Development of an enhanced lentiviral vector for gene therapy of ADA-SCID

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Adenosine deaminase deficiency is a condition in which affected children lack a functional immune system and die from infection in the first year of life. The nature of the gene defect is such that severe defects are also found in other systems of the body. Due to the limitations of other treatment options, gene therapy has recently been used with some success in correcting the immune defects. In this study we hope to build on this and test better gene therapy delivery vectors that may correct both the immune system and non-immune system problems. We will also use vectors that have improved safety profiles compared to presently used constructs. We will test our new vectors in cellular systems and also aim to correct a murine model of the disease.