Naturally enveloped AAV vectors for shielding neutralizing antibodies and robust gene delivery invivo

Biomaterials

Volumn 35, Issue 26, 2014, Pages 7598-7609

  György, Bence ^a   Fitzpatrick, Zachary ^a,b   Crommentuijn, Matheus H W ^a,c   Mu, Dakai ^a   Maguire, Casey A ^a  

^a MASSACHUSETTS GENERAL HOSPITAL   (United States)

^b LOUISIANA STATE UNIVERSITY   (United States)

^c VU UNIVERSITY MEDICAL CENTER   (Netherlands)

Author keywords

Adeno associated virus; Exosomes; Extracellular vesicles; Gene delivery; Gene therapy; Microvesicles

Indexed keywords

BACTERIOPHAGES; GENE THERAPY; GENE TRANSFER; MAMMALS; VECTORS;

ADENO-ASSOCIATED VIRUS; EXOSOMES; EXTRACELLULAR; GENE DELIVERY; MICROVESICLES;

ANTIBODIES;

NEUTRALIZING ANTIBODY;

ADENO ASSOCIATED VIRUS; ANIMAL CELL; ANIMAL EXPERIMENT; ANIMAL TISSUE; ARTICLE; BIOLUMINESCENCE; BRAIN; CONTROLLED STUDY; EX VIVO STUDY; GENE TARGETING; GENE TRANSFER; HUMAN; HUMAN CELL; IN VITRO STUDY; IN VIVO STUDY; MOUSE; NONHUMAN; PRIORITY JOURNAL; ANIMAL; CELL LINE; DEPENDOPARVOVIRUS; FEMALE; GENE THERAPY; GENE VECTOR; GENETIC TRANSDUCTION; GENETICS; IMMUNOLOGY; NUDE MOUSE;

ADENO-ASSOCIATED VIRUS; MUS;

ANIMALS; ANTIBODIES, NEUTRALIZING; CELL LINE; DEPENDOVIRUS; FEMALE; GENE TRANSFER TECHNIQUES; GENETIC THERAPY; GENETIC VECTORS; HUMANS; MICE; MICE, NUDE; TRANSDUCTION, GENETIC;

EID: 84902544740     PISSN: 01429612     EISSN: 18785905     Source Type: Journal    
DOI: 10.1016/j.biomaterials.2014.05.032     Document Type: Article

References (42)

