Splice modulation as potential therapy for Usher syndrome type IIa

Contains fulltext : 199057.pdf (publisher's version ) (Open Access)Radboud University, 17 januari 2019Promotor : Kremer, J.M.J. Co-promotores : Wijk, H.A.R. van, Pennings, R.J.E

Generation of Humanized Zebrafish Models for the In Vivo Assessment of Antisense Oligonucleotide-Based Splice Modulation Therapies

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 for this purpose is the reported poor recognition of human splice sites by the splicing machineries of other species. To overcome this problem, we provide a detailed guideline for the generation of functional humanized knock-in zebrafish models to assess the effect of mutation-induced aberrant splicing and subsequent AON-based splice modulation therapy

Eyes shut homolog is important for the maintenance of photoreceptor morphology and visual function in zebrafish

Contains fulltext : 194442.pdf (publisher's version ) (Open Access

NINL and DZANK1 Co-function in Vesicle Transport and Are Essential for Photoreceptor Development in Zebrafish

Contains fulltext : 151728.pdf (publisher's version ) (Open Access

Dissolution of weddellite, calcium oxalate diydrate in Pyxine subcinerea

Contains fulltext : 151728.pdf (publisher's version ) (Open Access