DNA construct comprising a muscle specific regulatory element for immunization or gene therapy

摘要

The present invention is directed to a DNA construct which is useful for immunization or gene therapy. The construct of the invention comprises muscle specific regulatory elements, such as a promoter or a promoter and one or more enhancer elements, and a DNA sequence under control of the muscle specific regulatory elements. Several DNA sequences may be incorporated into the DNA construct. In one embodiment, the DNA sequence codes for an antigen, antigenic determinant or an epitope of an antigen. In a second embodiment, the DNA sequence is a normal muscle gene which is effected in a muscle disease. In a third embodiment, the DNA sequence is an antisense for blocking an abnormal muscle gene. In a fourth embodiment, the DNA sequence codes for a protein which circulates in the mammalian blood or lymphatic systems. The present invention is useful for ameliorating the effects of diseases of muscle by expression of the normal gene or blocking abnormal gene expression within muscle cells, for the heterologous expression of a transgene which codes for a circulating protein or a protein which modifies a disease state in which muscle is not primarily involved and for vaccine development.