A new variation of gene therapy raises hopes for a safe and effective long-term treatment for X-linked severe combined immunodeficiency syndrome (SCID-X1), a life-threatening heritable disorder. The research was produced by a collaborative research team from Dana-Farber/Boston Children's Cancer and Blood Disorders Center, along with other institutions participating in an international clinical trial that involved boys from the United States and France. SCID-X1, dubbed bubble boy disease after a patient who lived for 12 years in a sterile bubble, is a rare genetic disorder that hinders the ability of individuals to combat infections. Because the disease is carried in an X-chromosome recessive pattern, the disorder occurs almost only in males. The resulting mutations inactivate a gene called IL-2 receptor gamma (IL2RG), severely weakening immune system functions. Left untreated, individuals who inherit the disorder usually die within a year. Previous gene therapy trials conducted in Europe over a decade ago promised dramatic progress, until a quarter of patients developed leukemia about two to five years following treatment. Scientists found that the previously used vectorthe device for transporting the correct gene in therapyinadvertently activated oncogenes, which can cause cancer. In this new study, the vector in use is a self-inactivating gammaretrovirus, which … Continue reading →

PUBLIC RELEASE DATE: 20-Oct-2014 Contact: Todd Murphy murphyt@ohsu.edu 503-494-8231 Oregon Health & Science University @ohsunews Oregon Health & Science University's Vaccine & Gene Therapy Institute (OHSU), has been awarded a $10 million contract from the National Institute of Allergy and Infectious Diseases (NIAID), part of the National Institutes of Health (NIH). Kineta, Inc., the University of Washington Center for Innate Immunity and Immune Disease (CIIID), and the Vaccine & Gene Therapy Institute of Florida (VGTI Florida) will collaborate with OHSU as major sub-contractors to develop new vaccine adjuvants that could boost the effectiveness of a wide range of human vaccines for infectious diseases including West Nile Virus, Dengue and Japanese Encephalitis. The work will also provide novel applications for enhancing the immune response against Ebola virus, HIV, and other virus infections. Infectious diseases affect millions of people in developed and developing nations, many with no effective protective vaccines. Vaccines are the first line of defense against infectious disease and have saved millions of lives over the years. However, some people with weakened immune systems and the elderly lose the ability to respond to vaccines. The effectiveness of vaccines can be improved by the addition of substances called adjuvants that not … Continue reading →