Investigating 3R In Vivo Approaches for Bio-Distribution and Efficacy Evaluation of Nucleic Acid Nanocarriers: Studies on Peptide-Mimicking Ionizable Lipid.

التفاصيل البيبلوغرافية
العنوان:	Investigating 3R In Vivo Approaches for Bio-Distribution and Efficacy Evaluation of Nucleic Acid Nanocarriers: Studies on Peptide-Mimicking Ionizable Lipid.
المؤلفون:	Giselbrecht J; Department of Medicinal Chemistry/Department of Pharmacology, Institute of Pharmacy Martin Luther University Halle-Wittenberg, Wolfgang-Langenbeck-Str. 4, 06120, Halle (Saale), Germany., Pinnapireddy SR; Department of Pharmaceutics and Biopharmaceutics, University of Marburg, Robert-Koch-Str. 4, 35037, Marburg, Germany.; CSL Behring Innovation GmbH, Emil-von-Behring-Str. 76, 35041, Marburg, Germany., Alioglu F; Faculty of Life Sciences, Department of Pharmaceutical Sciences, Laboratory of MacroMolecular Cancer Therapeutics (MMCT), University of Vienna, Althanstrasse 14, Vienna, 1090, Austria., Sami H; Faculty of Life Sciences, Department of Pharmaceutical Sciences, Laboratory of MacroMolecular Cancer Therapeutics (MMCT), University of Vienna, Althanstrasse 14, Vienna, 1090, Austria., Sedding D; Internal Medicine III, Medical Faculty of Martin Luther University Halle-Wittenberg, Ernst-Grube-Str. 40, 06120, Halle (Saale), Germany., Erdmann F; Department of Medicinal Chemistry/Department of Pharmacology, Institute of Pharmacy Martin Luther University Halle-Wittenberg, Wolfgang-Langenbeck-Str. 4, 06120, Halle (Saale), Germany., Janich C; Department of Medicinal Chemistry/Department of Pharmacology, Institute of Pharmacy Martin Luther University Halle-Wittenberg, Wolfgang-Langenbeck-Str. 4, 06120, Halle (Saale), Germany., Schulz-Siegmund M; Pharmaceutical Technology, Medical Faculty, University Leipzig, Eilenburger Straße 15a, 04317, Leipzig, Germany., Ogris M; Faculty of Life Sciences, Department of Pharmaceutical Sciences, Laboratory of MacroMolecular Cancer Therapeutics (MMCT), University of Vienna, Althanstrasse 14, Vienna, 1090, Austria., Bakowsky U; Department of Pharmaceutics and Biopharmaceutics, University of Marburg, Robert-Koch-Str. 4, 35037, Marburg, Germany., Langner A; Department of Medicinal Chemistry/Department of Pharmacology, Institute of Pharmacy Martin Luther University Halle-Wittenberg, Wolfgang-Langenbeck-Str. 4, 06120, Halle (Saale), Germany., Bussmann J; Division of BioTherapeutics, Leiden Academic Center for Drug Research, Leiden University, Einsteinweg 55, Leiden, 2333 CC, The Netherlands., Wölk C; Pharmaceutical Technology, Medical Faculty, University Leipzig, Eilenburger Straße 15a, 04317, Leipzig, Germany.
المصدر:	Small (Weinheim an der Bergstrasse, Germany) [Small] 2022 May; Vol. 18 (18), pp. e2107768. Date of Electronic Publication: 2022 Mar 30.
نوع المنشور:	Journal Article; Research Support, Non-U.S. Gov't
اللغة:	English
بيانات الدورية:	Publisher: Wiley-VCH Country of Publication: Germany NLM ID: 101235338 Publication Model: Print-Electronic Cited Medium: Internet ISSN: 1613-6829 (Electronic) Linking ISSN: 16136810 NLM ISO Abbreviation: Small Subsets: MEDLINE
أسماء مطبوعة:	Original Publication: Weinheim, Germany : Wiley-VCH, c2005-
مواضيع طبية MeSH:	Nucleic Acids*, Animals ; Endothelial Cells ; Lipids ; Liposomes ; Mammals ; Mice ; Nanostructures ; Peptides ; Tissue Distribution ; Transfection
مستخلص:	Formulations based on ionizable amino-lipids have been put into focus as nucleic acid delivery systems. Recently, the in vitro efficacy of the lipid formulation OH4:DOPE has been explored. However, in vitro performance of nanomedicines cannot correctly predict in vivo efficacy, thereby considerably limiting pre-clinical translation. This is further exacerbated by limited access to mammalian models. The present work proposes to close this gap by investigating in vivo nucleic acid delivery within simpler models, but which still offers physiologically complex environments and also adheres to the 3R guidelines (replace/reduce/refine) to improve animal experiments. The efficacy of OH4:DOPE as a delivery system for nucleic acids is demonstrated using in vivo approaches. It is shown that the formulation is able to transfect complex tissues using the chicken chorioallantoic membrane model. The efficacy of DNA and mRNA lipoplexes is tested extensively in the zebra fish (Danio rerio) embryo which allows the screening of biodistribution and transfection efficiency. Effective transfection of blood vessel endothelial cells is seen, especially in the endocardium. Both model systems allow an efficacy screening according to the 3R guidelines bypassing the in vitro-in vivo gap. Pilot studies in mice are performed to correlate the efficacy of in vivo transfection. (© 2022 The Authors. Small published by Wiley-VCH GmbH.)
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فهرسة مساهمة:	Keywords: cationic lipids; ionizable lipids; lipid nanoparticles; mRNA-transfection; pDNA-transfection; zebrafish embryos
المشرفين على المادة:	0 (Lipids) 0 (Liposomes) 0 (Nucleic Acids) 0 (Peptides)
تواريخ الأحداث:	Date Created: 20220331 Date Completed: 20220506 Latest Revision: 20220615
رمز التحديث:	20221213
DOI:	10.1002/smll.202107768
PMID:	35355412
قاعدة البيانات:	MEDLINE

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تدمد:	1613-6829
DOI:	10.1002/smll.202107768