Genes pharmacologic approaches

Although pharmacology still relies heavily on small chemical substances, biologicals like recombinant proteins, cell-based therapies, and gene-therapy approaches have great potential for the treatment of broad spectrum of diseases. Therefore, the focus of modem pharmacology should shift towards these innovative therapies. 

A scientific procedure that turns the classical pharmacology approach upside down. Instead of finding the elusive receptor for a known hormone or transmitter what classical pharmacology aims at, reverse pharmacology is initiated through the discovery of the receptor gene and aspires to the identification of the receptors unknown ligand. 

Figure 4. Therapeutic strategies to counteract CBP loss of function. CBP loss of function leads to a decrease in histone acetylation levels as well as a decrease in CBP-dependent transcription. Two main approaches can be tested to reverse diis process either resetting HAT functionality or resetting global acetylation levels widi die use of HDAC inhibitors. Whereas both strategies would increase histone acetylation levels, HDAC inhibition would act on a broad range of genes, while CBP activation (overexpression or by a pharmacological approach) would specifically target bodi CBP-dependent histone acetylation and transcription. The structure of some of the HDACi that have been tested in different models, such as small fatty acids and hydroxamic acids, are represented in the boxes...

Newer pharmacologic approaches targeting receptors and genes... 

Successful application of ocular gene therapy has been demonstrated in animal diseases or animal models of human disease. The most encouraging results in the field of ocular gene therapy involve treatment of genetically inherited retinal degenerations occurring in various animal species. Conventional pharmacologic approaches... 

Experiments using both knock-out and pharmacological approaches have demonstrated that serotonin plays a role in modulating synaptogenesis in rodents. Monoamine oxidase (MAO) is an enzyme which degrades neurotransmitters such as noradrenaline, dopamine, and serotonin. There are two distinct forms of the enzyme, A and B, which are encoded by two separate genes, both with 15 exons and lying adjacent to each other on human Xpl 1.23-11.4 [77]. Both MAO A and B deaminate dopamine but have some different characteristics with respect to time of expression and other enzymatic activities MAOA preferentially deaminates serotonin and noradrenaline and its maximal activity appears in the fetal brain. MAOB mainly deaminates neurotransmitters... 

Duncan R. Reduction of anticancer drug toxicity. In Zeller WJ, Eisenbrand G, Heilman K, eds. Pharmacologic, Biologic, Immunologic and Gene Therapeutic Approaches. Contrib Oncol 1995 48 170-180. 

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