New adeno-associated virus strategies to support momentum in the clinic

Human Gene Therapy

Volumn 22, Issue 5, 2011, Pages 519-521

  Wright, J Fraser ^a  

^a UNIVERSITY OF PENNSYLVANIA   (United States)

Author keywords

[No Author keywords available]

Indexed keywords

ADENOVIRUS VECTOR; CAPSID PROTEIN; GLUCURONOSYLTRANSFERASE; MYCOPHENOLIC ACID; MYCOPHENOLIC ACID 2 MORPHOLINOETHYL ESTER;

CELL STRAIN HEK293; CYTOTOXIC T LYMPHOCYTE; GENE TARGETING; GENETIC TRANSDUCTION; GENETIC TRANSFECTION; HELPER VIRUS; HEMOPHILIA B; IMMUNE RESPONSE; IMMUNOMODULATION; INVERTED TERMINAL REPEAT; NONHUMAN; NOTE; PLASMID; POLYMERASE CHAIN REACTION; QUALITY CONTROL; SPECTROPHOTOMETRY; VIRAL CLEARANCE;

BIOTECHNOLOGY; DEPENDOVIRUS; GENE THERAPY; GENETIC VECTORS; SAFETY;

ADENO-ASSOCIATED VIRUS;

EID: 79955416671     PISSN: 10430342     EISSN: None     Source Type: Journal    
DOI: 10.1089/hum.2011.4080     Document Type: Note

References (11)

1. Allay, J.A., Sleep, S.E., Long, S., et al. (2011). Good manufacturing practice production of self-complementary serotype 8 adeno-associated viral vector for a hemophilia B clinical trial. Hum. Gene Ther. 22, 595-604.
2. Hauck, B., Murphy, S.L., Smith, P.H., et al. (2009). Undetectable transcription of cap in a clinical AAV vector: Implications for pre-formed capsid in immune responses. Mol. Ther. 17, 144-152.
3. Jayandharan, G.R., Zhong, L., Li, B., et al. (2008). Strategies for improving the transduction efficiency of single-stranded adeno-associated virus vectors in vitro and in vivo. Gene Ther. 15, 1287-1293.
4. Lu, H., Qu, G., Yang, X., et al. (2011). Systemic elimination of de novo capsid protein synthesis from replication-competent AAV contamination in the liver. Hum. Gene Ther. 22, 625-632.
5. Manno, C.S., Pierce, G.F., Arruda, V., 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)
6. Mingozzi, F., Hasbrouck, N.C., Basner-Tschakarjan, E., et al. (2007). Modulation of tolerance to the transgene product in a nonhuman primate model of AAV-mediated gene transfer to liver. Blood 110, 2334-2341. (Pubitemid 47523153)
7. Montenegro-Miranda, P., Ten Bloemendaal, L., Kunne, C., et al. (2011). Mycophenolate mofetil impairs transduction of single-stranded adeno-associated viral vectors. Hum. Gene Ther. 22, 605-612.
8. Nathwani, A., Tuddenham, E., Rosales, C., et al. (2010). Early clinical trial results following administration of a low dose of a novel self complementary adeno associated viral vector encoding human factor IX in two subjects with severe hemophilia B. Blood 116, 114.
9. Qiao, C., Wang, B., Zhu, X., et al. (2002). A novel gene expression control system and its use in stable, high-titer 293 cell-based ade-no-associated virus packaging cell lines. J. Virol. 76, 13015-13027. (Pubitemid 35386985)
10. Srivastava, A., Ma, W., Li, B., et al. (2011). A simple method to increase the transduction efficiency of single-stranded adeno-associated virus vectors in vitro and in vivo. Hum. Gene Ther. 22, 633-640.
11. Yuan, Z., Qiao, C., Hu, P., et al. (2011). A versatile adeno-associated virus vector producer cell line method for scalable vector production of different serotypes. Hum Gene Ther. 22, 613-624.