Gene therapy for acute diseases

The use of gene transfer systems to study cell function makes it apparent t hat overexpression of a transgene can restore or improve the function of a protein and positively influence cell function in a predetermined manner fo r purposes of counterbalancing cellular pathophysiology. The ability of som e gene transfer vehicles to produce transgene product within hours of deliv ery positions gene transfer as a unique pharmaceutical administration syste m that can quickly affect production of biologic response modifiers in a hi ghly compartmentalized fashion. This approach can be expected to overcome m any of the adverse effects and high costs of systemic delivery of recombina nt pharmaceuticals. This review highlights recent advances toward developme nt of gene therapies for acute illnesses with particular emphasis on precli nical models of disease. In this context, a growing body of data suggests t hat gene therapies for polygenic and non-genetic diseases such as asthma, c ardiogenic and noncardiogenic pulmonary edema, stroke, subarachnoid hemorrh age, seizures, acute myocardial infarction, endovascular thrombosis, and in fections may someday be options for the treatment of patients.