Endosomal escape cell-penetrating peptides significantly enhance pharmacological effectiveness and CNS activity of systemically administered antisense oligonucleotides
Mohadeseh Dastpeyman, Ramin Sharifi, Azin Amin, John A. Karas, Brittany Cuic, Yijun Pan, Joseph A. Nicolazzo, Bradley J. Turner, Fazel Shabanpoor
Topics & Concepts
EndosomeBlood–brain barrierSpinal muscular atrophyTransgeneCell-penetrating peptideCell biologyCentral nervous systemSpinal cordPharmacologyCellIntracellularMedicineChemistryBiologyNeuroscienceGeneBiochemistryRNA Interference and Gene DeliveryNeurogenetic and Muscular Disorders ResearchAdvanced biosensing and bioanalysis techniques