Zhou, Haiyan;
Arechavala-Gomeza, Virginia;
Garanto, Alejandro;
(2023)
Experimental Model Systems Used in the Preclinical Development of Nucleic Acid Therapeutics.
Nucleic Acid Therapeutics
10.1089/nat.2023.0001.
(In press).
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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 molecule, followed by small interfering RNA. Animal models are less prevalent but also widely used among groups in the network, with transgenic mouse models ranking the top. Concerning the research fields represented in our survey, the mostly studied disease area is neuromuscular disorders, followed by neurometabolic diseases and cancers. Brain, skeletal muscle, heart, and liver are the top four tissues of interest reported. We expect that this snapshot of the current preclinical models will facilitate decision making and the share of resources between academics and industry worldwide to facilitate the development of NATs.
Type: | Article |
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Title: | Experimental Model Systems Used in the Preclinical Development of Nucleic Acid Therapeutics |
Location: | United States |
Open access status: | An open access version is available from UCL Discovery |
DOI: | 10.1089/nat.2023.0001 |
Publisher version: | https://doi.org/10.1089/nat.2023.0001 |
Language: | English |
Additional information: | Open access This Open Access article is distributed under the terms of the Creative Commons License [CC-BY] ( http://creativecommons.org/licenses/by/4.0), which permits unrestricted use, distribution, and reproduction in any medium, provided the original work is properly cited. |
Keywords: | Science & Technology, Life Sciences & Biomedicine, Biochemistry & Molecular Biology, Chemistry, Medicinal, Medicine, Research & Experimental, Pharmacology & Pharmacy, Research & Experimental Medicine, nucleic acid therapeutics, model systems, cellular models, animal models, antisense technology, efficacy, PLURIPOTENT STEM-CELLS, IN-VITRO, ANTISENSE OLIGONUCLEOTIDES, MESSENGER-RNA, GENERATION, DIFFERENTIATION, ORGANOIDS, MUTATION, BARRIER, BRAIN |
UCL classification: | UCL UCL > Provost and Vice Provost Offices > School of Life and Medical Sciences UCL > Provost and Vice Provost Offices > School of Life and Medical Sciences > Faculty of Population Health Sciences > UCL GOS Institute of Child Health UCL > Provost and Vice Provost Offices > School of Life and Medical Sciences > Faculty of Population Health Sciences > UCL GOS Institute of Child Health > Genetics and Genomic Medicine Dept |
URI: | https://discovery.ucl.ac.uk/id/eprint/10170170 |
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