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A protein domain-oriented approach to expand the opportunities of therapeutic exon skipping for USH2A-associated retinitis pigmentosa.
Mol Ther Nucleic Acids. 2023 May 20;32:980-994. doi: 10.1016/j.omtn.2023.05.020. eCollection 2023 Jun 13.
Mol Ther Nucleic Acids. 2023.
PMID: 37313440
Free PMC article.
Zebrafish as a Model to Evaluate a CRISPR/Cas9-Based Exon Excision Approach as a Future Treatment Option for EYS-Associated Retinitis Pigmentosa.
Schellens R, de Vrieze E, Graave P, Broekman S, Nagel-Wolfrum K, Peters T, Kremer H, Collin RWJ, van Wijk E.
Schellens R, et al. Among authors: graave p.
Int J Mol Sci. 2021 Aug 25;22(17):9154. doi: 10.3390/ijms22179154.
Int J Mol Sci. 2021.
PMID: 34502064
Free PMC article.
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