Engineering humoral immunity as prophylaxis or therapy

Current Opinion in Immunology

Volumn 35, Issue , 2015, Pages 113-122

  Deal, Cailin E ^a   Balazs, Alejandro B ^a  

^a MASSACHUSETTS INSTITUTE OF TECHNOLOGY   (United States)

Author keywords

[No Author keywords available]

Indexed keywords

ANTINEOPLASTIC AGENT; CHEMOKINE RECEPTOR 4 MONOCLONAL ANTIBODY; COCAINE MONOCLONAL ANTIBODY; IMMUNOGLOBULIN G NEUTRALIZING ANTIBODY; LIPOPROTEIN LIPASE; MONOCLONAL ANTIBODY; NEUTRALIZING ANTIBODY; NICOTINE MONOCLONAL ANTIBODY; PALIVIZUMAB; SINGLE CHAIN FRAGMENT VARIABLE ANTIBODY; TRASTUZUMAB; UNCLASSIFIED DRUG; VIRUS VECTOR; VACCINE;

ALZHEIMER DISEASE; ANTIBODY ENGINEERING; ANTIBODY PRODUCTION; CLINICAL TRIAL (TOPIC); COCAINE DEPENDENCE; CUTANEOUS T CELL LYMPHOMA; DEGENERATIVE DISEASE; DRUG DESIGN; GENE EXPRESSION; HEPATITIS C; HUMAN; HUMAN IMMUNODEFICIENCY VIRUS INFECTION; HUMORAL IMMUNITY; HUNTINGTON CHOREA; IMMUNOGENICITY; IMMUNOREACTIVITY; IN VIVO STUDY; INFECTION PREVENTION; NEOPLASM; NONHUMAN; PASSIVE IMMUNIZATION; PROTEIN TARGETING; PROTEIN TRANSPORT; REVIEW; SUBSTANCE ABUSE; TOBACCO DEPENDENCE; VIRAL GENE DELIVERY SYSTEM; VIRAL GENE THERAPY; VIRUS INFECTION; ANIMAL; GENE VECTOR; GENETICS; IMMUNOLOGY; IMMUNOTHERAPY; INFECTION; METABOLISM; PROTEIN ENGINEERING; TRANSGENE;

ANIMALS; ANTIBODIES, NEUTRALIZING; GENETIC VECTORS; HUMANS; IMMUNITY, HUMORAL; IMMUNOTHERAPY; INFECTION; PROTEIN ENGINEERING; TRANSGENES; VACCINES;

EID: 84936935341     PISSN: 09527915     EISSN: 18790372     Source Type: Journal    
DOI: 10.1016/j.coi.2015.06.014     Document Type: Review

References (58)

