Autoimmunity, recessive diseases, and gene replacement therapy

Molecular Therapy

Volumn 18, Issue 12, 2010, Pages 2045-2047

  Wilson, James M ^a  

^a UNIVERSITY OF PENNSYLVANIA   (United States)

Author keywords

[No Author keywords available]

Indexed keywords

DYSTROPHIN; PARVOVIRUS VECTOR;

AUTOIMMUNITY; DUCHENNE MUSCULAR DYSTROPHY; ENZYME LINKED IMMUNOSPOT ASSAY; GENE DELETION; GENE EXPRESSION; GENE REPLACEMENT THERAPY; HUMAN; IMMUNE RESPONSE; NONHUMAN; NOTE; T LYMPHOCYTE; T LYMPHOCYTE ACTIVATION; VIRAL GENE DELIVERY SYSTEM;

EID: 78650904638     PISSN: 15250016     EISSN: 15250024     Source Type: Journal    
DOI: 10.1038/mt.2010.253     Document Type: Note

References (8)

1. Mendell, JR, Campbell, K, Rodino-Klapac, L, Sahenk, Z, Shilling, C, Lewis, S et al. (2010). Dystrophin immunity in Duchenne's muscular dystrophy. N Engl J Med 363: 1429-1437.
2. Brantly, ML, Chulay, JD, Wang, L, Mueller, C, Humphries, M, Spencer, LT 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.
3. Manno, CS, Pierce, GF, Arruda, VR, Glader, B, Ragni, M, Rasko, JJ 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.
4. Gao, G, Wang, Q, Calcedo, R, Mays, L, Bell, P, Wang, L et al. (2009). Adeno-associated virus-mediated gene transfer to nonhuman primate liver can elicit destructive transgene-specifc T cell responses. Hum Gene Ther 20: 930-942.
5. Wang, Z, Kuhr, CS, Allen, JM, Blankinship, M, Gregorevic, P, Chamberlain, JS et al. (2007). Sustained AAV-mediated dystrophin expression in a canine model of Duchenne muscular dystrophy with a brief course of immunosuppression. Mol Ther 15: 1160-1166.
6. Lin, J, Zhi, Y, Mays, L and Wilson JM (2007). Vaccines based on novel adeno-associated virus vectors elicit aberrant CD8+ T-cell responses in mice. J Virol 81: 11840-11849.
7. Lin, SW, Hensley, SE, Tatsis, N, Lasaro, MO and Ertl, HC (2007). Recombinant adeno-associated virus vectors induce functionally impaired transgene product-specifc CD8+ T cells in mice. J Clin Invest 117: 3958-3970.
8. Gussoni, E, Pavlath, GK, Miller, RG, Panzara, MA, Powell M, Blau, HM et al. (1994). Specifc T cell receptor gene rearrangements at the site of muscle degeneration in Duchenne muscular dystrophy. J Immunol 153: 4798-4805.