DOI: 10.1038/sj.gt.3301829. Adeno-associated virus vector-mediated gene transfer into dystrophin-deficient skeletal muscles evokes enhanced immune response against the transgene product

Adeno-associated virus vector-mediated gene transfer into dystrophin-deficient skeletal muscles evokes enhanced immune response against the transgene product

10.1038/sj.gt.3301829

Crossref journal-article

Springer Science and Business Media LLC

Gene Therapy (297)

Bibliography

Yuasa, K., Sakamoto, M., Miyagoe-Suzuki, Y., Tanouchi, A., Yamamoto, H., Li, J., Chamberlain, J. S., Xiao, X., & Takeda, S. (2002). Adeno-associated virus vector-mediated gene transfer into dystrophin-deficient skeletal muscles evokes enhanced immune response against the transgene product. Gene Therapy, 9(23), 1576â1588.

Authors 9

K Yuasa (first)
M Sakamoto (additional)
Y Miyagoe-Suzuki (additional)
A Tanouchi (additional)
H Yamamoto (additional)
J Li (additional)
J S Chamberlain (additional)
X Xiao (additional)
S Takeda (additional)

References 40 Referenced 104

Emery AEH . Duchenne Muscular Dystrophy 2nd edn Oxford University Press 1993 / Duchenne Muscular Dystrophy by AEH Emery (1993)
Rogot T et al. Efficient adenovirus-mediated transfer of a human minidystrophin gene to skeletal muscle of mdx mice Nature 1993 361: 647–650 (10.1038/361647a0) / Nature by T Rogot (1993)
Acsadi G et al. Dystrophin expression in muscles of mdx mice after adenovirus-mediated in vivo gene transfer Hum Gene Ther 1996 7: 129–140 (10.1089/hum.1996.7.2-129) / Hum Gene Ther by G Acsadi (1996)
Kochanek S et al. A new adenoviral vector: replacement of all viral coding sequences with 28 kb of DNA independently expressing both full-length dystrophin and β-galactosidase Proc Natl Acad Sci USA 1996 93: 5731–5736 (10.1073/pnas.93.12.5731) / Proc Natl Acad Sci USA by S Kochanek (1996)
Tuner G, Duncley MG, Dickson G . Gene therapy of Duchenne muscular dystrophy Brown SC, Lucy JA (eds); Dystrophin: Gene, Protein and Cell Biology Cambridge University Press 1997 pp 274–309
Chen H-H et al. DNA from both high-capacity and first generation adenoviral vectors remains intact in skeletal muscle Hum Gene Ther 1999 10: 365–373 (10.1089/10430349950018814) / Hum Gene Ther by H-H Chen (1999)
Acsadi G et al. Human dystrophin expression in mdx mice after intramuscular injection of DNA constructs Nature 1991 352: 815–818 (10.1038/352815a0) / Nature by G Acsadi (1991)
Gussoni E, Blau HM, Kunkel LM . The fate of individual myoblasts after transplantation into muscles of DMD patients Nature Med 1997 3: 970–977 (10.1038/nm0997-970) / Nature Med by E Gussoni (1997)
Gussoni E et al. Dystrophin expression in mdx mouse restored by stem cell transplantation Nature 1999 40: 390–394 / Nature by E Gussoni (1999)
Rando TA, Disatnik MH, Zhou LZ . Rescue of dystrophin expression in mdx mouse muscle by RNA/DNA oligonucleotides Proc Natl Acad Sci USA 2000 97: 5363–5368 (10.1073/pnas.97.10.5363) / Proc Natl Acad Sci USA by TA Rando (2000)
Bartlett RJ et al. In vivo targeted repair of a point mutation in the canine dystrophin gene by a chimeric RNA/DNA oligonucleotide Nat Biotechnol 2000 18: 615–622 (10.1038/76448) / Nat Biotechnol by RJ Bartlett (2000)
Dunckley MG et al. Modification of splicing in dystrophin gene in cultured Mdx muscle cells by antisense oligonucleotides Hum Mol Genet 1998 7: 1083–1090 (10.1093/hmg/7.7.1083) / Hum Mol Genet by MG Dunckley (1998)