1. Amanna I.J., Slifka M.K. Contributions of humoral and cellular immunity to vaccine-induced protection in humans. Virology 2011, 411:206-215.
2. Fenner F., Henderson D.A., Arita I., Jezek Z., Ladnyi I.D. Smallpox and Its Eradication 1988, World Health Organization, Geneva.
3. Roush S.W., Murphy T.V. Historical comparisons of morbidity and mortality for vaccine-preventable diseases in the United States. JAMA 2007, 298:2155-2163. Vaccine-Preventable Disease Table Working Group.
4. Plotkin S.A. Correlates of protection induced by vaccination. Clin Vaccine Immunol 2010, 17:1055-1065.
5. Chames P., Van Regenmortel M., Weiss E., Baty D. Therapeutic antibodies: successes, limitations and hopes for the future. Br J Pharmacol 2009, 157:220-233.
6. Köhler G., Milstein C. Continuous cultures of fused cells secreting antibody of predefined specificity. Nature 1975, 256:495-497.
7. Keizer R.J., Huitema A.D., Schellens J.H., Beijnen J.H. Clinical pharmacokinetics of therapeutic monoclonal antibodies. Clin Pharmacokinet 2010, 49:493-507.
8. Zalevsky J., Chamberlain A.K., Horton H.M., Karki S., Leung I.W.L., Sproule T.J., et al. Enhanced antibody half-life improves in vivo activity. Nat Biotechnol 2010, 28:157-159.
9. Pescovitz M.D. Rituximab, an anti-cd20 monoclonal antibody: history and mechanism of action. Am J Transplant 2006, 6:859-866.
10. Tjelle T.E., Corthay A., Lunde E., Sandlie I., Michaelsen T.E., Mathiesen I., et al. Monoclonal antibodies produced by muscle after plasmid injection and electroporation. Mol Ther 2004, 9:328-336.
11. Muthumani K., Flingai S., Wise M., Tingey C., Ugen K.E., Weiner D.B. Optimized and enhanced DNA plasmid vector based in vivo construction of a neutralizing anti-HIV-1 envelope glycoprotein Fab. Hum Vaccin Immunother 2013, 9:2253-2262.
12. Luo X.M., Maarschalk E., O'Connell R.M., Wang P., Yang L., Baltimore D. Engineering human hematopoietic stem/progenitor cells to produce a broadly neutralizing anti-HIV antibody after in vitro maturation to human B lymphocytes. Blood 2009, 113:1422-1431.
13. Joseph A., Zheng J.H., Chen K., Dutta M., Chen C., Stiegler G., et al. Inhibition of in vivo HIV infection in humanized mice by gene therapy of human hematopoietic stem cells with a lentiviral vector encoding a broadly neutralizing anti-HIV antibody. J Virol 2010, 84:6645-6653.
14. Skaricic D., Traube C., De B., Joh J., Boyer J., Crystal R.G., et al. Genetic delivery of an anti-RSV antibody to protect against pulmonary infection with RSV. Virology 2008, 378:79-85.
15. Tutykhina I.L., Sedova E.S., Gribova I.Y., Ivanova T.I., Vasilev L.A., Rutovskaya M.V., et al. Passive immunization with a recombinant adenovirus expressing an HA (H5)-specific single-domain antibody protects mice from lethal influenza infection. Antiviral Res 2013, 97:318-328.
16. De B.P., Hackett N.R., Crystal R.G., Boyer J.L. Rapid/sustained anti-anthrax passive immunity mediated by co-administration of Ad/AAV. Mol Ther 2008, 16:203-209.
17. WU Z., Asokan A., SAMULSKI R. Adeno-associated virus serotypes: vector toolkit for human gene therapy. Mol Ther 2006, 14:316-327.
18. Chao H., Liu Y., Rabinowitz J., Li C., Samulski R.J., Walsh C.E. Several log increase in therapeutic transgene delivery by distinct adeno-associated viral serotype vectors. Mol Ther 2000, 2:619-623.
19. Gao G.-P., Alvira M.R., Wang L., Calcedo R., Johnston J., Wilson J.M. Novel adeno-associated viruses from rhesus monkeys as vectors for human gene therapy. Proc Natl Acad Sci U S A 2002, 99:11854-11859.
20. Mays L.E., Vandenberghe L.H., Xiao R., Bell P., Nam H.-J., Agbandje-Mckenna M., et al. Adeno-associated virus capsid structure drives CD4-dependent CD8+ T cell response to vector encoded proteins. J Immunol 2009, 182:6051-6060.
21. Burnett J.R., Hooper A.J. Alipogene tiparvovec, an adeno-associated virus encoding the Ser(447)X variant of the human lipoprotein lipase gene for the treatment of patients with lipoprotein lipase deficiency. Curr Opin Mol Ther 2009, 11:681-691.
