Serotype-independent method of recombinant adeno-associated virus (AAV) vector production and purification

Journal of Nippon Medical School

Volumn 79, Issue 6, 2012, Pages 394-402

  Miyake, Koichi ^a   Miyake, Noriko ^a   Yamazaki, Yoshiyuki ^a   Shimada, Takashi ^a   Hirai, Yukihiko ^a  

^a NIPPON MEDICAL SCHOOL   (Japan)

Author keywords

AAV vector; Purification; Self complementary AAV; Serotype

Indexed keywords

GREEN FLUORESCENT PROTEIN; IODIXANOL;

EXPRESSION VECTOR; FRACTIONATION; GEL PERMEATION CHROMATOGRAPHY; GENE EXPRESSION; GENETIC TRANSFECTION; HUMAN; IMMUNOHISTOCHEMISTRY; NONHUMAN; PARVOVIRUS VECTOR; PROTEIN PURIFICATION; REAL TIME POLYMERASE CHAIN REACTION; REVIEW; SEROTYPE; TARGET ORGAN; TRANSGENE; VIRAL GENE DELIVERY SYSTEM;

EID: 84871418860     PISSN: 13454676     EISSN: 13473409     Source Type: Journal    
DOI: 10.1272/jnms.79.394     Document Type: Review

References (16)

1. Nathwani AC, Tuddenham EG, Rangarajan S, et al.: Adenovirus-associated virus vector-mediated gene transfer in hemophilia B. The New England journal of medicine 2011; 365: 2357-2365.
2. Bowles DE, McPhee SW, Li C, et al.: Phase 1 gene therapy for Duchenne muscular dystrophy using a translational optimized AAV vector. Molecular therapy: the journal of the American Society of Gene Therapy 2012; 20: 443-455.
3. Flotte TR, Trapnell BC, Humphries M, et al.: Phase 2 clinical trial of a recombinant adeno-associated viral vector expressing alpha1-antitrypsin: interim results. Human gene therapy 2011; 22: 1239-1247.
4. Wright JF, Qu G, Tang C, Sommer JM: Recombinant adeno-associated virus: formulation challenges and strategies for a gene therapy vector. Current opinion in drug discovery & development 2003; 6: 174-178.
5. Gao GP, Alvira MR, Wang L, Calcedo R, Johnston J, Wilson JM: Novel adeno-associated viruses from rhesus monkeys as vectors for human gene therapy. Proc Natl Acad Sci U S A 2002; 99: 11854-11859.
6. Gao G, Vandenberghe LH, Wilson JM: New recombinant serotypes of AAV vectors. Current gene therapy 2005; 5: 285-297.
7. Schmidt M, Voutetakis A, Afione S, Zheng C, Mandikian D, Chiorini JA: Adeno-associated virus type 12 (AAV12): a novel AAV serotype with sialic acid-and heparan sulfate proteoglycan-independent transduction activity. Journal of virology 2008; 82: 1399-1406.
8. McCarty DM: Self-complementary AAV vectors; advances and applications. Molecular therapy: the journal of the American Society of Gene Therapy 2008; 16: 1648-1656.
9. McCarty DM, Monahan PE, Samulski RJ: Self-complementary recombinant adeno-associated virus (scAAV) vectors promote efficient transduction independently of DNA synthesis. Gene therapy 2001; 8: 1248-1254.
10. Wang L, Blouin V, Brument N, Bello-Roufai M, Francois A: Production and purification of recombinant adeno-associated vectors. Methods in molecular biology 2011; 807: 361-404.
11. Kotin RM: Large-scale recombinant adeno-associated virus production. Human molecular genetics 2011; 20: R2-6.
12. Lock M, Alvira M, Vandenberghe LH, et al.: Rapid, simple, and versatile manufacturing of recombinant adeno-associated viral vectors at scale. Human gene therapy 2010; 21: 1259-1271.
13. Guo P, El-Gohary Y, Prasadan K, et al.: Rapid and simplified purification of recombinant adeno-associated virus. Journal of virological methods 2012; 183: 139-146.
14. Reed SE, Staley EM, Mayginnes JP, Pintel DJ, Tullis GE: Transfection of mammalian cells using linear polyethylenimine is a simple and effective means of producing recombinant adeno-associated virus vectors. Journal of virological methods 2006; 138: 85-98.
15. Hermens WT, ter Brake O, Dijkhuizen PA, et al.: Purification of recombinant adeno-associated virus by iodixanol gradient ultracentrifugation allows rapid and reproducible preparation of vector stocks for gene transfer in the nervous system. Human gene therapy 1999; 10: 1885-1891.
16. Vandenberghe LH, Xiao R, Lock M, Lin J, Korn M, Wilson JM: Efficient serotype-dependent release of functional vector into the culture medium during adeno-associated virus manufacturing. Human gene therapy 2010; 21: 1251-1257.