Although these pre-clinical data are promising, there are likely to be significant challenges in translating BMPR2 gene therapy to humans with our current limitations in vector delivery to the pulmonary endothelium, immunogenicity and uncertainty regarding whether increasing BMPR-II expression results in sustainable long-term improvements in PAH.
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Precision medicine and personalising therapy in pulmonary hypertension: seeing the light from the dawn of a new era.
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