Nucleic acid-mediated intracellular protein delivery by lipid-like nanoparticles

Biomaterials

Volumn 35, Issue 24, 2014, Pages 6454-6461

  Eltoukhy, Ahmed A ^a,b   Chen, Delai ^b   Veiseh, Omid ^b   Pelet, Jeisa M ^b   Yin, Hao ^b   Dong, Yizhou ^b   Anderson, Daniel G ^a,b  

^a Massachusetts Institute of Technology   (United States)

^b MASSACHUSETTS INSTITUTE OF TECHNOLOGY   (United States)

Author keywords

Intracellular delivery; Lipid; Nanoparticles; Nucleic acid; Oligonucleotide; Protein delivery

Indexed keywords

BIOMOLECULES; CELL CULTURE; CELL PROLIFERATION; LIPIDS; NANOPARTICLES; NUCLEIC ACIDS; OLIGONUCLEOTIDES; PACKAGING MATERIALS;

ANTIGEN PRESENTING CELLS; HORSE-RADISH PEROXIDASE; INTRACELLULAR DELIVERY; INTRACELLULAR PROTEIN DELIVERY; MODEL PROTEINS; PROTEIN DELIVERY; PROTEIN ENTRAPMENT; THERAPEUTIC APPLICATION;

PROTEINS;

AVIDIN; CARBOCYANINE; CYANINE DYE 5; DNA; HORSERADISH PEROXIDASE; LIPID; NANOPARTICLE; NEUTRAVIDIN; NUCLEIC ACID; OLIGONUCLEOTIDE;

ANIMAL; BIOPHYSICS; C57BL MOUSE; CHEMISTRY; DRUG DELIVERY SYSTEM; FEMALE; HELA CELL LINE; HUMAN; IMMUNOHISTOCHEMISTRY; INTRACELLULAR SPACE; METABOLISM; NEAR INFRARED SPECTROSCOPY; POLYACRYLAMIDE GEL ELECTROPHORESIS; SPLEEN;

ANIMALS; AVIDIN; BIOPHYSICAL PHENOMENA; CARBOCYANINES; DNA; DRUG DELIVERY SYSTEMS; ELECTROPHORESIS, POLYACRYLAMIDE GEL; FEMALE; HELA CELLS; HORSERADISH PEROXIDASE; HUMANS; IMMUNOHISTOCHEMISTRY; INTRACELLULAR SPACE; LIPIDS; MICE, INBRED C57BL; NANOPARTICLES; NUCLEIC ACIDS; OLIGONUCLEOTIDES; SPECTROSCOPY, NEAR-INFRARED; SPLEEN;

EID: 84901447920     PISSN: 01429612     EISSN: 18785905     Source Type: Journal    
DOI: 10.1016/j.biomaterials.2014.04.014     Document Type: Article

References (39)

