دورية أكاديمية
أعرض في EDS

High-throughput evaluation of polymeric nanoparticles for tissue-targeted gene expression using barcoded plasmid DNA.

التفاصيل البيبلوغرافية
العنوان: High-throughput evaluation of polymeric nanoparticles for tissue-targeted gene expression using barcoded plasmid DNA.
المؤلفون: Kim J; Department of Biomedical Engineering and the Translational Tissue Engineering Center, Johns Hopkins University School of Medicine, Baltimore, MD, USA; Institute for Nanobiotechnology, Johns Hopkins University, Baltimore, MD, USA., Vaughan HJ; Department of Biomedical Engineering and the Translational Tissue Engineering Center, Johns Hopkins University School of Medicine, Baltimore, MD, USA; Institute for Nanobiotechnology, Johns Hopkins University, Baltimore, MD, USA., Zamboni CG; Department of Biomedical Engineering and the Translational Tissue Engineering Center, Johns Hopkins University School of Medicine, Baltimore, MD, USA; Institute for Nanobiotechnology, Johns Hopkins University, Baltimore, MD, USA., Sunshine JC; Department of Biomedical Engineering and the Translational Tissue Engineering Center, Johns Hopkins University School of Medicine, Baltimore, MD, USA; Institute for Nanobiotechnology, Johns Hopkins University, Baltimore, MD, USA; Department of Pathology, Johns Hopkins University, Baltimore, MD, USA., Green JJ; Department of Biomedical Engineering and the Translational Tissue Engineering Center, Johns Hopkins University School of Medicine, Baltimore, MD, USA; Institute for Nanobiotechnology, Johns Hopkins University, Baltimore, MD, USA; Department of Materials Science and Engineering, Johns Hopkins University, Baltimore, MD, USA; Department of Chemical and Biomolecular Engineering, Johns Hopkins University, Baltimore, MD, USA; Departments of Neurosurgery, Oncology, and Ophthalmology, Johns Hopkins University School of Medicine, Baltimore, MD, USA. Electronic address: green@jhu.edu.
المصدر: Journal of controlled release : official journal of the Controlled Release Society [J Control Release] 2021 Sep 10; Vol. 337, pp. 105-116. Date of Electronic Publication: 2021 Jul 20.
نوع المنشور: Journal Article; Research Support, N.I.H., Extramural; Research Support, Non-U.S. Gov't
اللغة: English
بيانات الدورية: Publisher: Elsevier Science Publishers Country of Publication: Netherlands NLM ID: 8607908 Publication Model: Print-Electronic Cited Medium: Internet ISSN: 1873-4995 (Electronic) Linking ISSN: 01683659 NLM ISO Abbreviation: J Control Release Subsets: MEDLINE
أسماء مطبوعة: Original Publication: Amsterdam : Elsevier Science Publishers, 1984-
مواضيع طبية MeSH: Nanoparticles*, Animals ; DNA/genetics ; Gene Expression ; Mice ; Plasmids/genetics ; Tissue Distribution ; Transfection
مستخلص: Successful systemic gene delivery requires specific tissue targeting as well as efficient intracellular transfection. Increasingly, research laboratories are fabricating libraries of novel nanoparticles, engineering both new biomaterial structures and composition ratios of multicomponent systems. Yet, methods for screening gene delivery vehicles directly in vivo are often low-throughout, limiting the number of candidate nanoparticles that can be investigated. Here, we report a comprehensive, high-throughput method to evaluate a library of polymeric nanoparticles in vivo for tissue-specific gene delivery. The method involves pairing each nanoparticle formulation with a plasmid DNA (pDNA) that harbors a unique nucleotide sequence serving as the identifying "barcode". Using real time quantitative PCR (qPCR) for detection of the barcoded pDNA and quantitative reverse transcription PCR (RT-qPCR) for transcribed barcoded mRNA, we can quantify accumulation and transfection in tissues of interest. The barcode pDNA and primers were designed with sufficient sensitivity and specificity to evaluate multiple nanoparticle formulations per mouse, improving screening efficiency. Using this platform, we evaluated the biodistribution and transfection of 8 intravenously administered poly(beta-amino ester; PBAE) nanoparticle formulations, each with a PBAE polymer of differential structure. Significant levels of nanoparticle accumulation and gene transfection were observed mainly in organs involved in clearance, including spleen, liver, and kidneys. Interestingly, higher levels of transfection of select organs did not necessarily correlate with higher levels of tissue accumulation, highlighting the importance of directly measuring in vivo transfection efficiency as the key barcoded parameter in gene delivery vector optimization. To validate this method, nanoparticle formulations were used individually for luciferase pDNA delivery in vivo. The distribution of luciferase expression in tissues matched the transfection analysis by the barcode qPCR method, confirming that this platform can be used to accurately evaluate systemic gene delivery.
(Copyright © 2021. Published by Elsevier B.V.)
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معلومات مُعتمدة: T32 CA130840 United States CA NCI NIH HHS; T32 CA153952 United States CA NCI NIH HHS; R01 EY031097 United States EY NEI NIH HHS; R01 CA228133 United States CA NCI NIH HHS; P41 EB028239 United States EB NIBIB NIH HHS
فهرسة مساهمة: Keywords: Biodistribution; Gene delivery; High-throughput screening; Polymeric nanoparticle; Transfection
المشرفين على المادة: 9007-49-2 (DNA)
تواريخ الأحداث: Date Created: 20210607 Date Completed: 20211028 Latest Revision: 20220911
رمز التحديث: 20221213
مُعرف محوري في PubMed: PMC8558975
DOI: 10.1016/j.jconrel.2021.05.047
PMID: 34097924
قاعدة البيانات: MEDLINE
الوصف
تدمد:1873-4995
DOI:10.1016/j.jconrel.2021.05.047