2024
Novel treatment for PXE: Recombinant ENPP1 enzyme therapy
Jacobs I, Obiri-Yeboah D, Stabach P, Braddock D, Li Q. Novel treatment for PXE: Recombinant ENPP1 enzyme therapy. Molecular Therapy 2024, 32: 3815-3820. PMID: 39342427, DOI: 10.1016/j.ymthe.2024.09.028.Peer-Reviewed Original ResearchAbcc6<sup>-/-</sup> micePseudoxanthoma elasticumAbcc6<sup>-/-</sup> mouse model of pseudoxanthoma elasticumPPi levelsMouse model of pseudoxanthoma elasticumModel of pseudoxanthoma elasticumEnzyme therapyEctopic calcificationPlasma PPi levelsMuzzle skinDose-dependent elevationHepatic efflux transportersPrevent ectopic calcificationPlasma PPiABCC6 geneATP secretionTherapeutic optionsDose-dependentlyEfflux transportersInactivating mutationsENPP1 activitySubcutaneous injectionWeeks of ageNovel treatmentCalcification disorders
2016
Novel Biologic Therapy for Untreated Diseases of Vascular Calcification
Braddock D, Kavanagh D, Li X. Novel Biologic Therapy for Untreated Diseases of Vascular Calcification. Blood 2016, 128: 2326. DOI: 10.1182/blood.v128.22.2326.2326.Peer-Reviewed Original ResearchConnective tissue diseasePlasma PPiTissue calcificationPseudoxanthoma elasticumABCC6 mutationsTissue diseaseVascular calcificationMembrane transport proteinsKnockout miceStem cell progenitorsMineralization inhibitorPotent mineralization inhibitorNovel biologic therapiesElastic fiber alterationsTransport proteinsFusion proteinVivo experimental evidenceCell progenitorsFunction mutationsBiologic therapyUntreated diseaseArterial calcificationNeonatal diseaseSpecific tissuesCommon disorder