Recent Advances in Lipid Nanoparticles for Delivery of mRNA

Abstract

Messenger RNA (mRNA), which is composed of ribonucleotides that carry genetic information and direct protein synthesis, is transcribed from a strand of DNA as a template. On this basis, mRNA technology can take advantage of the body’s own translation system to express proteins with multiple functions for the treatment of various diseases. Due to the advancement of mRNA synthesis and purification, modification and sequence optimization technologies, and the emerging lipid nanomaterials and other delivery systems, mRNA therapeutic regimens are becoming clinically feasible and exhibit significant reliability in mRNA stability, translation efficiency, and controlled immunogenicity. Lipid nanoparticles (LNPs), currently the leading non-viral delivery vehicles, have made many exciting advances in clinical translation as part of the COVID-19 vaccines and therefore have the potential to accelerate the clinical translation of gene drugs. Additionally, due to their small size, biocompatibility, and biodegradability, LNPs can effectively deliver nucleic acids into cells, which is particularly important for the current mRNA regimens. Therefore, the cutting-edge LNP@mRNA regimens hold great promise for cancer vaccines, infectious disease prevention, protein replacement therapy, gene editing, and rare disease treatment. To shed more lights on LNP@mRNA, this paper mainly discusses the rational of choosing LNPs as the non-viral vectors to deliver mRNA, the general rules for mRNA optimization and LNP preparation, and the various parameters affecting the delivery efficiency of LNP@mRNA, and finally summarizes the current research status as well as the current challenges. The latest research progress of LNPs in the treatment of other diseases such as oncological, cardiovascular, and infectious diseases is also given. Finally, the future applications and perspectives for LNP@mRNA are generally introduced.

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    Hanson G., Coller J. Codon optimality, bias and usage in translation and mRNA decay. Nat. Rev. Mol. Cell. Biol. 2018;19:20-30.

    Not in our library10.1038/nrm.2017.91

89. 89.

    Hubert B. The CureVac Vaccine, and a Brief Tour through Some of the Wonders of Nature. 2021.

    Not in our library

90. 90.

    Trepotec Z., Geiger J., Plank C., Aneja M.K., Rudolph C. Segmented poly(A) tails significantly reduce recombination of plasmid DNA without affecting mRNA translation efficiency or half-life. RNA. 2019;25:507-518.

    Not in our library10.1261/rna.069286.118

91. 91.

    Allen T.M., Cullis P.R. Liposomal drug delivery systems: From concept to clinical applications. Adv. Drug Deliv. Rev. 2013;65:36-48.

    Not in our library10.1016/j.addr.2012.09.037

92. 92.

    Hafez I.M., Maurer N., Cullis P.R. On the mechanism whereby cationic lipids promote intracellular delivery of polynucleic acids. Gene Ther. 2001;8:1188-1196.

    Not in our library10.1038/sj.gt.3301506

93. 93.
    Advances in Lipid Nanoparticles for siRNA Delivery · 2013

    No notice
94. 94.

    Gruner S.M., Cullis P.R., Hope M.J., Tilcock C.P. Lipid polymorphism: The molecular basis of nonbilayer phases. Annu. Rev. Biophys. Biophys. Chem. 1985;14:211-238.

    Not in our library10.1146/annurev.bb.14.060185.001235

95. 95.

    Witzigmann D., Kulkarni J.A., Leung J., Chen S., Cullis P.R., van der Meel R. Lipid nanoparticle technology for therapeutic gene regulation in the liver. Adv. Drug Deliv. Rev. 2020;159:344-363.

    Not in our library10.1016/j.addr.2020.06.026

96. 96.
    Membrane‐destabilizing ionizable lipid empowered imaging‐guided siRNA delivery and cancer treatment · 2021

    No notice
97. 97.

    Maier M.A., Jayaraman M., Matsuda S., Liu J., Barros S., Querbes W., Tam Y.K., Ansell S.M., Kumar V., Qin J., et al. Biodegradable lipids enabling rapidly eliminated lipid nanoparticles for systemic delivery of RNAi therapeutics. Mol. Ther. 2013;21:1570-1578.

    Not in our library10.1038/mt.2013.124

98. 98.

    Semple S.C., Klimuk S.K., Harasym T.O., Dos Santos N., Ansell S.M., Wong K.F., Maurer N., Stark H., Cullis P.R., Hope M.J., et al. Efficient encapsulation of antisense oligonucleotides in lipid vesicles using ionizable aminolipids: Formation of novel small multilamellar vesicle structures. Biochim. Biophys. Acta. 2001;1510:152-166.

