Protein-based Vehicles for Biomimetic RNAi Delivery

Overview

Journal J Biol Eng

Publisher Biomed Central

Specialty Biomedical Engineering

Date 2019 Mar 21

PMID 30891095

Citations 5

Authors

Alex Eli Pottash

Christopher Kuffner

Madeleine Noonan-Shueh

Steven M Jay

Affiliations

Soon will be listed here.

Abstract

Broad translational success of RNA interference (RNAi) technology depends on the development of effective delivery approaches. To that end, researchers have developed a variety of strategies, including chemical modification of RNA, viral and non-viral transfection approaches, and incorporation with delivery vehicles such as polymer- and lipid-based nanoparticles, engineered and native proteins, extracellular vesicles (EVs), and others. Among these, EVs and protein-based vehicles stand out as biomimetically-inspired approaches, as both proteins (e.g. Apolipoprotein A-1, Argonaute 2, and Arc) and EVs mediate intercellular RNA transfer physiologically. Proteins specifically offer significant therapeutic potential due to their biophysical and biochemical properties as well as their ability to facilitate and tolerate manipulation; these characteristics have made proteins highly successful translational therapeutic molecules in the last two decades. This review covers engineered protein vehicles for RNAi delivery along with what is currently known about naturally-occurring extracellular RNA carriers towards uncovering design rules that will inform future engineering of protein-based vehicles.

Citing Articles

Structure of the human systemic RNAi defective transmembrane protein 1 (hSIDT1) reveals the conformational flexibility of its lipid binding domain.

Navratna V, Kumar A, Rana J, Mosalaganti S Life Sci Alliance. 2024; 7(9).

PMID: 38925866 PMC: 11208740. DOI: 10.26508/lsa.202402624.

Structure of the human systemic RNAi defective transmembrane protein 1 (hSIDT1) reveals the conformational flexibility of its lipid binding domain.

Navratna V, Kumar A, Rana J, Mosalaganti S bioRxiv. 2024; .

PMID: 38187772 PMC: 10769365. DOI: 10.1101/2023.12.21.572875.

Molecular physiology of Arc/Arg3.1: The oligomeric state hypothesis of synaptic plasticity.

Eriksen M, Bramham C Acta Physiol (Oxf). 2022; 236(3):e13886.

PMID: 36073248 PMC: 9787330. DOI: 10.1111/apha.13886.

Nanosized Particles Assembled by a Recombinant Virus Protein Are Able to Encapsulate Negatively Charged Molecules and Structured RNA.

Mani H, Chen Y, Chen Y, Liu W, Lo S, Lin S Polymers (Basel). 2021; 13(6).

PMID: 33799623 PMC: 7998283. DOI: 10.3390/polym13060858.

Reconfiguring Nature's Cholesterol Accepting Lipoproteins as Nanoparticle Platforms for Transport and Delivery of Therapeutic and Imaging Agents.

Chuang S, Cruz S, Narayanaswami V Nanomaterials (Basel). 2020; 10(5).

PMID: 32397159 PMC: 7279153. DOI: 10.3390/nano10050906.

References

Voinnet O, Pinto Y, Baulcombe D . Suppression of gene silencing: a general strategy used by diverse DNA and RNA viruses of plants. Proc Natl Acad Sci U S A. 1999; 96(24):14147-52. PMC: 24205. DOI: 10.1073/pnas.96.24.14147. View

Lee H, Pardridge W . Pharmacokinetics and delivery of tat and tat-protein conjugates to tissues in vivo. Bioconjug Chem. 2001; 12(6):995-9. DOI: 10.1021/bc0155061. View

Silhavy D, Molnar A, Lucioli A, Szittya G, Hornyik C, Tavazza M . A viral protein suppresses RNA silencing and binds silencing-generated, 21- to 25-nucleotide double-stranded RNAs. EMBO J. 2002; 21(12):3070-80. PMC: 125389. DOI: 10.1093/emboj/cdf312. View

Lacko A, Nair M, Paranjape S, Johnso S, McConathy W . High density lipoprotein complexes as delivery vehicles for anticancer drugs. Anticancer Res. 2002; 22(4):2045-9. View

Martinez J, Patkaniowska A, Urlaub H, Luhrmann R, Tuschl T . Single-stranded antisense siRNAs guide target RNA cleavage in RNAi. Cell. 2002; 110(5):563-74. DOI: 10.1016/s0092-8674(02)00908-x. View

