| abstract |
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 transforma-tion of cells. The transformed cells contain a nucleic acid vector which will produce the protein neces-sary to correct the deficiency or disease. Examples of diseases which can be treated this way include diabetes mellitus, Parkinson's disease, cardiovascu-lar disease, hypercholesterolemia, hypertension, ane-mia, thrombosis, growth disorders, metabolic disor-ders such as PKU and serum protein disorders, such as hemophilia A. |