Article
Generation of Humanized Zebrafish Models for the In Vivo Assessment of Antisense Oligonucleotide-Based Splice Modulation Therapies.
Methods in molecular biology (Clifton, N.J.) - 1 Jan 2022
Schellens Renske, de Vrieze Erik, Slijkerman Ralph, Kremer Hannie, van Wijk Erwin
Abstract excerpt
Antisense oligonucleotide (AON)-based splice modulation is the most widely used therapeutic approach to redirect precursor messenger RNA (pre-mRNA) splicing. To study the functional effect of human mutations affecting pre-mRNA splicing for which AON-based splice redirection would be a potential therapeutic option, humanized knock-in animal models are pivotal. A major limitation of using humanized animal models...
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