Gu C, Rodriguez E, Reimert D, Shu T, Fritzsch B, Richards L . Neuropilin-1 conveys semaphorin and VEGF signaling during neural and cardiovascular development. Dev Cell. 2003; 5(1):45-57. PMC: 3918747. DOI: 10.1016/s1534-5807(03)00169-2. View

Ye K, Malinina L, Patel D . Recognition of small interfering RNA by a viral suppressor of RNA silencing. Nature. 2003; 426(6968):874-8. PMC: 4694583. DOI: 10.1038/nature02213. View

Vargason J, Szittya G, Burgyan J, Hall T . Size selective recognition of siRNA by an RNA silencing suppressor. Cell. 2003; 115(7):799-811. DOI: 10.1016/s0092-8674(03)00984-x. View

Takei Y, Kadomatsu K, Yuzawa Y, Matsuo S, Muramatsu T . A small interfering RNA targeting vascular endothelial growth factor as cancer therapeutics. Cancer Res. 2004; 64(10):3365-70. DOI: 10.1158/0008-5472.CAN-03-2682. View

10.

Hrzenjak A, Reicher H, Wintersperger A, Steinecker-Frohnwieser B, Sedlmayr P, Schmidt H . Inhibition of lung carcinoma cell growth by high density lipoprotein-associated alpha-tocopheryl-succinate. Cell Mol Life Sci. 2004; 61(12):1520-31. PMC: 11138836. DOI: 10.1007/s00018-004-4101-4. View

11.

Minakuchi Y, Takeshita F, Kosaka N, Sasaki H, Yamamoto Y, Kouno M . Atelocollagen-mediated synthetic small interfering RNA delivery for effective gene silencing in vitro and in vivo. Nucleic Acids Res. 2004; 32(13):e109. PMC: 506824. DOI: 10.1093/nar/gnh093. View

12.

Lorenz C, Hadwiger P, John M, Vornlocher H, Unverzagt C . Steroid and lipid conjugates of siRNAs to enhance cellular uptake and gene silencing in liver cells. Bioorg Med Chem Lett. 2004; 14(19):4975-7. DOI: 10.1016/j.bmcl.2004.07.018. View

13.

Bessis N, GarciaCozar F, Boissier M . Immune responses to gene therapy vectors: influence on vector function and effector mechanisms. Gene Ther. 2004; 11 Suppl 1:S10-7. DOI: 10.1038/sj.gt.3302364. View

14.

Rensen P, van Leeuwen S, Sliedregt L, Van Berkel T, Biessen E . Design and synthesis of novel N-acetylgalactosamine-terminated glycolipids for targeting of lipoproteins to the hepatic asialoglycoprotein receptor. J Med Chem. 2004; 47(23):5798-808. DOI: 10.1021/jm049481d. View

15.

Ayene I, Ford L, Koch C . Ku protein targeting by Ku70 small interfering RNA enhances human cancer cell response to topoisomerase II inhibitor and gamma radiation. Mol Cancer Ther. 2005; 4(4):529-36. DOI: 10.1158/1535-7163.MCT-04-0130. View

16.

Song E, Zhu P, Lee S, Chowdhury D, Kussman S, Dykxhoorn D . Antibody mediated in vivo delivery of small interfering RNAs via cell-surface receptors. Nat Biotechnol. 2005; 23(6):709-17. DOI: 10.1038/nbt1101. View

17.

Takeshita F, Minakuchi Y, Nagahara S, Honma K, Sasaki H, Hirai K . Efficient delivery of small interfering RNA to bone-metastatic tumors by using atelocollagen in vivo. Proc Natl Acad Sci U S A. 2005; 102(34):12177-82. PMC: 1183487. DOI: 10.1073/pnas.0501753102. View

18.

Baum C, Kustikova O, Modlich U, Li Z, Fehse B . Mutagenesis and oncogenesis by chromosomal insertion of gene transfer vectors. Hum Gene Ther. 2006; 17(3):253-63. DOI: 10.1089/hum.2006.17.253. View

19.

Zimmermann T, Lee A, Akinc A, Bramlage B, Bumcrot D, Fedoruk M . RNAi-mediated gene silencing in non-human primates. Nature. 2006; 441(7089):111-4. DOI: 10.1038/nature04688. View

20.

Grimm D, Streetz K, Jopling C, Storm T, Pandey K, Davis C . Fatality in mice due to oversaturation of cellular microRNA/short hairpin RNA pathways. Nature. 2006; 441(7092):537-41. DOI: 10.1038/nature04791. View