Encapsulation of Dexamethasone into mRNA–Lipid Nanoparticles Is a Promising Approach for the Development of Liver-Targeted Anti-Inflammatory Therapies

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Publicat a:International Journal of Molecular Sciences vol. 25, no. 20 (2024), p. 11254
Autor principal: Ignacio Rivero Berti
Altres autors: Gambaro, Rocío Celeste, Limeres, María José, Huck-Iriart, Cristián, Svensson, Malin, Silvia Fraude-El Ghazi, Pretsch, Leah, Si, Shutian, Lieberwirth, Ingo, Landfester, Katharina, Cacicedo, Maximiliano Luis, Germán Abel Islan, Gehring, Stephan
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024 7 |a 10.3390/ijms252011254  |2 doi 
035 |a 3120650592 
045 2 |b d20241015  |b d20241031 
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100 1 |a Ignacio Rivero Berti  |u Children’s Hospital, University Medical Center of the Johannes Gutenberg University, Langenbeckstr. 1, 55131 Mainz, Germany; <email>ignaciob@uni-mainz.de</email> (I.R.B.); <email>gambaror@uni-mainz.de</email> (R.C.G.); <email>mj.limeres@uni-mainz.de</email> (M.J.L.); <email>malin.svensson@uni-mainz.de</email> (M.S.); <email>fraudesi@uni-mainz.de</email> (S.F.-E.G.); <email>pretsch@uni-mainz.de</email> (L.P.); <email>maximilianocacicedo@gmail.com</email> (M.L.C.); CINDEFI—Centro de Investigación y Desarrollo en Fermentaciones Industriales, Laboratorio de Nanobiomateriales, Facultad de Ciencias Exactas, Universidad Nacional de La Plata (UNLP)-Consejo Nacional de Investigaciones Científicas y Técnicas (CONICET), La Plata 1900, Argentina 
245 1 |a Encapsulation of Dexamethasone into mRNA–Lipid Nanoparticles Is a Promising Approach for the Development of Liver-Targeted Anti-Inflammatory Therapies 
260 |b MDPI AG  |c 2024 
513 |a Journal Article 
520 3 |a The objective of this study was to develop two lipid nanoparticle (LNP) formulations capable of efficiently expressing a reporter mRNA while co-delivering the anti-inflammatory drug dexamethasone (DX) to reduce inflammatory side effects in protein replacement therapies. Two types of LNPs were developed, in which 25% of cholesterol was replaced by DX. These LNPs contained either 1,2-distearoyl-sn-glycero-3-phosphocholine (DSPC) or 1,2-dioleoyl-sn-glycero-3-phosphoethanolamine (DOPE) as a helper lipid. The resulting LNPs exhibited high stability, homogeneity, and near-neutral Zeta potentials. SAXS experiments confirmed DX incorporation into the LNP core, with slow in vitro DX release observed over 48 h. The LNPs achieved high mRNA encapsulation efficiency (95–100%) and effectively transfected HepG2 cells, dendritic cells, and hPBMCs. While LNPs increased cytokine release (IL-1β, TNF-α, MCP-1), LNPs-DX significantly reduced cytokine levels, demonstrating enhanced anti-inflammatory properties while maintaining mRNA expression levels. In vivo biodistribution showed predominant liver localization post-intramuscular injection, regardless of the DSPC or DOPE composition. LNPs co-loaded with mRNA and DX are promising candidates for continuous protein replacement. Due to their ability to reduce treatment-related inflammation while maintaining significant mRNA expression levels, these LNPs are perfectly suited for the treatment of liver-related metabolic diseases. 
651 4 |a Canada 
653 |a Thymus gland 
653 |a Pharmacokinetics 
653 |a Vaccines 
653 |a Lipids 
653 |a Polyethylene glycol 
653 |a Efficiency 
653 |a Cytokines 
653 |a Cholesterol 
653 |a Medical research 
700 1 |a Gambaro, Rocío Celeste  |u Children’s Hospital, University Medical Center of the Johannes Gutenberg University, Langenbeckstr. 1, 55131 Mainz, Germany; <email>ignaciob@uni-mainz.de</email> (I.R.B.); <email>gambaror@uni-mainz.de</email> (R.C.G.); <email>mj.limeres@uni-mainz.de</email> (M.J.L.); <email>malin.svensson@uni-mainz.de</email> (M.S.); <email>fraudesi@uni-mainz.de</email> (S.F.-E.G.); <email>pretsch@uni-mainz.de</email> (L.P.); <email>maximilianocacicedo@gmail.com</email> (M.L.C.) 
700 1 |a Limeres, María José  |u Children’s Hospital, University Medical Center of the Johannes Gutenberg University, Langenbeckstr. 1, 55131 Mainz, Germany; <email>ignaciob@uni-mainz.de</email> (I.R.B.); <email>gambaror@uni-mainz.de</email> (R.C.G.); <email>mj.limeres@uni-mainz.de</email> (M.J.L.); <email>malin.svensson@uni-mainz.de</email> (M.S.); <email>fraudesi@uni-mainz.de</email> (S.F.-E.G.); <email>pretsch@uni-mainz.de</email> (L.P.); <email>maximilianocacicedo@gmail.com</email> (M.L.C.) 
