Therapeutic approaches to improve BMPR2 expression and/or BMP signalling. (1) Exogenous delivery—endothelial-targeted replacement of dysfunctional BMPR2 through viruses, exosomes, or nanoparticles. (2) Regulation of DNA damage—activation of RAD51 DNA damage repair or inhibition of PARP1 DNA repair by the small molecule inhibitor, Olaparib. (3) Promotion of translational read-through—suppression of nonsense-mediated mutations using Ataluren/PTC124 improving BMPR2 expression. (4) Rescue of BMPR2 cysteine ECD mutations from the ER—rescue BMPR2 cell-surface expression and signalling by treatment with 4PBA.