1. Leader B., Baca Q.J., Golan D.E. Protein therapeutics: a summary and pharmacological classification. Nat Rev Drug Discov 2008, 7:21-39.
2. Gu Z., Biswas A., Zhao M., Tang Y. Tailoring nanocarriers for intracellular protein delivery. Chem Soc Rev 2011, 40:3638-3655.
3. Torchilin V. Intracellular delivery of protein and peptide therapeutics. Drug Discov Today Technol 2008, 5:e95-e103.
4. Ford K.G., Souberbielle B.E., Darling D., Farzaneh F. Protein transduction: an alternative to genetic intervention?. Gene Ther 2001, 8:1-4.
5. Heitz F., Morris M.C., Divita G. Twenty years of cell-penetrating peptides: from molecular mechanisms to therapeutics. Br J Pharmacol 2009, 157:195-206.
6. Rapoport M., Lorberboum-Galski H. Tat-based drug delivery system - new directions in protein delivery for new hopes?. Expert Opin Drug Deliv 2009, 6:453-463.
7. Fawell S., Seery J., Daikh Y., Moore C., Chen L.L., Pepinsky B., et al. Tat-mediated delivery of heterologous proteins into cells. Proc Natl Acad Sci USA 1994, 91:664-668.
8. Schwarze S.R., Ho A., Vocero-Akbani A., Dowdy S.F. Invivo protein transduction: delivery of a biologically active protein into the mouse. Science 1999, 285:1569-1572.
9. Wender P.A., Mitchell D.J., Pattabiraman K., Pelkey E.T., Steinman L., Rothbard J.B. The design, synthesis, and evaluation of molecules that enable or enhance cellular uptake: peptoid molecular transporters. Proc Natl Acad Sci USA 2000, 97:13003-13008.
10. Futaki S., Suzuki T., Ohashi W., Yagami T., Tanaka S., Ueda K., et al. Arginine-rich peptides. An abundant source of membrane-permeable peptides having potential as carriers for intracellular protein delivery. JBiol Chem 2001, 276:5836-5840.
11. Derossi D., Joliot A.H., Chassaing G., Prochiantz A. The third helix of the antennapedia homeodomain translocates through biological membranes. JBiol Chem 1994, 269:10444-10450.
12. Morris M.C., Depollier J., Mery J., Heitz F., Divita G. Apeptide carrier for the delivery of biologically active proteins into mammalian cells. Nat Biotechnol 2001, 19:1173-1176.
13. Elliott G., O'Hare P. Intercellular trafficking and protein delivery by a herpesvirus structural protein. Cell 1997, 88:223-233.
14. Phelan A., Elliott G., O'Hare P. Intercellular delivery of functional p53 by the herpesvirus protein vp22. Nat Biotechnol 1998, 16:440-443.
15. Cronican J.J., Thompson D.B., Beier K.T., McNaughton B.R., Cepko C.L., Liu D.R. Potent delivery of functional proteins into mammalian cells invitro and invivo using a supercharged protein. ACS Chem Biol 2010, 5:747-752.
16. El-Sayed A., Futaki S., Harashima H. Delivery of macromolecules using arginine-rich cell-penetrating peptides: ways to overcome endosomal entrapment. AAPS J 2009, 11:13-22.
17. Patel L.N., Zaro J.L., Shen W.C. Cell penetrating peptides: intracellular pathways and pharmaceutical perspectives. Pharm Res 2007, 24:1977-1992.
18. Debs R.J., Freedman L.P., Edmunds S., Gaensler K.L., Duzgunes N., Yamamoto K.R. Regulation of gene expression invivo by liposome-mediated delivery of a purified transcription factor. JBiol Chem 1990, 265:10189-10192.
19. Zelphati O., Wang Y., Kitada S., Reed J.C., Felgner P.L., Corbeil J. Intracellular delivery of proteins with a new lipid-mediated delivery system. JBiol Chem 2001, 276:35103-35110.
20. Lee Y., Ishii T., Cabral H., Kim H.J., Seo J.H., Nishiyama N., et al. Charge-conversional polyionic complex micelles-efficient nanocarriers for protein delivery into cytoplasm. Angew Chem Int Ed Engl 2009, 48:5309-5312.
21. Weill C.O., Biri S., Adib A., Erbacher P. Apractical approach for intracellular protein delivery. Cytotechnology 2008, 56:41-48.
22. Tinsley J.H., Hawker J., Yuan Y. Efficient protein transfection of cultured coronary venular endothelial cells. Am J Physiol 1998, 275:H1873-H1878.
23. Futami J., Kitazoe M., Maeda T., Nukui E., Sakaguchi M., Kosaka J., et al. Intracellular delivery of proteins into mammalian living cells by polyethylenimine-cationization. JBiosci Bioeng 2005, 99:95-103.
24. Coue G., Engbersen J.F. Functionalized linear poly(amidoamine)s are efficient vectors for intracellular protein delivery. JControl Release 2011, 152:90-98.
25. Yan M., Du J., Gu Z., Liang M., Hu Y., Zhang W., et al. Anovel intracellular protein delivery platform based on single-protein nanocapsules. Nat Nanotechnol 2010, 5:48-53.
26. Mintzer M.A., Simanek E.E. Nonviral vectors for gene delivery. Chem Rev 2009, 109:259-302.
27. Love K.T., Mahon K.P., Levins C.G., Whitehead K.A., Querbes W., Dorkin J.R., et al. Lipid-like materials for low-dose, invivo gene silencing. Proc Natl Acad Sci USA 2010, 107:1864-1869.
28. Akinc A., Zumbuehl A., Goldberg M., Leshchiner E.S., Busini V., Hossain N., et al. Acombinatorial library of lipid-like materials for delivery of RNAi therapeutics. Nat Biotechnol 2008, 26:561-569.
29. Novobrantseva T.I., Borodovsky A., Wong J., Klebanov B., Zafari M., Yucius K., et al. Systemic RNAi-mediated gene silencing in nonhuman primate and rodent myeloid cells. Mol Ther Nucleic Acids 2012, 1:e4.
30. Duckworth B.P., Chen Y., Wollack J.W., Sham Y., Mueller J.D., Taton T.A., et al. Auniversal method for the preparation of covalent protein-DNA conjugates for use in creating protein nanostructures. Angew Chem Int Ed Engl 2007, 46:8819-8822.
31. Chen D., Love K.T., Chen Y., Eltoukhy A.A., Kastrup C., Sahay G., et al. Rapid discovery of potent siRNA-containing lipid nanoparticles enabled by controlled microfluidic formulation. JAm Chem Soc 2012, 134:6948-6951.
32. Lee M.J.-E., Veiseh O., Bhattarai N., Sun C., Hansen S.J., Ditzler S., et al. Rapid pharmacokinetic and biodistribution studies using cholorotoxin-conjugated iron oxide nanoparticles: a novel non-radioactive method. PLoS One 2010, 5:e9536.
33. Peek L.J., Middaugh C.R., Berkland C. Nanotechnology in vaccine delivery. Adv Drug Deliv Rev 2008, 60:915-928.
34. Singh M., Chakrapani A., O'Hagan D. Nanoparticles and microparticles as vaccine-delivery systems. Expert Rev Vaccines 2007, 6:797-808.
35. Klinman D.M. Cpg DNA as a vaccine adjuvant. Expert Rev Vaccines 2003, 2:305-315.
36. Chu R.S., Targoni O.S., Krieg A.M., Lehmann P.V., Harding C.V. Cpg oligodeoxynucleotides act as adjuvants that switch on t helper 1 (th1) immunity. JExp Med 1997, 186:1623-1631.
37. Weeratna R.D., McCluskie M.J., Xu Y., Davis H.L. Cpg DNA induces stronger immune responses with less toxicity than other adjuvants. Vaccine 2000, 18:1755-1762.
38. Datta S.K., Cho H.J., Takabayashi K., Horner A.A., Raz E. Antigen-immunostimulatory oligonucleotide conjugates: mechanisms and applications. Immunol Rev 2004, 199:217-226.
39. Wilson J.T., Keller S., Manganiello M.J., Cheng C., Lee C.C., Opara C., et al. Ph-responsive nanoparticle vaccines for dual-delivery of antigens and immunostimulatory oligonucleotides. ACS Nano 2013, 7:3912-3925.