Ontology highlight
ABSTRACT:
SUBMITTER: Osborn MF
PROVIDER: S-EPMC4605330 | biostudies-literature | 2015 Oct
REPOSITORIES: biostudies-literature
Osborn Maire F MF Alterman Julia F JF Nikan Mehran M Cao Hong H Didiot Marie C MC Hassler Matthew R MR Coles Andrew H AH Khvorova Anastasia A
Nucleic acids research 20150922 18
One of the major obstacles to the pharmaceutical success of oligonucleotide therapeutics (ONTs) is efficient delivery from the point of injection to the intracellular setting where functional gene silencing occurs. In particular, a significant fraction of internalized ONTs are nonproductively sequestered in endo-lysosomal compartments. Here, we describe a two-step, robust assay for high-throughput de novo detection of small bioactive molecules that enhance cellular uptake, endosomal escape, and ...[more]