1. Nathwani A.C., Tuddenham E.G., Rangarajan S., Rosales C., McIntosh J., Linch D.C., et al. Adenovirus-associated virus vector-mediated gene transfer in hemophilia B. NEngl J Med 2011, 365(25):2357-2365.
2. Maguire A.M., High K.A., Auricchio A., Wright J.F., Pierce E.A., Testa F., et al. Age-dependent effects of RPE65 gene therapy for Leber's congenital amaurosis: a phase 1 dose-escalation trial. Lancet 2009, 374(9701):1597-1605.
3. Haddley K. Alipogene tiparvovec for the treatment of lipoprotein lipase deficiency. Drugs Today (Barc) 2013, 49(3):161-170.
4. Muramatsu S., Fujimoto K., Kato S., Mizukami H., Asari S., Ikeguchi K., et al. Aphase I study of aromatic L-amino acid decarboxylase gene therapy for Parkinson's disease. Mol Ther 2010, 18(9):1731-1735.
5. Scallan C.D., Jiang H., Liu T., Patarroyo-White S., Sommer J.M., Zhou S., et al. Human immunoglobulin inhibits liver transduction by AAV vectors at low AAV2 neutralizing titers in SCID mice. Blood 2006, 107(5):1810-1817.
6. Louis Jeune V., Joergensen J.A., Hajjar R.J., Weber T. Pre-existing anti-adeno-associated virus antibodies as a challenge in AAV gene therapy. Hum Gene Ther Methods 2013, 24(2):59-67.
7. Mathivanan S., Fahner C.J., Reid G.E., Simpson R.J. ExoCarta 2012: database of exosomal proteins, RNA and lipids. Nucleic Acids Res 2012, 40(Database issue):D1241-D1244.
8. ELA S., Mager I., Breakefield X.O., Wood M.J. Extracellular vesicles: biology and emerging therapeutic opportunities. Nat Rev Drug Discov 2013, 12(5):347-357.
9. Feng Z., Hensley L., McKnight K.L., Hu F., Madden V., Ping L., et al. Apathogenic picornavirus acquires an envelope by hijacking cellular membranes. Nature 2013, 496(7445):367-371.
10. Ramakrishnaiah V., Thumann C., Fofana I., Habersetzer F., Pan Q., de Ruiter P.E., et al. Exosome-mediated transmission of hepatitis C virus between human hepatoma Huh7.5 cells. Proc Natl Acad Sci U S A 2013, 110(32):13109-13113.
11. Tian Y., Li S., Song J., Ji T., Zhu M., Anderson G.J., et al. Adoxorubicin delivery platform using engineered natural membrane vesicle exosomes for targeted tumor therapy. Biomaterials 2014, 35(7):2383-2390.
12. Zhang Y., Li L., Yu J., Zhu D., Zhang Y., Li X., et al. Microvesicle-mediated delivery of transforming growth factor beta1 siRNA for the suppression of tumor growth in mice. Biomaterials 2014, 35(14):4390-4400.
13. Maguire C.A., Balaj L., Sivaraman S., Crommentuijn M.H., Ericsson M., Mincheva-Nilsson L., et al. Microvesicle-associated AAV vector as a novel gene delivery system. Mol Ther 2012, 20(5):960-971.
14. Alvarez-Erviti L., Seow Y., Yin H., Betts C., Lakhal S., Wood M.J. Delivery of siRNA to the mouse brain by systemic injection of targeted exosomes. Nat Biotechnol 2011, 29(4):341-345.
15. Sun D., Zhuang X., Xiang X., Liu Y., Zhang S., Liu C., et al. Anovel nanoparticle drug delivery system: the anti-inflammatory activity of curcumin is enhanced when encapsulated in exosomes. Mol Ther 2010, 18(9):1606-1614.
16. Ohno S., Takanashi M., Sudo K., Ueda S., Ishikawa A., Matsuyama N., et al. Systemically injected exosomes targeted to EGFR deliver antitumor microRNA to breast cancer cells. Mol Ther 2013, 21(1):185-191.
17. Dai S., Wei D., Wu Z., Zhou X., Wei X., Huang H., et al. Phase I clinical trial of autologous ascites-derived exosomes combined with GM-CSF for colorectal cancer. Mol Ther 2008, 16(4):782-790.
18. Maguire C.A., Crommentuijn M.H., Mu D., Hudry E., Serrano-Pozo A., Hyman B.T., et al. Mouse gender influences brain transduction by intravascularly administered AAV9. Mol Ther 2013, 21(8):1470-1471.
19. Atai N.A., Balaj L., van Veen H., Breakefield X.O., Jarzyna P.A., Van Noorden C.J., et al. Heparin blocks transfer of extracellular vesicles between donor and recipient cells. JNeurooncol 2013, 115(3):343-351.
20. Christianson H.C., Svensson K.J., van Kuppevelt T.H., Li J.P., Belting M. Cancer cell exosomes depend on cell-surface heparan sulfate proteoglycans for their internalization and functional activity. Proc Natl Acad Sci U S A 2013, 110(43):17380-17385.
21. Bell C.L., Vandenberghe L.H., Bell P., Limberis M.P., Gao G.P., Van Vliet K., et al. The AAV9 receptor and its modification to improve invivo lung gene transfer in mice. JClin Invest 2011, 121(6):2427-2435.
