Validation therapeutic target

Inhibitors of the Angiopoietin/Tie-2 and Ephrin/Eph systems are in preclinical development which parallels the biological target validation of these molecules. As vascular assembly, maturation, and homeostasis regulating molecules, therapeutic interference with these molecular systems may hold promise for a number of vascular indications. 

Target validation, to determine that a gene product is causative of disease symptoms or that activation of the target protein ameliorates disease symptoms. Agonist/activator or an inhibitor which may be therapeutic could be identified using microarrays... 

Nature has created a diverse array of plant and animal toxins that act at mammalian muscle and ganglionic nAChRs or invertebrate nAChRs because the critical physiological functions of these receptors make them prime targets for defensive or predatory strategies. More recently, the perceived validity of neuronal nAChR as therapeutic targets has prompted the generation of new synthetic ligands. Examples are listed in Table 1. 

An important validation of the mice that overexpress human mutant 3APP as a platform for testing therapeutics targeting 3-peptide deposition has been provided by the Elan company, using their PDAPP mouse [146]. Immunization of the mice, either at an early age or after plaques had formed, resulted in clearance of immunoreactive plaques and peptide from the subjects brains. Although the elucidation of the mechanism explaining... 

Semicarbazide Sensitive Amine Oxidase and Vascular Adhesion Protein-1 One Protein Being Validated as a Therapeutic Target for Inflammatory Diseases... 

The pre-clinical drug discovery process is typically a sequential selection and optimization process focusing, as summarized in Table 2, on different essential properties at each step. " Tool compounds required for early in vitro or in vivo target validation typically do not need to satisfy the same stringent in vivo efficacy and safety criteria as clinical candidates and drugs, which again depend on the targeted therapeutic indication. ... 

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