Ontology highlight
ABSTRACT:
SUBMITTER: Kuespert S
PROVIDER: S-EPMC7139664 | biostudies-literature | 2020 Mar
REPOSITORIES: biostudies-literature
Kuespert Sabrina S Heydn Rosmarie R Peters Sebastian S Wirkert Eva E Meyer Anne-Louise AL Siebörger Mareile M Johannesen Siw S Aigner Ludwig L Bogdahn Ulrich U Bruun Tim-Henrik TH
International journal of molecular sciences 20200312 6
Antisense Oligonucleotides (ASOs) are an emerging drug class in gene modification. In our study we developed a safe, stable, and effective ASO drug candidate in locked nucleic acid (LNA)-gapmer design, targeting TGFβ receptor II (TGFBR2) mRNA. Discovery was performed as a process using state-of-the-art library development and screening. We intended to identify a drug candidate optimized for clinical development, therefore human specificity and gymnotic delivery were favored by design. A staggere ...[more]