GENE THERAPY FOR HAEMOPHILIA

摘要

The invention provides a recombinant nucleic acid construct comprising: (i) a mammalian myosin heavy chain promoter; (ii) nucleic acid encoding a signal sequence; and (iii) nucleic acid encoding a functional blood clotting protein; the elements (i)-(iii) being operably linked to provide for expression of the functional protein. The construct is useful for the treatment of diseases such as haemophilia by introducing it into muscle cells. The activity of the promoter can be enhanced by exercise or stimulation of the muscle cells.