    Not in our library10.1016/s0005-2736(00)00343-6

99. 99.

    Semple S.C., Akinc A., Chen J., Sandhu A.P., Mui B.L., Cho C.K., Sah D.W., Stebbing D., Crosley E.J., Yaworski E., et al. Rational design of cationic lipids for siRNA delivery. Nat. Biotechnol. 2010;28:172-176.

    Not in our library10.1038/nbt.1602

100. 100.
     Maximizing the Potency of siRNA Lipid Nanoparticles for Hepatic Gene Silencing In Vivo** · 2012

     No notice
101. 101.

     Akinc A., Zumbuehl A., Goldberg M., Leshchiner E.S., Busini V., Hossain N., Bacallado S.A., Nguyen D.N., Fuller J., Alvarez R., et al. A combinatorial library of lipid-like materials for delivery of RNAi therapeutics. Nat. Biotechnol. 2008;26:561-569.

     Not in our library10.1038/nbt1402

102. 102.

     Sabnis S., Kumarasinghe E.S., Salerno T., Mihai C., Ketova T., Senn J.J., Lynn A., Bulychev A., McFadyen I., Chan J., et al. A Novel Amino Lipid Series for mRNA Delivery: Improved Endosomal Escape and Sustained Pharmacology and Safety in Non-human Primates. Mol. Ther. 2018;26:1509-1519.

     Not in our library10.1016/j.ymthe.2018.03.010

103. 103.

     Kauffman K.J., Dorkin J.R., Yang J.H., Heartlein M.W., DeRosa F., Mir F.F., Fenton O.S., Anderson D.G. Optimization of Lipid Nanoparticle Formulations for mRNA Delivery in Vivo with Fractional Factorial and Definitive Screening Designs. Nano Lett. 2015;15:7300-7306.

     Not in our library10.1021/acs.nanolett.5b02497

104. 104.

     Yu X., Liu S., Cheng Q., Wei T., Lee S., Zhang D., Siegwart D.J. Lipid-Modified Aminoglycosides for mRNA Delivery to the Liver. Adv. Healthc. Mater. 2020;9:e1901487.

     Not in our library10.1002/adhm.201901487

105. 105.
     An ionizable lipid toolbox for RNA delivery · 2021

     No notice
106. 106.

     Miao L., Li L., Huang Y., Delcassian D., Chahal J., Han J., Shi Y., Sadtler K., Gao W., Lin J., et al. Delivery of mRNA vaccines with heterocyclic lipids increases anti-tumor efficacy by STING-mediated immune cell activation. Nat. Biotechnol. 2019;37:1174-1185.

     Not in our library10.1038/s41587-019-0247-3

107. 107.

     INNORNA. INNORNA: Explore Frontier of mRNA Application Based on Platform Technology. 2022.

     Not in our library

108. 108.

     Xiao Q., Li X., Liu C., Yang Y., Hou Y., Wang Y., Su M., He W. Liposome-based anchoring and core-encapsulation for combinatorial cancer therapy. Chin. Chem. Lett. 2022;33:4191-4196.

     Not in our library10.1016/j.cclet.2022.01.083

109. 109.

     Hu J., Yuan X., Wang F., Gao H., Liu X., Zhang W. The progress and perspective of strategies to improve tumor penetration of nanomedicines. Chin. Chem. Lett. 2021;32:1341-1347.

     Not in our library10.1016/j.cclet.2020.11.006

110. 110.

     Cheng Q., Wei T., Jia Y., Farbiak L., Zhou K., Zhang S., Wei Y., Zhu H., Siegwart D.J. Dendrimer-Based Lipid Nanoparticles Deliver Therapeutic FAH mRNA to Normalize Liver Function and Extend Survival in a Mouse Model of Hepatorenal Tyrosinemia Type I. Adv. Mater. 2018;30:e1805308.

     Not in our library10.1002/adma.201805308

111. 111.

     Kulkarni J.A., Witzigmann D., Leung J., Tam Y.Y.C., Cullis P.R. On the role of helper lipids in lipid nanoparticle formulations of siRNA. Nanoscale. 2019;11:21733-21739.

     Not in our library10.1039/c9nr09347h

112. 112.

     Miller J.B., Zhang S., Kos P., Xiong H., Zhou K., Perelman S.S., Zhu H., Siegwart D.J. Non-Viral CRISPR/Cas Gene Editing In Vitro and In Vivo Enabled by Synthetic Nanoparticle Co-Delivery of Cas9 mRNA and sgRNA. Angew. Chem. Int. Ed. Engl. 2017;56:1059-1063.

     Not in our library10.1002/anie.201610209

113. 113.