22. Nathwani A.C., Reiss U.M., Tuddenham E.G.D., Rosales C., Chowdary P., McIntosh J., et al. Long-term safety and efficacy of factor IX gene therapy in hemophilia B. N Engl J Med 2014, 371:1994-2004.
23. Kou J., Kim H., Pattanayak A., Song M., Lim J.-E., Taguchi H., et al. Anti-amyloid-β single-chain antibody brain delivery via AAV reduces amyloid load but may increase cerebral hemorrhages in an Alzheimer's disease mouse model. J Alzheimers Dis 2011, 27:23-38.
24. Wang Y.-J., Gao C.-Y., Yang M., Liu X.-H., Sun Y., Pollard A., et al. Intramuscular delivery of a single chain antibody gene prevents brain Aβ deposition and cognitive impairment in a mouse model of Alzheimer's disease. Brain Behav Immun 2010, 24:1281-1293.
25. Patel P., Kriz J., Gravel M., Soucy G., Bareil C., Gravel C., et al. Adeno-associated virus-mediated delivery of a recombinant single-chain antibody against misfolded superoxide dismutase for treatment of amyotrophic lateral sclerosis. Mol Ther 2014, 22:498-510.
26. Wuertzer C.A., Sullivan M.A., Qiu X., Federoff H.J. CNS delivery of vectored prion-specific single-chain antibodies delays disease onset. Mol Ther 2008, 16:481-486.
27. Fang J., Qian J.-J., Yi S., Harding T.C., Tu G.H., VanRoey M., et al. Stable antibody expression at therapeutic levels using the 2A peptide. Nat Biotechnol 2005, 23:584-590.
28. Fang J., Yi S., Simmons A., Tu G.H., Nguyen M., Harding T.C., et al. An antibody delivery system for regulated expression of therapeutic levels of monoclonal antibodies in vivo. Mol Ther 2007, 15:1153-1159.
29. Lewis A.D., Chen R., Montefiori D.C., Johnson P.R., Clark K.R. Generation of neutralizing activity against human immunodeficiency virus type 1 in serum by antibody gene transfer. J Virol 2002, 76:8769-8775.
30. Johnson P.R., Schnepp B.C., Zhang J., Connell M.J., Greene S.M., Yuste E., et al. Vector-mediated gene transfer engenders long-lived neutralizing activity and protection against SIV infection in monkeys. Nat Med 2009, 15:901-906.
31. Balazs A.B., Chen J., Hong C.M., Rao D.S., Yang L., Baltimore D. Antibody-based protection against HIV infection by vectored immunoprophylaxis. Nature 2011, 481:81-84.
32. Balazs A.B., Ouyang Y., Hong C.M., Chen J., Nguyen S.M., Rao D.S., et al. Vectored immunoprophylaxis protects humanized mice from mucosal HIV transmission. Nat Med 2014, 20:296-300.
33. Horwitz J.A., Halper-Stromberg A., Mouquet H., Gitlin A.D., Tretiakova A., Eisenreich T.R., et al. HIV-1 suppression and durable control by combining single broadly neutralizing antibodies and antiretroviral drugs in humanized mice. Proc Natl Acad Sci U S A 2013, 110:16538-16543.
34. Gardner M.R., Kattenhorn L.M., Kondur H.R., von Schaewen M., Dorfman T., Chiang J.J., et al. AAV-expressed eCD4-Ig provides durable protection from multiple SHIV challenges. Nature 2015, 519:87-91.
35. Deal C., Balazs A.B., Espinosa D.A., Zavala F., Baltimore D., Ketner G. Vectored antibody gene delivery protects against Plasmodium falciparum sporozoite challenge in mice. Proc Natl Acad Sci U S A 2014, 111:12528-12532.
36. de Jong Y.P., Dorner M., Mommersteeg M.C., Xiao J.W., Balazs A.B., Robbins J.B., et al. Broadly neutralizing antibodies abrogate established hepatitis C virus infection. Sci Transl Med 2014, 6:254ra129.
37. Balazs A.B., Bloom J.D., Hong C.M., Rao D.S., Baltimore D. Broad protection against influenza infection by vectored immunoprophylaxis in mice. Nat Biotechnol 2013, 31:647-652.
38. Limberis M.P., Adam V.S., Wong G., Gren J., Kobasa D., Ross T.M., et al. Intranasal antibody gene transfer in mice and ferrets elicits broad protection against pandemic influenza. Sci Transl Med 2013, 5:187ra72.
39. Zhou C., Emadi S., Sierks M.R., Messer A. A human single-chain Fv intrabody blocks aberrant cellular effects of overexpressed alpha-synuclein. Mol Ther 2004, 10:1023-1031.