700 1 |a Huck-Iriart, Cristián  |u ALBA Synchrotron Light Source, Carrer de la Llum 2–26, Cerdanyola del Vallès, 08290 Barcelona, Spain; <email>chuck@cells.es</email> 
700 1 |a Svensson, Malin  |u Children’s Hospital, University Medical Center of the Johannes Gutenberg University, Langenbeckstr. 1, 55131 Mainz, Germany; <email>ignaciob@uni-mainz.de</email> (I.R.B.); <email>gambaror@uni-mainz.de</email> (R.C.G.); <email>mj.limeres@uni-mainz.de</email> (M.J.L.); <email>malin.svensson@uni-mainz.de</email> (M.S.); <email>fraudesi@uni-mainz.de</email> (S.F.-E.G.); <email>pretsch@uni-mainz.de</email> (L.P.); <email>maximilianocacicedo@gmail.com</email> (M.L.C.) 
700 1 |a Silvia Fraude-El Ghazi  |u Children’s Hospital, University Medical Center of the Johannes Gutenberg University, Langenbeckstr. 1, 55131 Mainz, Germany; <email>ignaciob@uni-mainz.de</email> (I.R.B.); <email>gambaror@uni-mainz.de</email> (R.C.G.); <email>mj.limeres@uni-mainz.de</email> (M.J.L.); <email>malin.svensson@uni-mainz.de</email> (M.S.); <email>fraudesi@uni-mainz.de</email> (S.F.-E.G.); <email>pretsch@uni-mainz.de</email> (L.P.); <email>maximilianocacicedo@gmail.com</email> (M.L.C.) 
700 1 |a Pretsch, Leah  |u Children’s Hospital, University Medical Center of the Johannes Gutenberg University, Langenbeckstr. 1, 55131 Mainz, Germany; <email>ignaciob@uni-mainz.de</email> (I.R.B.); <email>gambaror@uni-mainz.de</email> (R.C.G.); <email>mj.limeres@uni-mainz.de</email> (M.J.L.); <email>malin.svensson@uni-mainz.de</email> (M.S.); <email>fraudesi@uni-mainz.de</email> (S.F.-E.G.); <email>pretsch@uni-mainz.de</email> (L.P.); <email>maximilianocacicedo@gmail.com</email> (M.L.C.) 
700 1 |a Si, Shutian  |u Max Planck Institute for Polymer Research, Department of Physical Chemistry of Polymers, Ackermannweg 10, 55128 Mainz, Germany; <email>sis2@mpip-mainz.mpg.de</email> (S.S.); <email>lieberw@mpip-mainz.mpg.de</email> (I.L.); <email>landfest@uni-mainz.de</email> (K.L.) 
700 1 |a Lieberwirth, Ingo  |u Max Planck Institute for Polymer Research, Department of Physical Chemistry of Polymers, Ackermannweg 10, 55128 Mainz, Germany; <email>sis2@mpip-mainz.mpg.de</email> (S.S.); <email>lieberw@mpip-mainz.mpg.de</email> (I.L.); <email>landfest@uni-mainz.de</email> (K.L.) 
700 1 |a Landfester, Katharina  |u Max Planck Institute for Polymer Research, Department of Physical Chemistry of Polymers, Ackermannweg 10, 55128 Mainz, Germany; <email>sis2@mpip-mainz.mpg.de</email> (S.S.); <email>lieberw@mpip-mainz.mpg.de</email> (I.L.); <email>landfest@uni-mainz.de</email> (K.L.) 
700 1 |a Cacicedo, Maximiliano Luis  |u Children’s Hospital, University Medical Center of the Johannes Gutenberg University, Langenbeckstr. 1, 55131 Mainz, Germany; <email>ignaciob@uni-mainz.de</email> (I.R.B.); <email>gambaror@uni-mainz.de</email> (R.C.G.); <email>mj.limeres@uni-mainz.de</email> (M.J.L.); <email>malin.svensson@uni-mainz.de</email> (M.S.); <email>fraudesi@uni-mainz.de</email> (S.F.-E.G.); <email>pretsch@uni-mainz.de</email> (L.P.); <email>maximilianocacicedo@gmail.com</email> (M.L.C.) 
700 1 |a Germán Abel Islan  |u Children’s Hospital, University Medical Center of the Johannes Gutenberg University, Langenbeckstr. 1, 55131 Mainz, Germany; <email>ignaciob@uni-mainz.de</email> (I.R.B.); <email>gambaror@uni-mainz.de</email> (R.C.G.); <email>mj.limeres@uni-mainz.de</email> (M.J.L.); <email>malin.svensson@uni-mainz.de</email> (M.S.); <email>fraudesi@uni-mainz.de</email> (S.F.-E.G.); <email>pretsch@uni-mainz.de</email> (L.P.); <email>maximilianocacicedo@gmail.com</email> (M.L.C.); CINDEFI—Centro de Investigación y Desarrollo en Fermentaciones Industriales, Laboratorio de Nanobiomateriales, Facultad de Ciencias Exactas, Universidad Nacional de La Plata (UNLP)-Consejo Nacional de Investigaciones Científicas y Técnicas (CONICET), La Plata 1900, Argentina 
700 1 |a Gehring, Stephan  |u Children’s Hospital, University Medical Center of the Johannes Gutenberg University, Langenbeckstr. 1, 55131 Mainz, Germany; <email>ignaciob@uni-mainz.de</email> (I.R.B.); <email>gambaror@uni-mainz.de</email> (R.C.G.); <email>mj.limeres@uni-mainz.de</email> (M.J.L.); <email>malin.svensson@uni-mainz.de</email> (M.S.); <email>fraudesi@uni-mainz.de</email> (S.F.-E.G.); <email>pretsch@uni-mainz.de</email> (L.P.); <email>maximilianocacicedo@gmail.com</email> (M.L.C.) 
773 0 |t International Journal of Molecular Sciences  |g vol. 25, no. 20 (2024), p. 11254 
786 0 |d ProQuest  |t Health & Medical Collection 
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