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. Childhood Cancer and the Promise of Gene Therapy At its most basic level, gene therapy is a powerful technique for correcting mistakes (called mutations) in DNA of human cells. Lately, the therapy has been gaining traction as a potentially life-saving treatment for children with an array of inherited rare blood and immune disorders, as well as certain cancers.Gene therapies are being carefully tested in clinical trials, using improved, safer DNA delivery methods to reduce the risk of complications: some patients in earlier trials developed leukemia when treated with previous "vectors" - engineered viruses that transfer copies of normally functioning genes into cells of ill patients. So far, the improved vectors have been associated with fewer complications. One example of the use of gene therapy is in immune deficiencies. Because they have inherited a defective gene, children born with these immune disorders generally lack a vital protein needed for their bodies to develop a robust assortment of immune defender cells to protect against infections. To correct these often-fatal conditions, scientists modify viruses - rendering them incapable of harmful infection - to ferry normal copies of the gene into blood stem cells removed from the patient; those cells are then re-infused and, if successful, the gene directs the body to make the missing protein.Another new trial is testing a gene therapy technique aimed at reducing graft-versus-host disease in patients who have received a stem cell transplant from a donor who is only a 50 percent genetic match. These donors are usually a parent, making extended searches for donor unnecessary. A common complication of transplants, graft-versus-host disease is a mistaken attack on the recipient's normal body tissues by the transplanted immune cells.The sixth clinical trial involves an exciting new immunotherapy for cancer, known as CAR T-cells, in patients with relapsed acute lymphoblastic leukemia (ALL), the most common type of pediatric cancer. Gene therapy plays an important role in this experimental treatment. . . . Full text in. . t.me/biotechgene 🌐Telegram channel .. #biotech_gene#genetherapy #cancer#children#gene https://www.instagram.com/p/CKgxtMXB1nd/?igshid=1pox9p0cqiivo