if left untreated can be fatal within the first year of life. The groundbreaking treatment was developed by renowned stem cell researcher and UCLA Eli and Edythe Broad Center of Regenerative Medicine and Stem Cell Research member Dr. Donald Kohn, whose breakthrough was developed over three decades of research to create a gene therapy that safely restores immune systems in children with ADA-deficient SCID using the patients own cells with no side effects. To date, 18 children with SCID have been cured of the disease after receiving the stem cell gene therapy in clinical trials at UCLA and the National Institutes of Health. All of the children with SCID that I have treated in these stem cell clinical trials would have died in a year or less without this gene therapy, instead they are all thriving with fully functioning immune systems said Kohn, a professor of pediatrics and of microbiology, immunology and molecular genetics in Life Sciences. To protect children … Continue reading →

The gene therapy that has seemingly cured a 2-year-old girl could be used to treat other diseases, like Sickle Cell Anemia. UCLA DR. DONALD KHON has perfected the treatment that has now affected the lives of over a dozen families, including the parents of Evangelina, who was born with the deadly SCID. The immune system disease is known by its common name, Bubble Baby disease, because the children have to live in bubbles, since their immune systems do not function. Most die within the first year of life, unless they can get and survive a bone marrow transplant. Evangelina's twin sister, Annabella, was not a bone marrow transplant. So Dr Kohn included the sick twin in his study, transfering the patient's own bone marrow to the child. At two, her immune system is working well. It's a difficult and time consuming process, but it seems to work. The same procedure could work with other blood diseases that are curable with bone marrow transfusions. That includes sickle cell anemia, which will be the focus of the next trial. The implications are huge, as there are 30 or 40 diseases where the treatment could work. Follow this link: Bubble Baby: Gene Therapy … Continue reading →

Some of the most promising and powerful applications of genetic engineering are in the field of medicine. Researchers are using it to diagnose and predict disease, and to develop therapies and drugs to treat devastating diseases like cancer, Alzheimer's, diabetes, and cystic fibrosis. Explore more about one of the common uses of genetic engineering in medicine; gene therapy. Gene therapy can theoretically be used to treat, cure or even prevent disease. Gene therapy is still very experimental and controversial, with some stunning success stories and some devastating failures attributed to its use. Uses Gene therapy can be used to fix defective genes or to replace missing genes. Many diseases are the result of just one gene malfunctioning; sickle cell anemia, cystic fibrosis, SCID, are all caused by one defective gene. To correct the problem, gene therapy is used to deliver genes that function correctly. Other diseases are the result of a missing gene; juvenile Paget's disease - an extremely rare bone metabolism disorder - is one example. In those cases, gene therapy can be used to deliver genes to replace the missing one. Delivery Just like a mailtruck delivers mail, or the UPS truck delivers packages, there has to be … Continue reading →

Dr Zwerling on Gene Therapy Ian's school project. By: Ian Zwerling … Continue reading →