Experimental Model Systems Used in the Preclinical Development of Nucleic Acid Therapeutics.

ABSTRACT: Preclinical evaluation of nucleic acid therapeutics (NATs) in relevant experimental model systems is essential for NAT drug development. As part of COST Action "DARTER" (Delivery of Antisense RNA ThERapeutics), a network of researchers in the field of RNA therapeutics, we have conducted a survey on the experimental model systems routinely used by our members in preclinical NAT development. The questionnaire focused on both cellular and animal models. Our survey results suggest that skin fibroblast cultures derived from patients is the most commonly used cellular model, while induced pluripotent stem cell-derived models are also highly reported, highlighting the increasing potential of this technology. Splice-switching antisense oligonucleotide is the most frequently investigated RNA molecu

SUBMITTER: Zhou H 

PROVIDER: S-EPMC10457615 | biostudies-literature | 2023 Aug

REPOSITORIES: biostudies-literature