22. Gyorgy B., Modos K., Pallinger E., Paloczi K., Pasztoi M., Misjak P., et al. Detection and isolation of cell-derived microparticles are compromised by protein complexes resulting from shared biophysical parameters. Blood 2011, 117(4):e39-e48.
23. Kumar P., Wu H., McBride J.L., Jung K.E., Kim M.H., Davidson B.L., et al. Transvascular delivery of small interfering RNA to the central nervous system. Nature 2007, 448(7149):39-43.
24. Tannous B.A., Grimm J., Perry K.F., Chen J.W., Weissleder R., Breakefield X.O. Metabolic biotinylation of cell surface receptors for invivo imaging. Nat Methods 2006, 3(5):391-396.
25. Gao Q., Liu Y.J., Guan Z.Z. Oxidative stress might be a mechanism connected with the decreased alpha 7 nicotinic receptor influenced by high-concentration of fluoride in SH-SY5Y neuroblastoma cells. Toxicol InVitro 2008, 22(4):837-843.
26. Murphy S.L., Li H., Zhou S., Schlachterman A., High K.A. Prolonged susceptibility to antibody-mediated neutralization for adeno-associated vectors targeted to the liver. Mol Ther 2008, 16(1):138-145.
27. Jiang H., Couto L.B., Patarroyo-White S., Liu T., Nagy D., Vargas J.A., et al. Effects of transient immunosuppression on adeno-associated, virus-mediated, liver-directed gene transfer in rhesus macaques and implications for human gene therapy. Blood 2006, 108(10):3321-3328.
28. Manno C.S., Pierce G.F., Arruda V.R., Glader B., Ragni M., Rasko J.J., et al. Successful transduction of liver in hemophilia by AAV-Factor IX and limitations imposed by the host immune response. Nat Med 2006, 12(3):342-347.
29. Lin J., Calcedo R., Vandenberghe L.H., Figueredo J.M., Wilson J.M. Impact of preexisting vector immunity on the efficacy of adeno-associated virus-based HIV-1 Gag vaccines. Hum Gene Ther 2008, 19(7):663-669.
30. Bar S., Daeffler L., Rommelaere J., Nuesch J.P. Vesicular egress of non-enveloped lytic parvoviruses depends on gelsolin functioning. PLoS Pathog 2008, 4(8):e1000126.
31. Bhattacharya B., Roy P. Role of lipids on entry and exit of bluetongue virus, a complex non-enveloped virus. Viruses 2010, 2(5):1218-1235.
32. Doria M., Ferrara A., Auricchio A. AAV2/8 vectors purified from culture medium with a simple and rapid protocol transduce murine liver, muscle, and retina efficiently. Hum Gene Ther Methods 2013, 24(6):392-398.
33. Manning W.C., Zhou S., Bland M.P., Escobedo J.A., Dwarki V. Transient immunosuppression allows transgene expression following readministration of adeno-associated viral vectors. Hum Gene Ther 1998, 9(4):477-485.
34. Mingozzi F., Anguela X.M., Pavani G., Chen Y., Davidson R.J., Hui D.J., et al. Overcoming preexisting humoral immunity to AAV using capsid decoys. Sci Transl Med 2013, 5(194):194ra92.
35. Chicoine L., Montgomery C., Bremer W., Shontz K., Griffin D., Heller K., et al. Plasmapheresis eliminates the negative impact of AAV antibodies on microdystrophin gene expression following vascular delivery. Mol Ther 2014, 22(2):338-347.
36. Mimuro J., Mizukami H., Hishikawa S., Ikemoto T., Ishiwata A., Sakata A., et al. Minimizing the inhibitory effect of neutralizing antibody for efficient gene expression in the liver with adeno-associated virus 8 vectors. Mol Ther 2013, 21(2):318-323.
37. Maheshri N., Koerber J.T., Kaspar B.K., Schaffer D.V. Directed evolution of adeno-associated virus yields enhanced gene delivery vectors. Nat Biotechnol 2006, 24(2):198-204.
38. Foust K.D., Nurre E., Montgomery C.L., Hernandez A., Chan C.M., Kaspar B.K. Intravascular AAV9 preferentially targets neonatal neurons and adult astrocytes. Nat Biotechnol 2009, 27(1):59-65.
39. Sallach J., Di Pasquale G., Larcher F., Niehoff N., Rubsam M., Huber A., et al. Tropism-modified AAV vectors overcome barriers to successful cutaneous therapy. Mol Ther 2014, 22(5):929-939.
40. Gray S.J., Blake B.L., Criswell H.E., Nicolson S.C., Samulski R.J., McCown T.J., et al. Directed evolution of a novel adeno-associated virus (AAV) vector that crosses the seizure-compromised blood-brain barrier (BBB). Mol Ther 2010, 18(3):570-578.
41. Grimm D., Lee J.S., Wang L., Desai T., Akache B., Storm T.A., et al. Invitro and invivo gene therapy vector evolution via multispecies interbreeding and retargeting of adeno-associated viruses. JVirol 2008, 82(12):5887-5911.
42. Kim S.S., Ye C., Kumar P., Chiu I., Subramanya S., Wu H., et al. Targeted delivery of siRNA to macrophages for anti-inflammatory treatment. Mol Ther 2010, 18(5):993-1001.