     Liu S., Cheng Q., Wei T., Yu X., Johnson L.T., Farbiak L., Siegwart D.J. Membrane-destabilizing ionizable phospholipids for organ-selective mRNA delivery and CRISPR-Cas gene editing. Nat. Mater. 2021;20:701-710.

     Not in our library10.1038/s41563-020-00886-0

114. 114.

     Takahashi H., Sinoda K., Hatta I. Effects of cholesterol on the lamellar and the inverted hexagonal phases of dielaidoylphosphatidylethanolamine. Biochim. Biophys. Acta. 1996;1289:209-216.

     Not in our library10.1016/0304-4165(95)00170-0

115. 115.

     Choi H.S., Liu W., Misra P., Tanaka E., Zimmer J.P., Itty Ipe B., Bawendi M.G., Frangioni J.V. Renal clearance of quantum dots. Nat. Biotechnol. 2007;25:1165-1170.

     Not in our library10.1038/nbt1340

116. 116.

     Braet F., Wisse E., Bomans P., Frederik P., Geerts W., Koster A., Soon L., Ringer S. Contribution of high-resolution correlative imaging techniques in the study of the liver sieve in three-dimensions. Microsc. Res. Tech. 2007;70:230-242.

     Not in our library10.1002/jemt.20408

117. 117.

     Moghimi S.M., Hunter A.C., Andresen T.L. Factors controlling nanoparticle pharmacokinetics: An integrated analysis and perspective. Annu. Rev. Pharmacol. Toxicol. 2012;52:481-503.

     Not in our library10.1146/annurev-pharmtox-010611-134623

118. 118.

     Belliveau N.M., Huft J., Lin P.J., Chen S., Leung A.K., Leaver T.J., Wild A.W., Lee J.B., Taylor R.J., Tam Y.K., et al. Microfluidic Synthesis of Highly Potent Limit-size Lipid Nanoparticles for In Vivo Delivery of siRNA. Mol. Ther. Nucleic Acids. 2012;1:e37.

     Not in our library10.1038/mtna.2012.28

119. 119.

     Basha G., Novobrantseva T.I., Rosin N., Tam Y.Y., Hafez I.M., Wong M.K., Sugo T., Ruda V.M., Qin J., Klebanov B., et al. Influence of cationic lipid composition on gene silencing properties of lipid nanoparticle formulations of siRNA in antigen-presenting cells. Mol. Ther. 2011;19:2186-2200.

     Not in our library10.1038/mt.2011.190

120. 120.

     Chen S., Tam Y.Y.C., Lin P.J.C., Sung M.M.H., Tam Y.K., Cullis P.R. Influence of particle size on the in vivo potency of lipid nanoparticle formulations of siRNA. J. Control. Release. 2016;235:236-244.

     Not in our library10.1016/j.jconrel.2016.05.059

121. 121.

     Yanez Arteta M., Kjellman T., Bartesaghi S., Wallin S., Wu X., Kvist A.J., Dabkowska A., Székely N., Radulescu A., Bergenholtz J., et al. Successful reprogramming of cellular protein production through mRNA delivered by functionalized lipid nanoparticles. Proc. Natl. Acad. Sci. USA. 2018;115:3351-3360.

     Not in our library10.1073/pnas.1720542115

122. 122.

     Hassett K.J., Higgins J., Woods A., Levy B., Xia Y., Hsiao C.J., Acosta E., Almarsson Ö., Moore M.J., Brito L.A. Impact of lipid nanoparticle size on mRNA vaccine immunogenicity. J. Control. Release. 2021;335:237-246.

     Not in our library10.1016/j.jconrel.2021.05.021

123. 123.

     Kedmi R., Veiga N., Ramishetti S., Goldsmith M., Rosenblum D., Dammes N., Hazan-Halevy I., Nahary L., Leviatan-Ben-Arye S., Harlev M., et al. A modular platform for targeted RNAi therapeutics. Nat. Nanotechnol. 2018;13:214-219.

     Not in our library10.1038/s41565-017-0043-5

124. 124.
     mRNA Vaccines Encoding the HA Protein of Influenza A H1N1 Virus Delivered by Cationic Lipid Nanoparticles Induce Protective Immune Responses in Mice · 2020

     No notice
125. 125.

     Rurik J.G., Tombácz I., Yadegari A., Méndez Fernández P.O., Shewale S.V., Li L., Kimura T., Soliman O.Y., Papp T.E., Tam Y.K., et al. CAR T cells produced in vivo to treat cardiac injury. Science. 2022;375:91-96.

     Not in our library10.1126/science.abm0594

126. 126.

