Good manufacturing practice production of self-complementary serotype 8 adeno-associated viral vector for a hemophilia B clinical trial

Human Gene Therapy

Volumn 22, Issue 5, 2011, Pages 595-604

  Allay, James A ^a   Sleep, Susan ^a   Long, Scott ^a   Tillman, David M ^a   Clark, Rob ^a   Carney, Gael ^b   Fagone, Paolo ^b   McIntosh, Jenny H ^c   Nienhuis, Arthur W ^b   Davidoff, Andrew M ^b   Nathwani, Amit C ^c   Gray, John T ^b  

^a Children's GMP LLC   (United States)

^b ST JUDE CHILDREN S RESEARCH HOSPITAL   (United States)

^c UNIVERSITY COLLEGE LONDON   (United Kingdom)

Author keywords

[No Author keywords available]

Indexed keywords

BENZONASE; DODECYL SULFATE SODIUM; ENDONUCLEASE; HUMAN SERUM ALBUMIN; PARVOVIRUS VECTOR; PHOSPHATE BUFFERED SALINE; POLYACRYLAMIDE GEL; SEPHAROSE; UNCLASSIFIED DRUG;

ANION EXCHANGE CHROMATOGRAPHY; ARTICLE; COLUMN CHROMATOGRAPHY; CONTROLLED CLINICAL TRIAL; CONTROLLED STUDY; DOT HYBRIDIZATION; GEL FILTRATION; GENE TRANSFER; GENETIC TRANSFECTION; GOOD MANUFACTURING PRACTICE; HEMOPHILIA B; HUMAN; HUMAN CELL; NONHUMAN; NUCLEOTIDE SEQUENCE; PHASE 1 CLINICAL TRIAL; PHASE 2 CLINICAL TRIAL; POLYACRYLAMIDE GEL ELECTROPHORESIS; ROOM TEMPERATURE; SEROTYPE; ULTRAFILTRATION; ULTRAVIOLET SPECTROPHOTOMETRY; UNITED KINGDOM; UNITED STATES;

BIOTECHNOLOGY; CELL LINE; CHROMATOGRAPHY, GEL; CHROMATOGRAPHY, ION EXCHANGE; CLINICAL TRIALS AS TOPIC; DEPENDOVIRUS; GENE THERAPY; GENETIC VECTORS; HEMOPHILIA B; HUMANS; IMMUNOBLOTTING; SPECTROPHOTOMETRY;

ADENO-ASSOCIATED VIRUS;

EID: 79955405275     PISSN: 10430342     EISSN: None     Source Type: Journal    
DOI: 10.1089/hum.2010.202     Document Type: Article

References (52)

