Category Archives: Gene Therapy

Chuck Bednar for redOrbit.com Your Universe Online A new stem cell gene therapy developed by researchers at UCLA is set to begin clinical trials early next year after the technique reportedly cured 18 children who were born without working immune systems due to a condition known as ADA-deficient Severe Combined Immunodeficiency (SCID) or Bubble Baby disease. The treatment was developed by Dr. Donald Kohn, a member of the UCLA Eli and Edythe Broad Center of Regenerative Medicine and Stem Cell Research, and his colleagues, and according to the university, it is able to identify and correct faulty genes by using the DNA of the youngsters born with this life-threatening condition. Left untreated, ADA-deficient SCID is often fatal within the first year of a childs life, reports Peter M. Bracke for UCLA. However, after more than three decades of research, Dr. Kohns team managed to develop a gene therapy that can safely restore the immune systems of children with the disease by using their own cells and with no noticeable side effects. 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 … Continue reading →

HOPE, HYPE REALITY: ZINC FINGER GENE THERAPY TO CONTROL OR CURE HIV A video to answer your questions about a gene therapy approach to an HIV cure. On Nov 5, 2014, the defeatHIV Community Advisory Board hosted a talk with Dr. Philip D. Gregory (Chief Scientific... By: defeatHIV … Continue reading →

Roughly 40 million people across the world are blind and, for a long time, most forms of blindness were permanent conditions. The same situation held for degenerative diseases that affect eyesight. But recently, scientists have made some surprising headway into changing that. New treatments like gene therapy, stem-cell therapy, and even bionic implants are already starting to restore some patients' sight. And these technologies are expected to keep improving in the future. Here's a look at all the ways scientists have tried and, increasingly, succeeded in curing the blind: Children's Hospital of Philadelphia, Daniel Burke/AP Photo This undated image released by the Children's Hospital of Philadelphia shows doctors Albert Maguire, left, along with wife Jean Bennett at the University of Pennsylvania. The two are part of two teams of scientists in the United States and Britain that are using gene therapy to dramatically improve vision in four patients with an inherited eye disease that causes blindness in children. Tweaking genes is one promising route to treat blindness. In 2011, a group led by Jean Bennett of the University of Pennsylvania used gene therapy to treat some patients with a congenital blindness disorder. The patients in question all hada hereditary disease … Continue reading →

Future Gene Therapy for Cancer Future Gene Therapy for Cancer. Video streamed by http://www.AllthingsScience.com. By: Davies Robinson … Continue reading →

By Cyndi Root The Vaccine & Gene Therapy Institute of Florida (VGTI), a non-profit research institute, and TapImmune have formed a partnership to develop TapImmunes cancer vaccines. The companies announced the collaboration in a press release stating that they will move experimental vaccines for breast and ovarian cancers into Phase 2 clinical trials. Keith Knutson, PhD, VGTIs Director of Cancer Vaccines and Immune Therapies Program, explained the need for vaccines, All it takes is a few malignant cells to continue to circulate in the body until they eventually anchor and metastasize. Because these cancer cells already survived primary therapy, they are typically drug-resistant and much more difficult to treat. VGTI and TapImmune Agreement VGTI and TapImmune have agreed to coordinate efforts on cancer vaccines, including study design and trial site selection. VGTI will work with TapImmune to recruit clinical advisors, select manufacturers, and procure outsourced resources as necessary. The two will also work together in executing the clinical trials. Upon successful regulatory approvals, TapImmune holds the exclusive commercialization rights for the vaccines. Cancer Vaccine Candidates Investigators from VGTI and TapImmune hope to vaccinate women who have achieved remission in their breast or ovarian cancer in order to prevent cancer recurrence. … Continue reading →

SOUTH BEND, Ind.--- Little Hunter Miller's motor is always running. Like most toddlers he's sometimes one step away from trouble, but for Hunter being rough and tumble can have serious side effects. Hunter has severe hemophilia. Twenty-thousand Americans live with hemophilia; it's a condition preventing the blood from clotting easily after a cut or injury. Patients are also more susceptible to internal bleeding, which can damage joints, organs and tissue. Three days after Hunter was born a routine circumcision caused a major scare. "You know a baby gets up in the morning and their diapers are just full, said Tina Miller, Hunters grandmother. Well his was full, but it was full of blood." Doctors diagnosed Hunter with hemophilia a, which means his blood is missing a protein known as clotting factor eight. When he gets hurt doctors need to inject the clotting factor to stop the bleeding. He's had eight emergency room visits in 19 months. "Him falling, bumping his head too hard; just little cuts, said Heather Frederick, Hunters mother. He cut the roof of his mouth with a tortilla chip and that was a hospital trip." Original post: Doctors working on gene therapy to help patients with hemophilia … Continue reading →

Delivering stem cell factor directly into damaged heart muscle after a heart attack may help repair and regenerate injured tissue, according to a study led by researchers from Icahn School of Medicine at Mount Sinai presented November 18 at the American Heart Association Scientific Sessions 2014 in Chicago, IL. "Our discoveries offer insight into the power of stem cells to regenerate damaged muscle after a heart attack," says lead study author Kenneth Fish, PhD, Director of the Cardiology Laboratory for Translational Research, Cardiovascular Research Center, Mount Sinai Heart, Icahn School of Medicine at Mount Sinai. In the study, Mount Sinai researchers administered stem cell factor (SCF) by gene transfer shortly after inducing heart attacks in pre-clinical models directly into damaged heart tissue to test its regenerative repair response. A novel SCF gene transfer delivery system induced the recruitment and expansion of adult c-Kit positive (cKit+) cardiac stem cells to injury sites that reversed heart attack damage. In addition, the gene therapy improved cardiac function, decreased heart muscle cell death, increased regeneration of heart tissue blood vessels, and reduced the formation of heart tissue scarring. "It is clear that the expression of the stem cell factor gene results in the generation … Continue reading →

Bobby Gaspar: "Translational research in the ex vivo gene therapy of monogenic diseases" Educational Day* at ESGCT Conference in Madrid. "Translational research in the ex vivo gene therapy of monogenic diseases". Bobby Gaspar, Professor of Paedi... By: European Society for Gene and Cell Therapy … Continue reading →

Chiara Bonini: "Bases of Gene Therapy in leukemias" Chiara Bonini, dept. of Experimental Hematology at Fondazione San Raffaele del Monte Tabor - Milano, talks about "Bases of Gene Therapy in leukemias". http:/... By: European Society for Gene and Cell Therapy … Continue reading →

Contact Information Available for logged-in reporters only Newswise (MEMPHIS, Tenn. November 19, 2014) Gene therapy developed at St. Jude Childrens Research Hospital, University College London (UCL) and the Royal Free Hospital has transformed life for men with a severe form of hemophilia B by providing a safe, reliable source of the blood clotting protein Factor IX that has allowed some to adopt a more active lifestyle, researchers reported. The results appear in the November 20 edition of The New England Journal of Medicine. In this study, participants received one of three doses of gene therapy that used a modified adeno-associated virus (AAV) 8 as the vector to deliver the genetic material for making Factor IX. The vector was administered as a single infusion into a peripheral vein in the arm of each participant. Factor IX levels rose in all 10 men with severe hemophilia B following gene therapy and have remained stable for more than four years. Overall, episodes of spontaneous bleeding declined 90 percent. Use of Factor IX replacement therapy dropped about 92 percent in the first 12 months after the treatment with the investigational therapy. In the six participants who received the highest gene therapy dose, levels of … Continue reading →