     Dammes N., Goldsmith M., Ramishetti S., Dearling J.L.J., Veiga N., Packard A.B., Peer D. Conformation-sensitive targeting of lipid nanoparticles for RNA therapeutics. Nat. Nanotechnol. 2021;16:1030-1038.

     Not in our library10.1038/s41565-021-00928-x

127. 127.

     Marcos-Contreras O.A., Greineder C.F., Kiseleva R.Y., Parhiz H., Walsh L.R., Zuluaga-Ramirez V., Myerson J.W., Hood E.D., Villa C.H., Tombacz I., et al. Selective targeting of nanomedicine to inflamed cerebral vasculature to enhance the blood-brain barrier. Proc. Natl. Acad. Sci. USA. 2020;117:3405-3414.

     Not in our library10.1073/pnas.1912012117

128. 128.

     Parhiz H., Shuvaev V.V., Pardi N., Khoshnejad M., Kiseleva R.Y., Brenner J.S., Uhler T., Tuyishime S., Mui B.L., Tam Y.K., et al. PECAM-1 directed re-targeting of exogenous mRNA providing two orders of magnitude enhancement of vascular delivery and expression in lungs independent of apolipoprotein E-mediated uptake. J. Control. Release. 2018;291:106-115.

     Not in our library10.1016/j.jconrel.2018.10.015

129. 129.

     Su F.Y., Zhao Q.H., Dahotre S.N., Gamboa L., Bawage S.S., Silva Trenkle A.D., Zamat A., Phuengkham H., Ahmed R., Santangelo P.J., et al. In vivo mRNA delivery to virus-specific T cells by light-induced ligand exchange of MHC class I antigen-presenting nanoparticles. Sci. Adv. 2022;8:eabm7950.

     Not in our library10.1126/sciadv.abm7950

130. 130.

     Sago C.D., Lokugamage M.P., Paunovska K., Vanover D.A., Monaco C.M., Shah N.N., Gamboa Castro M., Anderson S.E., Rudoltz T.G., Lando G.N., et al. High-throughput in vivo screen of functional mRNA delivery identifies nanoparticles for endothelial cell gene editing. Proc. Natl. Acad. Sci. USA. 2018;115:9944-9952.

     Not in our library10.1073/pnas.1811276115

131. 131.

     Cheng Q., Wei T., Farbiak L., Johnson L.T., Dilliard S.A., Siegwart D.J. Selective organ targeting (SORT) nanoparticles for tissue-specific mRNA delivery and CRISPR-Cas gene editing. Nat. Nanotechnol. 2020;15:313-320.

     Not in our library10.1038/s41565-020-0669-6

132. 132.

     Dilliard S.A., Cheng Q., Siegwart D.J. On the mechanism of tissue-specific mRNA delivery by selective organ targeting nanoparticles. Proc. Natl. Acad. Sci. USA. 2021;118:2109256118.

     Not in our library10.1073/pnas.2109256118

133. 133.

     Fenton O.S., Kauffman K.J., Kaczmarek J.C., McClellan R.L., Jhunjhunwala S., Tibbitt M.W., Zeng M.D., Appel E.A., Dorkin J.R., Mir F.F., et al. Synthesis and Biological Evaluation of Ionizable Lipid Materials for the In Vivo Delivery of Messenger RNA to B Lymphocytes. Adv. Mater. 2017;29:1606944.

     Not in our library10.1002/adma.201606944

134. 134.

     Zhao X., Chen J., Qiu M., Li Y., Glass Z., Xu Q. Imidazole-Based Synthetic Lipidoids for In Vivo mRNA Delivery into Primary T Lymphocytes. Angew. Chem. Int. Ed. 2020;59:20083-20089.

     Not in our library10.1002/anie.202008082

135. 135.

     Qiu M., Tang Y., Chen J., Muriph R., Ye Z., Huang C., Evans J., Henske E.P., Xu Q. Lung-selective mRNA delivery of synthetic lipid nanoparticles for the treatment of pulmonary lymphangioleiomyomatosis. Proc. Natl. Acad. Sci. USA. 2022;119:2116271119.

     Not in our library10.1073/pnas.2116271119

136. 136.

     Gan Z., Lokugamage M.P., Hatit M.Z.C., Loughrey D., Paunovska K., Sato M., Cristian A., Dahlman J.E. Nanoparticles containing constrained phospholipids deliver mRNA to liver immune cells in vivo without targeting ligands. Bioeng. Transl. Med. 2020;5:e10161.

     Not in our library10.1002/btm2.10161

137. 137.