1. Ali, R.R., Sarra, G.M., Stephens, C., et al. (2000). Restoration of photoreceptor ultrastructure and function in retinal degeneration slow mice by gene therapy. Nat. Genet. 25, 306-310. (Pubitemid 30437318)
2. Allen, J.M., Debelak, D.J., Reynolds, T.C., and Miller, A.D. (1997). Identification and elimination of replication-competent adeno-associated virus (AAV) that can arise by nonhomolo-gous recombination during AAV vector production. J. Virol. 71, 6816-6822. (Pubitemid 27355342)
3. Ayuso, E., Mingozzi, F., Montane, J., et al. (2010). High AAV vector purity results in serotype- and tissue-independent enhancement of transduction efficiency. Gene Ther. 17, 503-510.
4. Bainbridge, J.W., Smith, A.J., Barker, S.S., et al. (2008). Effect of gene therapy on visual function in Leber's congenital amau-rosis. N. Engl. J. Med. 358, 2231-2239. (Pubitemid 351724452)
5. Brument, N, Morenweiser, R., Blouin, V., et al. (2002). A versatile and scalable two-step ion-exchange chromatography process for the purification of recombinant adeno-associated virus serotypes-2 and -5. Mol. Ther. 6, 678-686. (Pubitemid 35363701)
6. Chadeuf, G., Ciron, C, Moullier, P., and Salvetti, A. (2005). Evidence for encapsidation of prokaryotic sequences during recombinant adeno-associated virus production and their in vivo persistence after vector delivery. Mol. Ther. 12, 744-753. (Pubitemid 41350156)
7. Clement, N, Knop, D.R., and Byrne, B.J. (2009). Large-scale adeno-associated viral vector production using a herpesvirus-based system enables manufacturing for clinical studies. Hum. Gene Ther. 20, 796-806.
8. Davidoff, A.M., Ng, C.Y., Sleep, S., et al. (2004). Purification of recombinant adeno-associated virus type 8 vectors by ion exchange chromatography generates clinical grade vector stock. J. Virol. Methods 121, 209-215. (Pubitemid 39295711)
9. Davidoff, A.M., Gray, J.T., Ng, C.Y., et al. (2005). Comparison of the ability of adeno-associated viral vectors pseudotyped with serotype 2, 5, and 8 capsid proteins to mediate efficient transduction of the liver in murine and nonhuman primate models. Mol. Ther. 11, 875-888. (Pubitemid 40724106)
10. Duan, D., Yue, Y., Yan, Z., et al. (2000). Endosomal processing limits gene transfer to polarized airway epithelia by adeno-associated virus. J. Clin. Invest. 105, 1573-1587. (Pubitemid 30390308)
11. Fisher, K.J., Jooss, K., Alston, J., et al. (1997). Recombinant adeno-associated virus for muscle directed gene therapy. Nat. Med. 3, 306-312. (Pubitemid 27112928)
12. Flannery, J.G, Zolotukhin, S., Vaquero, M.I., et al. (1997). Efficient photoreceptor-targeted gene expression in vivo by recombinant adeno-associated virus. Proc. Natl. Acad. Sci. U.S.A. 94, 6916-6921. (Pubitemid 27281965)
13. Flotte, T., Carter, B., Conrad, C, et al. (1996). A phase I study of an adeno-associated virus-CFTR gene vector in adult CF patients with mild lung disease. Hum. Gene Ther. 7, 1145-1159. (Pubitemid 26202733)
14. 1-antitrypsin (rAAV2-CB-hAAT) gene vector to AAT-deficient adults Hum. Gene Ther. 15: 93-128 (Pubitemid 38147192)
15. Gao, G.P., Alvira, M.R, Wang, L., et al. (2002). Novel adeno-associated viruses from rhesus monkeys as vectors for human gene therapy. Proc. Natl. Acad. Sci. U.S.A. 99, 11854-11859. (Pubitemid 34994488)
16. Halbert, C.L., Rutledge, E.A., Allen, J.M., et al. (2000). Repeat transduction in the mouse lung by using adeno-associated virus vectors with different serotypes. J. Virol. 74, 1524-1532. (Pubitemid 30044103)
17. Hargrove, P.W., Vanin, E.F., Kurtzman, G.J., and Nienhuis, A.W. (1997). High-level globin gene expression mediated by a recombinant adeno-associated virus genome that contains the 3' gamma globin gene regulatory element and integrates as tandem copies in erythroid cells. Blood 89, 2167-2175. (Pubitemid 27132135)
18. Hauck, B., Murphy, S.L., Smith, P.H., et al. (2009). Undetectable transcription of cap in a clinical AAV vector: Implications for preformed capsid in immune responses. Mol. Ther. 17,144-152.
