COMPOSITIONS AND METHODS FOR GENE THERAPY TO TREAT DISEASE

摘要

Method of gene therapy for the treatment of a variety of genetic defects or diseases comprising the steps of either introducing transformed cells into an organism such as a human or the in vivo transformation of cells. The transformed cells contain a nucleic acid vector which will produce the protein necessary to correct the deficiency or disease. Examples of diseases which can be treated this way include diabetes mellitus, Parkinson's disease, cardiovascular disease, hypercholesterolemia, hypertension, anemia, thrombosis, growth disorders, metabolic disorders such as PKU and serum protein disorders, such as hemophilia A.