     Kim M., Jeong M., Hur S., Cho Y., Park J., Jung H., Seo Y., Woo H.A., Nam K.T., Lee K., et al. Engineered ionizable lipid nanoparticles for targeted delivery of RNA therapeutics into different types of cells in the liver. Sci. Adv. 2021;7:eabf4398.

     Not in our library10.1126/sciadv.abf4398

138. 138.

     Banerji A., Wickner P.G., Saff R., Stone C.A., Robinson L.B., Long A.A., Wolfson A.R., Williams P., Khan D.A., Phillips E., et al. mRNA Vaccines to Prevent COVID-19 Disease and Reported Allergic Reactions: Current Evidence and Suggested Approach. J. Allergy Clin. Immunol. Pract. 2021;9:1423-1437.

     Not in our library10.1016/j.jaip.2020.12.047

139. 139.

     Selvaraj G., Kaliamurthi S., Peslherbe G.H., Wei D.Q. Are the Allergic Reactions of COVID-19 Vaccines Caused by mRNA Constructs or Nanocarriers? Immunological Insights. Interdiscip. Sci. 2021;13:344-347.

     Not in our library10.1007/s12539-021-00438-3

140. 140.

     Aalberse R.C. Structural biology of allergens. J. Allergy Clin. Immunol. 2000;106:228-238.

     Not in our library10.1067/mai.2000.108434

141. 141.

     Kauffman K.J., Mir F.F., Jhunjhunwala S., Kaczmarek J.C., Hurtado J.E., Yang J.H., Webber M.J., Kowalski P.S., Heartlein M.W., DeRosa F., et al. Efficacy and immunogenicity of unmodified and pseudouridine-modified mRNA delivered systemically with lipid nanoparticles in vivo. Biomaterials. 2016;109:78-87.

     Not in our library10.1016/j.biomaterials.2016.09.006

142. 142.

     Halamoda-Kenzaoui B., Bremer-Hoffmann S. Main trends of immune effects triggered by nanomedicines in preclinical studies. Int. J. Nanomed. 2018;13:5419-5431.

     Not in our library10.2147/ijn.s168808

143. 143.

     Abrams M.T., Koser M.L., Seitzer J., Williams S.C., DiPietro M.A., Wang W., Shaw A.W., Mao X., Jadhav V., Davide J.P., et al. Evaluation of efficacy, biodistribution, and inflammation for a potent siRNA nanoparticle: Effect of dexamethasone co-treatment. Mol. Ther. 2010;18:171-180.

     Not in our library10.1038/mt.2009.208

144. 144.

     Tahtinen S., Tong A.J., Himmels P., Oh J., Paler-Martinez A., Kim L., Wichner S., Oei Y., McCarron M.J., Freund E.C., et al. IL-1 and IL-1ra are key regulators of the inflammatory response to RNA vaccines. Nat. Immunol. 2022;23:532-542.

     Not in our library10.1038/s41590-022-01160-y

145. 145.

     Sedic M., Senn J.J., Lynn A., Laska M., Smith M., Platz S.J., Bolen J., Hoge S., Bulychev A., Jacquinet E., et al. Safety Evaluation of Lipid Nanoparticle-Formulated Modified mRNA in the Sprague-Dawley Rat and Cynomolgus Monkey. Vet. Pathol. 2018;55:341-354.

     Not in our library10.1177/0300985817738095

146. 146.

     Szebeni J. Complement activation-related pseudoallergy: A stress reaction in blood triggered by nanomedicines and biologicals. Mol. Immunol. 2014;61:163-173.

     Not in our library10.1016/j.molimm.2014.06.038

147. 147.

     Chen S., Zaifman J., Kulkarni J.A., Zhigaltsev I.V., Tam Y.K., Ciufolini M.A., Tam Y.Y.C., Cullis P.R. Dexamethasone prodrugs as potent suppressors of the immunostimulatory effects of lipid nanoparticle formulations of nucleic acids. J. Control. Release. 2018;286:46-54.

     Not in our library10.1016/j.jconrel.2018.07.026

148. 148.

     Tao W., Mao X., Davide J.P., Ng B., Cai M., Burke P.A., Sachs A.B., Sepp-Lorenzino L. Mechanistically probing lipid-siRNA nanoparticle-associated toxicities identifies Jak inhibitors effective in mitigating multifaceted toxic responses. Mol. Ther. 2011;19:567-575.

     Not in our library10.1038/mt.2010.282

149. 149.

     Semple S.C., Harasym T.O., Clow K.A., Ansell S.M., Klimuk S.K., Hope M.J. Immunogenicity and rapid blood clearance of liposomes containing polyethylene glycol-lipid conjugates and nucleic Acid. J. Pharmacol. Exp. Ther. 2005;312:1020-1026.