19. Herzog, R.W., Hagstrom, J.N., Kung, S.H., et al. (1997). Stable gene transfer and expression of human blood coagulation factor IX after intramuscular injection of recombinant adeno-associated virus. Proc. Natl. Acad. Sci. U.S.A. 94, 5804-5809. (Pubitemid 27241852)
20. Herzog, R.W., Yang, E.Y., Couto, L.B., et al. (1999). Long-term correction of canine hemophilia B by gene transfer of blood coagulation factor IX mediated by adeno-associated viral vector. Nat. Med. 5, 56-63. (Pubitemid 29050999)
21. Hurlbut, G.D., Ziegler, R.J., Nietupski, J.B., et al. (2010). Preexisting immunity and low expression in primates highlight translational challenges for liver-directed AAV8-mediated gene therapy. Mol. Ther. 18, 1983-1994.
22. Janson, C., McPhee, S., Bilaniuk, L., et al. (2002). Gene therapy of Canavan disease: AAV-2 vector for neurosurgical delivery of aspartoacylase gene (ASPA) to the human brain [clinical protocol]. Hum. Gene Ther. 13, 1391-1412. (Pubitemid 35034518)
23. Kaludov, N., Handelman, B., and Chiorini, J.A. (2002). Scalable purification of adeno-associated virus type 2, 4, or 5 using ion-exchange chromatography. Hum. Gene Ther. 13, 1235-1243. (Pubitemid 34746703)
24. Kaplitt, M.G., Leone, P., Samulski, R.J., et al. (1994). Long-term gene expression and phenotypic correction using adeno-associated virus vectors in the mammalian brain. Nat. Genet. 8, 148-154. (Pubitemid 24308362)
25. Maguire, A.M., Simonelli, F., Pierce, E.A., et al. (2008). Safety and efficacy of gene transfer for Leber's congenital amaurosis. N. Engl. J. Med. 358, 2240-2248. (Pubitemid 351724453)
26. Mandel, R.J., Spratt, S.K., Snyder, R.O., and Leff, S.E. (1997). Midbrain injection of recombinant adeno-associated virus encoding rat glial cell line-derived neurotrophic factor protects nigral neurons in a progressive 6-hydroxydopamine-induced degeneration model of Parkinson's disease in rats. Proc. Natl. Acad. Sci. U.S.A. 94, 14083-14088.
27. Manno, C.S., Chew, A.J., Hutchison, S., et al. (2003). AAV-me-diated factor IX gene transfer to skeletal muscle in patients with severe hemophilia B. Blood 101, 2963-2972. (Pubitemid 36857981)
28. Manno, C.S., Pierce, G.F., Arruda, V.R., et al. (2006). Successful transduction of liver in hemophilia by AAV-Factor IX and limitations imposed by the host immune response. Nat. Med. 12, 342-347. (Pubitemid 43355084)
29. McCarty, D.M., Monahan, P.E., and Samulski, R.J. (2001). Self-complementary recombinant adeno-associated virus (scAAV) vectors promote efficient transduction independently of DNA synthesis. Gene Ther. 8, 1248-1254. (Pubitemid 32780813)
30. McCarty, D.M., Fu, H., Monahan, P.E., et al. (2003). Adeno-associated virus terminal repeat (TR) mutant generates self-complementary vectors to overcome the rate-limiting step to transduction in vivo. Gene Ther. 10, 2112-2118. (Pubitemid 37536760)
31. Mount, J.D., Herzog, R.W., Tillson, D.M., et al. (2002). Sustained phenotypic correction of hemophilia B dogs with a factor IX null mutation by liver-directed gene therapy. Blood 99, 2670-2676. (Pubitemid 34525351)
32. Nakai, H., Yant, S.R., Storm, T.A., et al. (2001). Extra-chromosomal recombinant adeno-associated virus vector genomes are primarily responsible for stable liver transduction in vivo. J. Virol. 75, 6969-6976. (Pubitemid 32641584)
33. Nathwani, A.C., Davidoff, A., Hanawa, H., et al. (2001). Factors influencing in vivo transduction by recombinant adeno-associated viral vectors expressing the human factor IX cDNA. Blood 97, 1258-1265. (Pubitemid 32183747)
34. Nathwani, A.C., Davidoff, A.M., Hanawa, H., et al. (2002). Sustained high-level expression of human factor IX (hFIX) after liver-targeted delivery of recombinant adeno-associated virus encoding the hFIX gene in rhesus macaques. Blood 100, 1662-1669. (Pubitemid 34925143)
