Cell type-selective targeted delivery of a recombinant lysosomal enzyme for enzyme therapies
Andrew D. Baik, Philip Calafati, Xiaoli Zhang, Nina Aaron, Antonia Mehra, Sven Møller-Tank, Lawrence Miloscio, Maria Praggastis, Nicholas Giovannone, Cheryl Pan, Yajun Tang, Susannah Bridges, Alejo Mujica, Peter Barbounis, Jean Yanolatos, Nicholas W. Gale, Ning Li, Christos A. Kyratsous, Christopher J. Schoenherr, Andrew Murphy, Aris N. Economides, Katherine D. Cygnar
Topics & Concepts
Enzyme replacement therapyLysosomeEnzymeLysosomal storage diseaseRecombinant DNABiologyMannose 6-phosphate receptorPhenotypeCellTransmembrane proteinAntibodyGenetic enhancementBiochemistryDiseaseGeneImmunologyReceptorMedicinePathologyLysosomal Storage Disorders ResearchCellular transport and secretionCalcium signaling and nucleotide metabolism