New Approaches to Pulmonary Hypertension
Will Therapies in Mice Work in Humans?
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Gene manipulation in mice is now a routine approach to the investigation of disease pathophysiology. Examination of cardiovascular physiology in mice have required the development of miniaturized hemodynamic monitoring systems, or essentially mouse “cath labs,” in which catheters and guidewires are manipulated in mouse vessels often <1 mm in size. Cardiovascular integrative physiology has become miniaturized in scale but magnified in impact. Studies testing the pharmacological or physiological effects of infused drugs, which once could be done only in large animals, are now performed in mice by genetic manipulation and/or gene transfer, with equally rigorous hemodynamic monitoring. The potential for defining disease pathophysiology and developing new therapies is enormous.
Given these advances in technology, what have we learned about cardiovascular biology from mouse models? And importantly, will we be able to translate these discoveries into novel therapies for humans? The study by Champion et al1 in this issue of Circulation brings these questions under close scrutiny. These investigators examine the function of prepro-calcitonin gene–related …