The very first adgrv1-zebrafish model has been presented!



Recently, an article was published in the journal Cells about the scientific research on USH2C. Merel Stemerdink and her colleagues at the Radboudumc have developed a zebrafish model with a gene defect in adgrv1 (the gene associated with USH2C). This is the very first animal model for ADGRV1-associated retinal dysfunction, and therefore a crucial step to enable further research on therapy development for ADGRV1-related RP.

Merel Stemerdink (right) has developed the mini gene products and shows them to Ivonne Bressers (Stichting Ushersyndroom). Ivonne (left) looks at the PCR strip in her hand.

Using these fish as an animal model, Merel will also start testing the effectiveness of the ADGRV1-minigene therapy in the coming year.

The official title of the paper is ‘Generation and Characterization of a Zebrafish Model for ADGRV1-Associated Retinal Dysfunction Using CRISPR/Cas9 Genome Editing Technology’ and it has been published in a Special Issue of Cells titled ‘CRISPR-Based Genome Editing in Translational Research’.

 

Read also:
How are things going with the ‘minigenes USH2c’ study?

Development of gene therapy for large USH2C gene