     Not in our library10.1124/jpet.104.078113

150. 150.

     Ambegia E., Ansell S., Cullis P., Heyes J., Palmer L., MacLachlan I. Stabilized plasmid-lipid particles containing PEG-diacylglycerols exhibit extended circulation lifetimes and tumor selective gene expression. Biochim. Biophys. Acta. 2005;1669:155-163.

     Not in our library10.1016/j.bbamem.2005.02.001

151. 151.

     Allison S.J., Milner J. RNA Interference by Single- and Double-stranded siRNA With a DNA Extension Containing a 3’ Nuclease-resistant Mini-hairpin Structure. Mol. Ther. Nucleic Acids. 2014;2:e141.

     Not in our library10.1038/mtna.2013.68

152. 152.

     Nogueira S.S., Schlegel A., Maxeiner K., Weber B., Barz M., Schroer M.A., Blanchet C.E., Svergun D.I., Ramishetti S., Peer D., et al. Polysarcosine-Functionalized Lipid Nanoparticles for Therapeutic mRNA Delivery. ACS Appl. Nano Mater. 2020;3:10634-10645.

     Not in our library10.1021/acsanm.0c01834

153. 153.

     Kostoff R.N., Calina D., Kanduc D., Briggs M.B., Vlachoyiannopoulos P., Svistunov A.A., Tsatsakis A. RETRACTED: Why are we vaccinating children against COVID-19? Toxicol. Rep. 2021;8:1665-1684.

     Not in our library10.1016/j.toxrep.2021.08.010

154. 154.

     Fan Y., Marioli M., Zhang K. Analytical characterization of liposomes and other lipid nanoparticles for drug delivery. J. Pharm. Biomed. Anal. 2021;192:113642.

     Not in our library10.1016/j.jpba.2020.113642

155. 155.

     Jaeger J., Sorensen K., Wolff S.P. Peroxide accumulation in detergents. J. Biochem. Biophys. Methods. 1994;29:77-81.

     Not in our library10.1016/0165-022x(94)90058-2

156. 156.

     Ball R.L., Bajaj P., Whitehead K.A. Achieving long-term stability of lipid nanoparticles: Examining the effect of pH, temperature, and lyophilization. Int. J. Nanomed. 2016;12:305-315.

     Not in our library10.2147/ijn.s123062

157. 157.
     mRNA-lipid nanoparticle COVID-19 vaccines: Structure and stability · 2021

     No notice
158. 158.

     Pascolo S. Vaccination with messenger RNA (mRNA). Toll-Like Receptors (TLRs) and Innate Immunity. 2008;221-235.

     Not in our library10.1007/978-3-540-72167-3_11

159. 159.
     Theoretical basis for stabilizing messenger RNA through secondary structure design · 2021

     No notice
160. 160.

     Karikó K., Muramatsu H., Ludwig J., Weissman D. Generating the optimal mRNA for therapy: HPLC purification eliminates immune activation and improves translation of nucleoside-modified, protein-encoding mRNA. Nucleic Acids Res. 2011;39:e142.

     Not in our library10.1093/nar/gkr695

161. 161.
     A Facile Method for the Removal of dsRNA Contaminant from In Vitro-Transcribed mRNA · 2019

     No notice
162. 162.

     Zhao P., Hou X., Yan J., Du S., Xue Y., Li W., Xiang G., Dong Y. Long-term storage of lipid-like nanoparticles for mRNA delivery. Bioact. Mater. 2020;5:358-363.

     Not in our library10.1016/j.bioactmat.2020.03.001

163. 163.

     Jayesh K., Austin H., Pieter C., Blair L., Terri P., Pamela W. Compositions and Systems Comprising Transfection-Competent Vesicles Free of Organic-Solvents and Detergents and Methods Related Thereto. U.S. Patent. 2022.

     Not in our library

164. 164.
     Lyophilized mRNA-lipid nanoparticle vaccines with long-term stability and high antigenicity against SARS-CoV-2 · 2023

     No notice
165. 165.
     Aerosolizable Lipid Nanoparticles for Pulmonary Delivery of mRNA through Design of Experiments · 2020

     No notice
166. 166.

     Lokugamage M.P., Vanover D., Beyersdorf J., Hatit M.Z.C., Rotolo L., Echeverri E.S., Peck H.E., Ni H., Yoon J.K., Kim Y., et al. Optimization of lipid nanoparticles for the delivery of nebulized therapeutic mRNA to the lungs. Nat. Biomed. Eng. 2021;5:1059-1068.

