Ontology highlight
ABSTRACT:
SUBMITTER: Jyotsana N
PROVIDER: S-EPMC7116733 | biostudies-literature | 2019 Aug
REPOSITORIES: biostudies-literature
Jyotsana Nidhi N Sharma Amit A Chaturvedi Anuhar A Budida Ramachandramouli R Scherr Michaela M Kuchenbauer Florian F Lindner Robert R Noyan Fatih F Sühs Kurt-Wolfram KW Stangel Martin M Grote-Koska Denis D Brand Korbinian K Vornlocher Hans-Peter HP Eder Matthias M Thol Felicitas F Ganser Arnold A Humphries R Keith RK Ramsay Euan E Cullis Pieter P Heuser Michael M
Annals of hematology 20190518 8
Efficient and safe delivery of siRNA in vivo is the biggest roadblock to clinical translation of RNA interference (RNAi)-based therapeutics. To date, lipid nanoparticles (LNPs) have shown efficient delivery of siRNA to the liver; however, delivery to other organs, especially hematopoietic tissues still remains a challenge. We developed DLin-MC3-DMA lipid-based LNP-siRNA formulations for systemic delivery against a driver oncogene to target human chronic myeloid leukemia (CML) cells in vivo. A mi ...[more]