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A novel nonviral gene delivery system for treating Leber's congenital amaurosis

2017; Future Science Ltd; Volume: 8; Issue: 10 Linguagem: Inglês

10.4155/tde-2017-0072

2041-6008

Da Sun, Rebecca M. Schur, Zheng‐Rong Lu,

Ocular Disorders and Treatments

Therapeutic DeliveryVol. 8, No. 10 CommentaryA novel nonviral gene delivery system for treating Leber's congenital amaurosisDa Sun, Rebecca M Schur & Zheng-Rong LuDa Sun Case Center for Biomolecular Engineering, Department of Biomedical Engineering, School of Engineering, Case Western Reserve University, Cleveland, OH 44106, USA, Rebecca M Schur Case Center for Biomolecular Engineering, Department of Biomedical Engineering, School of Engineering, Case Western Reserve University, Cleveland, OH 44106, USA & Zheng-Rong Lu*Author for correspondence: Tel.: +1 216 368 0187; E-mail Address: zxl125@case.edu Case Center for Biomolecular Engineering, Department of Biomedical Engineering, School of Engineering, Case Western Reserve University, Cleveland, OH 44106, USA M. Frank Rudy & Margaret Domiter Rudy Professor of Biomedical Engineering, Department of Biomedical Engineering, Case Western Reserve University, Wickenden 427, Mail Stop 7207, 10900 Euclid Avenue, Cleveland, OH 44106, USAPublished Online:25 Sep 2017https://doi.org/10.4155/tde-2017-0072AboutSectionsView ArticleView Full TextPDF/EPUB ToolsAdd to favoritesDownload CitationsTrack CitationsPermissionsReprints ShareShare onFacebookTwitterLinkedInReddit View articleKeywords: ECOgene therapyLCAnanoparticlenonviralretinaRPE65References1 Stone EM. Leber congenital amaurosis – a model for efficient genetic testing of heterogeneous disorders: LXIV Edward Jackson memorial lecture. Am. J. Ophthalmol. 144(6), 791–811.e796 (2007).Crossref, Medline, CAS, Google Scholar2 Kiser PD, Zhang J, Badiee M et al. Catalytic mechanism of a retinoid isomerase essential for vertebrate vision. Nat. Chem. 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Gene Ther. 18(7), 637–645 (2011).Crossref, Medline, CAS, Google ScholarFiguresReferencesRelatedDetailsCited ByDirecting the Way—Receptor and Chemical Targeting Strategies for Nucleic Acid Delivery15 September 2022 | Pharmaceutical Research, Vol. 40, No. 1Structure and Function of Cationic and Ionizable Lipids for Nucleic Acid Delivery4 January 2023 | Pharmaceutical Research, Vol. 40, No. 1Formulation and efficacy of ECO/pRHO-ABCA4-SV40 nanoparticles for nonviral gene therapy of Stargardt disease in a mouse modelJournal of Controlled Release, Vol. 330Dramatic influence of the hydroxy functionality of azasugar moiety in the head group region of tocopherol-based cationic lipids on in vitro gene transfection efficacies1 January 2021 | New Journal of Chemistry, Vol. 45, No. 2Non-viral Gene Therapy for Stargardt Disease with ECO/pRHO-ABCA4 Self-Assembled NanoparticlesMolecular Therapy, Vol. 28, No. 1Synthesis and Evaluation of pH-Sensitive Multifunctional Lipids for Efficient Delivery of CRISPR/Cas9 in Gene Editing24 December 2018 | Bioconjugate Chemistry, Vol. 30, No. 3 Vol. 8, No. 10 Follow us on social media for the latest updates Metrics Downloaded 73 times History Published online 25 September 2017 Published in print October 2017 Information© 2017 Future Science LtdKeywordsECOgene therapyLCAnanoparticlenonviralretinaRPE65Financial & competing interests disclosureThe authors have no relevant affiliations or financial involvement with any organization or entity with a financial interest in or financial conflict with the subject matter or materials discussed in the manuscript. This includes employment, consultancies, honoraria, stock ownership or options, expert testimony, grants or patents received or pending, or royalties.No writing assistance was utilized in the production of this manuscript.PDF download