What is suppression of anti-adeno-associated virus capsid T-cells achieving?

Human Gene Therapy

Volumn 25, Issue 3, 2014, Pages 178-179

  Flotte, Terence R ^a   Mueller, Christian ^a  

^a UNIVERSITY OF MASSACHUSETTS MEDICAL SCHOOL   (United States)

Author keywords

[No Author keywords available]

Indexed keywords

CORTICOSTEROID; EPITOPE; GAMMA INTERFERON; LIPOPROTEIN LIPASE; PARVOVIRUS VECTOR;

CELL INFILTRATION; ENZYME LINKED IMMUNOSPOT ASSAY; EXPRESSION VECTOR; GENE THERAPY; HUMAN; IMMUNE RESPONSE; IMMUNOSUPPRESSIVE TREATMENT; NOTE; REGULATORY T LYMPHOCYTE; SEROTYPE; T LYMPHOCYTE; TRANSGENE; VIRUS CAPSID;

DEPENDOVIRUS; GENETIC THERAPY; GENETIC VECTORS; HUMANS; HYPERLIPOPROTEINEMIA TYPE I;

EID: 84896325652     PISSN: 10430342     EISSN: 15577422     Source Type: Journal    
DOI: 10.1089/hum.2014.2506     Document Type: Note

References (6)

1. Brantly, M.L., Chulay, J.D., Wang, L., et al. (2009). Sustained transgene expression despite T lymphocyte responses in a clinical trial of rAAV1-AAT gene therapy. Proc. Natl. Acad. Sci. USA 106, 16363-16368.
2. Ferreira, V., Twisk, J., Kwikkers, K., et al. (2014). Immune responses to intramuscular administration of alipogene ti-parvovec (AAV1-LPLS447X) in a phase II clinical trial of lipoprotein lipase deficiency gene therapy. Hum. Gene Ther. 25, 180-188.
3. Flotte, T.R., Trapnell, B.C., Humphries, M., et al. (2011). Phase 2 clinical trial of a recombinant adeno-associated viral vector expressing alpha1-antitrypsin: interim results. Hum. Gene Ther. 22, 1239-1247.
4. 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.
5. Mueller, C., Chulay, J.D., Trapnell, B.C., et al. (2013). Human Treg responses allow sustained recombinant adeno-associated virus-mediated transgene expression. J. Clin. Invest. 123, 5310-5318.
6. Nathwani, A.C., Tuddenham, E.G., Rangarajan, S., et al. (2011). Adenovirus-associated virus vector-mediated gene transfer in hemophilia B. N. Engl. J. Med. 365, 2357-2365.