35. Nathwani, A.C., Gray, J.T., Ng, C.Y., et al. (2006). Self-complementary adeno-associated virus vectors containing a novel liver-specific human factor IX expression cassette enable highly efficient transduction of murine and nonhuman primate liver. Blood 107, 2653-2661.
36. Nathwani, A.C., Gray, J.T., McIntosh, J., et al. (2007). Safe and efficient transduction of the liver after peripheral vein infusion of self-complementary AAV vector results in stable therapeutic expression of human FIX in nonhuman primates. Blood 109, 1414-1421. (Pubitemid 46239572)
37. Nathwani, A.C., Tuddenham, E.G., Gray, J.T., et al. (2009). Oral presentations. J. Thromb. Haemost. 7, 34.
38. Okada, T., Nonaka-Sarukawa, M., Uchibori, R., et al. (2009). Scalable purification of adeno-associated virus serotype 1 (AAV1) and AAV8 vectors, using dual ion-exchange adsorp-tive membranes. Hum. Gene Ther. 20, 1013-1021.
39. Parks, W.P., Boucher, D.W., Melnick, J.L., et al. (1970). Ser-oepidemiological and ecological studies of the adenovirus-associated satellite viruses. Infect. Immun. 2, 716-722.
40. Rabinowitz, J.E., Rolling, F., Li, C., et al. (2002). Cross-packaging of a single adeno-associated virus (AAV) type 2 vector genome into multiple AAV serotypes enables transduction with broad specificity. J. Virol. 76, 791-801. (Pubitemid 34033377)
41. Snyder, R.O., Miao, C., Meuse, L., et al. (1999). Correction of hemophilia B in canine and murine models using recombinant adeno-associated viral vectors. Nat. Med. 5, 64-70. (Pubitemid 29051000)
42. Sommer, J.J., Smith, P.H., Parthasarathy, S., et al. (2003). Quantification of adeno-associated virus particles and empty cap-sids by optical density measurement. Mol. Ther. 7, 122-128. (Pubitemid 36305364)
43. Stedman, H., Wilson, J.M., Finke, R., et al. (2000). Phase I clinical trial utilizing gene therapy for limb girdle muscular dystrophy: a-, b-, g-, or d-sarcoglycan gene delivered with intramuscular instillations of adeno-associated vectors. Hum. Gene Ther. 11, 777-790. (Pubitemid 30180282)
44. Thomas, C.E., Storm, T.A., Huang, Z., and Kay, M.A. (2004). Rapid uncoating of vector genomes is the key to efficient liver transduction with pseudotyped adeno-associated virus vectors. J. Virol. 78, 3110-3122. (Pubitemid 38314369)
45. Vandenberghe, L.H., Wang, L., Somanathan, S., et al. (2006). Heparin binding directs activation of T cells against adeno-associated virus serotype 2 capsid. Nat. Med. 12, 967-971. (Pubitemid 44175151)
46. Virag, T., Cecchini, S., and Kotin, R.M. (2009). Producing re-combinant adeno-associated virus in foster cells: Overcoming production limitations using a baculovirus-insect cell expression strategy. Hum. Gene Ther. 20, 807-817.
47. Wagner, J.A., Reynolds, T., Moran, M.L., et al. (1998). Efficient and persistent gene transfer of AAV-CFTR in maxillary sinus. Lancet 351, 1702-1703. (Pubitemid 28254535)
48. Wagner, J.A., Messner, A.H., Moran, M.L., et al. (1999a). Safety and biological efficacy of an adeno-associated virus vector-cystic fibrosis transmembrane regulator (AAV-CFTR) in the cystic fibrosis maxillary sinus. Laryngoscope 109, 266-274. (Pubitemid 29075570)
49. Wagner, J.A., Nepomuceno, I.B., Shah, N., et al. (1999b). Maxillary sinusitis as a surrogate model for CF gene therapy clinical trials in patients with antrostomies. J. Gene Med. 1, 13-21. (Pubitemid 129631059)
50. Wang, L., Nichols, T.C., Read, M.S., et al. (2000). Sustained expression of therapeutic level of factor IX in hemophilia B dogs by AAV-mediated gene therapy in liver. Mol. Ther. 1, 154-158.
51. Wang, Z., Ma, H.I., Li, J., et al. (2003). Rapid and highly efficient transduction by double-stranded adeno-associated virus vectors in vitro and in vivo. Gene Ther. 10, 2105-2111. (Pubitemid 37536759)
52. Yuan, Z., Qiao, C., Hu, P., et al. (2011). A versatile AAV producer cell line method for scalable vector production of different serotypes. Hum. Gene Ther. (in press).