Gene therapy using bone marrow transplants transfected with therapeutic genes under the control of tissue-specific promoters

摘要

Methods for expressing exogenous genes in differentiated cells of a specific type, such as skeletal tissue cells are disclosed. According to the method, pluripotent stem cells capable of maturing into differentiated cells of a specific type are contacted with a nucleic acid comprising an exogenous gene operatively linked to a regulatory element capable of controlling expression of the gene in the differentiated cell. As a result of the method, a population of transduced stem cells capable of maturing into differentiated cells expressing the exogenous gene is produced.