40. Emadi S., Barkhordarian H., Wang M.S., Schulz P., Sierks M.R. Isolation of a human single chain antibody fragment against oligomeric alpha-synuclein that inhibits aggregation and prevents alpha-synuclein-induced toxicity. J Mol Biol 2007, 368:1132-1144.
41. Lecerf J.M., Shirley T.L., Zhu Q., Kazantsev A., Amersdorfer P., Housman D.E., et al. Human single-chain Fv intrabodies counteract in situ huntingtin aggregation in cellular models of Huntington's disease. Proc Natl Acad Sci U S A 2001, 98:4764-4769.
42. Messer A., Joshi S.N. Intrabodies as neuroprotective therapeutics. Neurotherapeutics 2013, 10:447-458.
43. Bowers W.J., Breakefield X.O., Sena-Esteves M. Genetic therapy for the nervous system. Hum Mol Genet 2011, 20:R28-R41.
44. Butler D.C., McLear J.A., Messer A. Engineered antibody therapies to counteract mutant huntingtin and related toxic intracellular proteins. Prog Neurobiol 2012, 97:190-204.
45. Southwell A.L., Ko J., Patterson P.H. Intrabody gene therapy ameliorates motor, cognitive, and neuropathological symptoms in multiple mouse models of Huntington's disease. J Neurosci 2009, 29:13589-13602.
46. Snyder-Keller A., McLear J.A., Hathorn T., Messer A. Early or late-stage anti-N-terminal Huntingtin intrabody gene therapy reduces pathological features in B6. HDR6/1 mice. J Neuropathol Exp Neurol 2010, 69:1078.
47. DeMattos R.B., Bales K.R., Cummins D.J., Dodart J.C., Paul S.M., Holtzman D.M. Peripheral anti-A beta antibody alters CNS and plasma A beta clearance and decreases brain A beta burden in a mouse model of Alzheimer's disease. Proc Natl Acad Sci U S A 2001, 98:8850-8855.
48. Ryan D.A., Mastrangelo M.A., Narrow W.C., Sullivan M.A., Federoff H.J., Bowers W.J. Abeta-directed single-chain antibody delivery via a serotype-1 AAV vector improves learning behavior and pathology in Alzheimer's disease mice. Mol Ther 2010, 18:1471-1481.
49. Wang Y.-J., Pollard A., Zhong J.-H., Dong X.-Y., Wu X.-B., Zhou H.-D., et al. Intramuscular delivery of a single chain antibody gene reduces brain Abeta burden in a mouse model of Alzheimer's disease. Neurobiol Aging 2009, 30:364-376.
50. Shimada M., Abe S., Takahashi T., Shiozaki K., Okuda M., Mizukami H., et al. Prophylaxis and treatment of Alzheimer's disease by delivery of an adeno-associated virus encoding a monoclonal antibody targeting the amyloid Beta protein. PLoS ONE 2013, 8:e57606.
51. Lyons T.J., Liu H., Goto J.J., Nersissian A., Roe J.A., Graden J.A., et al. Mutations in copper-zinc superoxide dismutase that cause amyotrophic lateral sclerosis alter the zinc binding site and the redox behavior of the protein. Proc Natl Acad Sci U S A 1996, 93:12240-12244.
52. Moda F., Vimercati C., Campagnani I., Ruggerone M., Giaccone G., Morbin M., et al. Brain delivery of AAV9 expressing an anti-PrP monovalent antibody delays prion disease in mice. Prion 2012, 6:383-390.
53. Leyland-Jones B., Arnold A., Gelmon K., Verma S., Ayoub J.P., Seidman A., et al. Pharmacologic insights into the future of trastuzumab. Ann Oncol 2001, 12(Suppl 1):S43-S47.
54. Wang G., Qiu J., Wang R., Krause A., Boyer J.L., Hackett N.R., et al. Persistent expression of biologically active anti-HER2 antibody by AAVrh.10-mediated gene transfer. Cancer Gene Ther 2010, 17:559-570.
55. Ishida T., Ueda R. CCR4 as a novel molecular target for immunotherapy of cancer. Cancer Sci 2006, 97:1139-1146.
56. Han T., Abdel-Motal U.M., Chang D.-K., Sui J., Muvaffak A., Campbell J., et al. Human anti-CCR4 minibody gene transfer for the treatment of cutaneous T-cell lymphoma. PLoS ONE 2012, 7:e44455.
57. Hicks M.J., Rosenberg J.B., De B.P., Pagovich O.E., Young C.N., Qiu J.-P., et al. AAV-directed persistent expression of a gene encoding anti-nicotine antibody for smoking cessation. Sci Transl Med 2012, 4:140ra87.
58. Rosenberg J.B., Hicks M.J., De B.P., Pagovich O., Frenk E., Janda K.D., et al. AAVrh.10-mediated expression of an anti-cocaine antibody mediates persistent passive immunization that suppresses cocaine-induced behavior. Hum Gene Ther 2012, 23:451-459.