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Mutation-specific potency and efficacy of cftr chloride channel potentiators
Journal of Pharmacology and Experimental Therapeutics, 06/04/09
Caputo A et al. - Results indicate that CFTR potentiators correct the basic defect caused by CF mutations residing in different CFTR domains. However, there are differences among potentiators, with felodipine and PG-01 having a wider pharmacological activity whereas SF-01 is more mutation specific. The authors observations are useful in the prioritization and development of drugs targeting the CF basic defect.
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Targeting of a mutant plasminogen activator to circulating red blood cells for prophylactic fibrinolysis
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