Targeted Somatic Gene Transfer to Predict Drug Effects in Complex Cells and Tissues

The instant invention provides novel somatic gene transfer methods for mimicking one or more effects of a drug candidate compound. Generally stated, the methods are in vitro assays that modulate expression of a selected target molecule and analyze consequences of that modulation to evaluate effects of the drug candidate compound. Preferred methods of the invention positively identify target molecules for drug candidate compounds with capacity to potentiate or suppress activity of that molecule. Particularly preferred methods mimic human diseases or disorders impacted by the target molecule and the drug candidate compound. Significantly, the present methods are complementary to traditional drug screening approaches and can enhance those approaches by providing identified target molecules, particularly in the absence of lead compounds. Description (Set) Proposed Use (Set) Traditional approaches for developing drugs, such as ,"natural products testing" and "synthetic chemical testing" have recognized shortcomings. Natural products testing requires access to a large variety of organisms many of which may be rare or produce drug candidate compounds in limiting quantities. In most cases, there is incomplete knowledge as to the structure and function of the compounds and their molecular targets. Synthetic chemical testing requires substantial knowledge with respect to the structure and function of that lead compound as well as the manipulations necessary to develop a drug candidate compound from the lead compound. Chemical manipulations required to make a drug candidate compound can be labor intensive and require several years of research effort. This method circumvents these shortcomings and complements traditional approaches.
Patent (Set) 7,037,648;

Patents:
US 7,037,648

Inventor(s): Marban, Eduardo

Type of Offer: Licensing



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