Ontology highlight
ABSTRACT:
SUBMITTER: Kim Y
PROVIDER: S-EPMC6731179 | biostudies-literature | 2019 Sep
REPOSITORIES: biostudies-literature
Kim Youngsoo Y Jo Minji M Schmidt Joanna J Luo Xiaolin X Prakash Thazha P TP Zhou Tianyuan T Klein Stephanie S Xiao Xiaokun X Post Noah N Yin Zhengfeng Z MacLeod A Robert AR
Molecular therapy : the journal of the American Society of Gene Therapy 20190629 9
Antisense oligonucleotides (ASOs) are a novel therapeutic approach to target difficult-to-drug protein classes by targeting their corresponding mRNAs. Significantly enhanced ASO activity has been achieved by the targeted delivery of ASOs to selected tissues. One example is the targeted delivery of ASOs to hepatocytes, achieved with N-acetylgalactosamine (GalNAc) conjugation to ASO, which results in selective uptake by asialoglycoprotein receptor (ASGR). Here we have evaluated the potential of Ga ...[more]