Proteomics: State of the Art and Its Relevance for Gene Therapy

Abstract

The molecular mechanisms underlying most diseases, including those of the cardiovascular system, are widely unknown. Basically, pathological changes in the organism arise from protein alterations. Proteomics comprises a set of tools allowing the identification of protein alterations, i.e. changes of protein abundance and posttranslational modifications, associated with diseases. The linkage of information about such protein changes with functional alterations as revealed by physiological studies constitutes functional proteomics that enables the disclosure of disease mechanisms. Disease-linked protein alterations include those of suitable candidates for drug targets and disease biomarkers as well as therapeutic proteins/peptides. Since gene therapy depends on the function of a therapeutic protein encoded by a “therapeutic” gene, proteomic analyses provide the basis for the design and application of gene therapies. The storage and administration of experimental data obtained by the application of proteomic analyses is supported by speciesand tissue-specific protein databases and specific software. Publications in this field are reviewed in this chapter.