A highly efficient procedure for transferring functional genes into mammalian cells has been developed using retroviral vectors as a delivery system. Retroviral vectors containing the human gene for the enzyme adenosine deaminase (ADA) as well as the NeoR gene have been made. Using the knowledge obtained from murine systems a non- human primate autologous bone marrow transplantation/gene transfer protocol has been developed. Low levels of both the human ADA gene and the NeoR gene have been expressed in the peripheral blood cells of several monkeys. In addition, these vectors have been used to introduce exogenous genes into human tumor infiltrating lymphocytes (TIL). These studies are preliminary to attempting human gene therapy in patients suffering from ADA severe combined immunodeficiency disease and advanced cancer.