     Not in our library10.1038/s41551-021-00786-x

167. 167.

     Kim J., Jozic A., Sahay G. Naturally Derived Membrane Lipids Impact Nanoparticle-Based Messenger RNA Delivery. Cell. Mol. Bioeng. 2020;13:463-474.

     Not in our library10.1007/s12195-020-00619-y

168. 168.

     Guimaraes P.P.G., Zhang R., Spektor R., Tan M., Chung A., Billingsley M.M., El-Mayta R., Riley R.S., Wang L., Wilson J.M., et al. Ionizable lipid nanoparticles encapsulating barcoded mRNA for accelerated in vivo delivery screening. J. Control. Release. 2019;316:404-417.

     Not in our library10.1016/j.jconrel.2019.10.028

169. 169.

     Hashiba A., Toyooka M., Sato Y., Maeki M., Tokeshi M., Harashima H. The use of design of experiments with multiple responses to determine optimal formulations for in vivo hepatic mRNA delivery. J. Control. Release. 2020;327:467-476.

     Not in our library10.1016/j.jconrel.2020.08.031

170. 170.
     Shielding of Lipid Nanoparticles for siRNA Delivery: Impact on Physicochemical Properties, Cytokine Induction, and Efficacy · 2014

     No notice
171. 171.

     Saadati R., Dadashzadeh S., Abbasian Z., Soleimanjahi H. Accelerated blood clearance of PEGylated PLGA nanoparticles following repeated injections: Effects of polymer dose, PEG coating, and encapsulated anticancer drug. Pharm. Res. 2013;30:985-995.

     Not in our library10.1007/s11095-012-0934-y

172. 172.

     Zukancic D., Suys E.J.A., Pilkington E.H., Algarni A., Al-Wassiti H., Truong N.P. The Importance of Poly(ethylene glycol) and Lipid Structure in Targeted Gene Delivery to Lymph Nodes by Lipid Nanoparticles. Pharmaceutics. 2020;12:1068.

     Not in our library10.3390/pharmaceutics12111068

173. 173.

     Del Pozo-Rodríguez A., Solinís M., Rodríguez-Gascón A. Applications of lipid nanoparticles in gene therapy. Eur. J. Pharm. Biopharm. 2016;109:184-193.

     Not in our library10.1016/j.ejpb.2016.10.016

174. 174.
     Modified mRNA/lipid nanoparticle-based vaccines expressing respiratory syncytial virus F protein variants are immunogenic and protective in rodent models of RSV infection · 2020

     No notice
175. 175.

     Aldrich C., Leroux-Roels I., Huang K.B., Bica M.A., Loeliger E., Schoenborn-Kellenberger O., Walz L., Leroux-Roels G., von Sonnenburg F., Oostvogels L. Proof-of-concept of a low-dose unmodified mRNA-based rabies vaccine formulated with lipid nanoparticles in human volunteers: A phase 1 trial. Vaccine. 2021;39:1310-1318.

     Not in our library10.1016/j.vaccine.2020.12.070

176. 176.

     Saxena S., Sonwane A.A., Dahiya S.S., Patel C.L., Saini M., Rai A., Gupta P.K. Induction of immune responses and protection in mice against rabies using a self-replicating RNA vaccine encoding rabies virus glycoprotein. Vet. Microbiol. 2009;136:36-44.

     Not in our library10.1016/j.vetmic.2008.10.030

177. 177.

     VanBlargan L.A., Himansu S., Foreman B.M., Ebel G.D., Pierson T.C., Diamond M.S. An mRNA Vaccine Protects Mice against Multiple Tick-Transmitted Flavivirus Infections. Cell Rep. 2018;25:3382-3392.e3.

     Not in our library10.1016/j.celrep.2018.11.082

178. 178.
     Murine liver repair via transient activation of regenerative pathways in hepatocytes using lipid nanoparticle-complexed nucleoside-modified mRNA · 2021

     Corrected
179. 179.

     Grunwitz C., Kranz L.M. mRNA Cancer Vaccines-Messages that Prevail. Curr. Top. Microbiol. Immunol. 2017;405:145-164.

     Not in our library10.1007/82_2017_509

180. 180.

     Zhang R., Billingsley M.M., Mitchell M.J. Biomaterials for vaccine-based cancer immunotherapy. J. Control. Release. 2018;292:256-276.

     Not in our library10.1016/j.jconrel.2018.10.008

181. 181.

     Lee K., Kim S.Y., Seo Y., Kim M.H., Chang J., Lee H. Adjuvant incorporated lipid nanoparticles for enhanced mRNA-mediated cancer immunotherapy. Biomater. Sci. 2020;8:1101-1105.

