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mRNA trans-splicing dual AAV vectors for (epi)genome editing and gene therapy.
Riedmayr LM, Hinrichsmeyer KS, Thalhammer SB, Mittas DM, Karguth N, Otify DY, Böhm S, Weber VJ, Bartoschek MD, Splith V, Brümmer M, Ferreira R, Boon N, Wögenstein GM, Grimm C, Wijnholds J, Mehlfeld V, Michalakis S, Fenske S, Biel M, Becirovic E. Riedmayr LM, et al. Among authors: boon n. Nat Commun. 2023 Oct 18;14(1):6578. doi: 10.1038/s41467-023-42386-0. Nat Commun. 2023. PMID: 37852949 Free PMC article.
Crumbs2 Is an Essential Slit Diaphragm Protein of the Renal Filtration Barrier.
Möller-Kerutt A, Rodriguez-Gatica JE, Wacker K, Bhatia R, Siebrasse JP, Boon N, Van Marck V, Boor P, Kubitscheck U, Wijnholds J, Pavenstädt H, Weide T. Möller-Kerutt A, et al. Among authors: boon n. J Am Soc Nephrol. 2021 May 3;32(5):1053-1070. doi: 10.1681/ASN.2020040501. Epub 2021 Mar 9. J Am Soc Nephrol. 2021. PMID: 33687977 Free PMC article.
AAV-mediated gene augmentation therapy of CRB1 patient-derived retinal organoids restores the histological and transcriptional retinal phenotype.
Boon N, Lu X, Andriessen CA, Moustakas I, Buck TM, Freund C, Arendzen CH, Böhringer S, Boon CJF, Mei H, Wijnholds J. Boon N, et al. Among authors: boon cjf. Stem Cell Reports. 2023 Jun 13;18(6):1388. doi: 10.1016/j.stemcr.2023.05.008. Stem Cell Reports. 2023. PMID: 37315526 Free PMC article. No abstract available.
CRB1 is required for recycling by RAB11A+ vesicles in human retinal organoids.
Buck TM, Quinn PMJ, Pellissier LP, Mulder AA, Jongejan A, Lu X, Boon N, Koot D, Almushattat H, Arendzen CH, Vos RM, Bradley EJ, Freund C, Mikkers HMM, Boon CJF, Moerland PD, Baas F, Koster AJ, Neefjes J, Berlin I, Jost CR, Wijnholds J. Buck TM, et al. Among authors: boon n, boon cjf. Stem Cell Reports. 2023 Sep 12;18(9):1793-1810. doi: 10.1016/j.stemcr.2023.07.001. Epub 2023 Aug 3. Stem Cell Reports. 2023. PMID: 37541258 Free PMC article.
Usherin defects lead to early-onset retinal dysfunction in zebrafish.
Dona M, Slijkerman R, Lerner K, Broekman S, Wegner J, Howat T, Peters T, Hetterschijt L, Boon N, de Vrieze E, Sorusch N, Wolfrum U, Kremer H, Neuhauss S, Zang J, Kamermans M, Westerfield M, Phillips J, van Wijk E. Dona M, et al. Among authors: boon n. Exp Eye Res. 2018 Aug;173:148-159. doi: 10.1016/j.exer.2018.05.015. Epub 2018 May 16. Exp Eye Res. 2018. PMID: 29777677 Free PMC article.
729 results