Category Archives: Gene Therapy

Meet Mark Kay Mark A. Kay, MD, PhD, is the Director of the Program in Human Gene Therapy, and Professor in the Department of Pediatrics and Genetics at Stanford University School of Medicine. Dr. Kay is... By: stanfordonline … Continue reading →

Gene Therapy - AAV Virus Explore Research at the University of Florida: A common virus known as AAV, or Adeno-Associated Virus, has been described as boring because while it affect... By: FloridaMuseum … Continue reading →

Gene therapy attempts to treat genetic diseases at the molecular level by correcting what is wrong with defective genes. Clinical research into gene therapys safety and effectiveness has just begun. No one knows if gene therapy will work, or for what diseases. If gene therapy is successful, it could work by preventing a protein from doing something that causes harm, restoring the normal function of a protein, giving proteins new functions, or enhancing the existing functions of proteins. How Do You Do It? Gene therapy relies on finding a dependable delivery system to carry the correct gene to the affected cells. The gene must be delivered inside the target cells and work properly without causing adverse effects. Delivering genes that will work correctly for the long term is the greatest challenge of gene therapy. Human ex vivo Gene Therapy 1986 1989 1993 Present See more here: Gene Therapy - A Revolution in Progress: Human Genetics ... … Continue reading →

PHILADELPHIA A new form ofgene therapyfor boys with X-linked severe combined immunodeficiency syndrome (SCID-X1), a life-threatening condition also known as bubble boy disease, appears to be both effective and safe, according to an international clinical trial with sites inBoston, Cincinnati, Los Angeles, London, and Paris. Early data published in theNew England Journal of Medicinesuggests that the therapy may avoid the late-developing leukemiaseen in a quarter of SCID-X1 patients in previous gene-therapy trials in Europe that took place more than a decade ago. Left untreated, boys with SCID-X1 usually die of infection before their first birthday. The lab of coauthorFrederic Bushman, PhD, professor of Microbiology, from thePerelman School of Medicine at the University of Pennsylvania, carried out the deep DNA sequencing on patient specimens to track and verify distributions of integration sites of the vector.The vector used in the new trial was engineered to remove molecular signals implicated in cancers in the first trial. Eight of nine boys recruited to date to the present trial are alive between 12 and 38 months after treatment, with no SCID-X1-associated infections. The gene therapy alone generated functioning immune systems in seven of eight boys. Genetic studies showed that the new viral vector did not … Continue reading →

Gene therapy offers a safe and effective way to treat bubble boy disease Health Updates Gene therapy offers a safe and effective way to treat bubble boy disease.'Health Updates' connects health-conscious individuals with important news and info... By: Health Updates … Continue reading →

Dr. Indrin Chetty at TEDx Detroit 2014 Dr. Indrin Chetty at TEDx Detroit 2014 "Pioneering Edge technology, Novel gene therapy program and the futuristic Raman spectroscopy research at Henry Ford Hospital. By: HFiiDetroit2012 … Continue reading →

Amy Norton HealthDay Reporter Posted: Thursday, October 9, 2014, 5:00 AM (HealthDay News) -- A new form of gene therapy may offer a safe and effective way to treat "bubble boy" disease -- a severe immune deficiency that is fatal unless treated in infancy. Researchers have long known that gene therapy can cure the disease, known medically as severe combined immunodeficiency, or SCID. Over a decade ago, trials in Europe showed that gene therapy worked -- but five of the 20 children treated developed leukemia (a type of cancer) within two to five years, according to background information in the study. In the new trial, reported in the Oct. 9 New England Journal of Medicine, researchers refined the gene therapy approach to hopefully negate the leukemia risk. Eight of nine children who received the therapy are still alive one to three years later, the investigators report. And so far, none has developed leukemia. It's too early to say the therapy carries no leukemia risk, cautioned researcher Dr. David Williams, a pediatric hematologist/oncologist at Dana-Farber Cancer Institute and Boston Children's Hospital. "We'll continue to follow these children for 15 years," Williams said. But based on the early results, he noted, the … Continue reading →

Date: October 8, 2014 Source: NIH/National Institute of Allergy and Infectious Diseases Summary: Gene therapy using a modified delivery system, or vector, can restore the immune systems of children with X-linked severe combined immunodeficiency (SCID-X1), a rare, life-threatening inherited condition that primarily affects boys, researchers have discovered. Researchers have found that gene therapy using a modified delivery system, or vector, can restore the immune systems of children with X-linked severe combined immunodeficiency (SCID-X1), a rare, life-threatening inherited condition that primarily affects boys. Previous efforts to treat SCID-X1 with gene therapy were initially successful, but approximately one-quarter of the children developed leukemia two to five years after treatment. Results from a study partially funded by the National Institute of Allergy and Infectious Diseases (NIAID), a component of the National Institutes of Health (NIH), suggest that the new vector is equally effective at restoring immunity and may be safer than previous approaches. In SCID-X1, mutations in a specific gene prevent the development of infection-fighting T cells. The standard therapy for SCID is transplantation of blood-forming stem cells, but some patients lack a suitable donor. In gene therapy, doctors remove stem cells from the patient's bone marrow, use a vector to insert a … Continue reading →

Gene therapy (5 of 5) Created using PowToon -- Free sign up at http://www.powtoon.com/ . Make your own animated videos and animated presentations for free. PowToon is a free tool that allows you to develop... By: Beau Johanan dela Cruz … Continue reading →

Gene therapy (3 of 5) Created using PowToon -- Free sign up at http://www.powtoon.com/ . Make your own animated videos and animated presentations for free. PowToon is a free to... By: Beau Johanan dela Cruz … Continue reading →