     Not in our library10.1039/c9bm01564g

182. 182.
     Combining p53 mRNA nanotherapy with immune checkpoint blockade reprograms the immune microenvironment for effective cancer therapy · 2022

     No notice
183. 183.

     Li W., Zhang X., Zhang C., Yan J., Hou X., Du S., Zeng C., Zhao W., Deng B., McComb D.W., et al. Biomimetic nanoparticles deliver mRNAs encoding costimulatory receptors and enhance T cell mediated cancer immunotherapy. Nat. Commun. 2021;12:7264.

     Not in our library10.1038/s41467-021-27434-x

184. 184.

     Billingsley M.M., Singh N., Ravikumar P., Zhang R., June C.H., Mitchell M.J. Ionizable Lipid Nanoparticle-Mediated mRNA Delivery for Human CAR T Cell Engineering. Nano Lett. 2020;20:1578-1589.

     Not in our library10.1021/acs.nanolett.9b04246

185. 185.

     Newick K., O’Brien S., Moon E., Albelda S.M. CAR T Cell Therapy for Solid Tumors. Annu. Rev. Med. 2017;68:139-152.

     Not in our library10.1146/annurev-med-062315-120245

186. 186.

     Haanen J.B. CT002—BNT211: A Phase I trial to evaluate safety and efficacy of CLDN6 CAR-T cells and CARVac-mediated in vivo expansion in patients with CLDN6-positive advanced solid tumors. Cancer Res. 2022;82((Suppl. 12)):CT002.

     Not in our library10.1158/1538-7445.am2022-ct002

187. 187.

     Johnson V. CAR T-Cell Therapy Plus Amplifying Vaccine Shows Initial Safety, Efficacy in Solid Tumors. 2021.

     Not in our library

188. 188.

     Auricchio A., Smith A.J., Ali R.R. The Future Looks Brighter After 25 Years of Retinal Gene Therapy. Hum. Gene Ther. 2017;28:982-987.

     Not in our library10.1089/hum.2017.164

189. 189.

     Russell S., Bennett J., Wellman J.A., Chung D.C., Yu Z.F., Tillman A., Wittes J., Pappas J., Elci O., McCague S., et al. Efficacy and safety of voretigene neparvovec (AAV2-hRPE65v2) in patients with RPE65-mediated inherited retinal dystrophy: A randomised, controlled, open-label, phase 3 trial. Lancet. 2017;390:849-860.

     Not in our library10.1016/s0140-6736(17)31868-8

190. 190.

     Russell D.W., Grompe M. Adeno-associated virus finds its disease. Nat. Genet. 2015;47:1104-1105.

     Not in our library10.1038/ng.3407

191. 191.

     Patel S., Ryals R.C., Weller K.K., Pennesi M.E., Sahay G. Lipid nanoparticles for delivery of messenger RNA to the back of the eye. J. Control. Release. 2019;303:91-100.

     Not in our library10.1016/j.jconrel.2019.04.015

192. 192.

     Xiong H., Liu S., Wei T., Cheng Q., Siegwart D.J. Theranostic dendrimer-based lipid nanoparticles containing PEGylated BODIPY dyes for tumor imaging and systemic mRNA delivery in vivo. J. Control. Release. 2020;325:198-205.

     Not in our library10.1016/j.jconrel.2020.06.030

193. 193.

     Nguyen J., Szoka F.C. Nucleic acid delivery: The missing pieces of the puzzle? Acc. Chem. Res. 2012;45:1153-1162.

     Not in our library10.1021/ar3000162

194. 194.

     Gilleron J., Querbes W., Zeigerer A., Borodovsky A., Marsico G., Schubert U., Manygoats K., Seifert S., Andree C., Stöter M., et al. Image-based analysis of lipid nanoparticle-mediated siRNA delivery, intracellular trafficking and endosomal escape. Nat. Biotechnol. 2013;31:638-646.

     Not in our library10.1038/nbt.2612

195. 195.

     Patel S., Kim J., Herrera M., Mukherjee A., Kabanov A.V., Sahay G. Brief update on endocytosis of nanomedicines. Adv. Drug Deliv. Rev. 2019;144:90-111.

     Not in our library10.1016/j.addr.2019.08.004

196. 196.

     Let’s Talk about Lipid Nanoparticles. Nat. Rev. Mater. 2021;6:99.

     Not in our library10.1038/s41578-021-00281-4

Cited by papers we hold 1

• AGILE platform: a deep learning powered approach to accelerate LNP development for mRNA delivery · 2024
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