Clearer Day For Gene Therapy: New Vector Carries Big Genes Linked To Inherited Blindness
For some inherited diseases, one barrier to successful gene therapy is that a commonly used vector (package into which the curative gene is placed) cannot accommodate the large size of the curative gene. However, a newly developed vector derived from the AAV5 form of adeno-associated virus has been used to successfully carry large genes into cells and to improve eye function in a mouse model of an inherited disease causing progressive loss of sight.