Barton-Davis ER et al. Aminoglycoside antibiotics restore dystrophin function to skeletal muscles of mdx mice J Clin Invest 1999 104: 375–381 (10.1172/JCI7866) / J Clin Invest by ER Barton-Davis (1999)
Wagner KR et al. Gentamicin treatment of Duchenne and Becker muscular dystrophy due to nonsense mutations Ann Neurol 2001 49: 706–711 (10.1002/ana.1023) / Ann Neurol by KR Wagner (2001)
Yamamoto K et al. Immune response to adenovirus-delivered antigens upregulates utrophin and results in mitigation of muscle pathology in mdx mice Hum Gene Ther 2000 11: 669–680 (10.1089/10430340050015572) / Hum Gene Ther by K Yamamoto (2000)
Xiao X, Li J, Samulski RJ . Efficient long-term gene transfer into muscle tissue of immunocompetent mice by adeno-associated virus vector J Virol 1996 70: 8098–8108 (10.1128/jvi.70.11.8098-8108.1996) / J Virol by X Xiao (1996)
Kessler PD et al. Gene delivery to skeletal muscle results in sustained expression and systemic delivery of a therapeutic protein Proc Natl Acad Sci USA 1996 93: 14082–14087 (10.1073/pnas.93.24.14082) / Proc Natl Acad Sci USA by PD Kessler (1996)
Fisher KJ et al. Recombinant adeno-associated virus for muscle directed gene therapy Nat Med 1997 3: 306–312 (10.1038/nm0397-306) / Nat Med by KJ Fisher (1997)
Kaplitt MG et al. Long-term gene expression and phenotypic correction using adeno-associated virus vectors in mammalian brain Nat Genet 1994 8: 148–153 (10.1038/ng1094-148) / Nat Genet by MG Kaplitt (1994)
Jooss K, Yang Y, Fisher KJ, Wilson JM . Transduction of dendritic cells by DNA viral vectors directs the immune response to transgene products in muscle cells J Virol 1998 72: 4212–4223 (10.1128/JVI.72.5.4212-4223.1998) / J Virol by K Jooss (1998)
Greelish JP et al. Stable restoration of the sarcoglycan complex in dystrophic muscle perfused with histamine and a recombinant adeno-associated viral vector Nat Med 1999 5: 439–443 (10.1038/7439) / Nat Med by JP Greelish (1999)
Li J et al. rAAV vector-mediated sarcoglycan gene transfer in a hamster model for limb girdle muscular dystrophy Gene Therapy 1999 6: 74–82 (10.1038/sj.gt.3300830) / Gene Therapy by J Li (1999)
Cordier L et al. Rescue of skeletal muscles of γ-sarcoglycan-deficient mice with adeno-associated virus-mediated gene transfer Mol Ther 2000 1: 119–129 (10.1006/mthe.1999.0019) / Mol Ther by L Cordier (2000)
Cordier L et al. Muscle-specific promoters may be necessary for adeno-associated virus-mediated gene transfer in the treatment of muscular dystrophies Hum Gene Ther 2001 12: 205–215 (10.1089/104303401750061267) / Hum Gene Ther by L Cordier (2001)
England SB et al. Very mild muscular dystrophy associated with the deletion of 46% of dystrophin Nature 1990 343: 180–182 (10.1038/343180a0) / Nature by SB England (1990)
Phelps SF et al. Expression of full-length and truncated dystrophin mini-gene in transgenic mdx mice Hum Gene Ther 1995 4: 1251–1258 / Hum Gene Ther by SF Phelps (1995)
Yuasa K et al. Effective restoration of dystrophin-associated proteins in vivo by adenovirus-mediated transfer of truncated dystrophin cDNAs FEBS Lett 1998 425: 329–336 (10.1016/S0014-5793(98)00251-8) / FEBS Lett by K Yuasa (1998)
Wang B, Li J, Xiao X . Adeno-associated virus vector carrying human minidystrophin genes effectively ameliorates muscular dystrophy in mdx mouse model Proc Natl Acad Sci USA 2000 97: 13714–13719 (10.1073/pnas.240335297) / Proc Natl Acad Sci USA by B Wang (2000)
Straub V, Rafael JA, Chamberlain JS, Campbell KP . Animal models for muscular dystrophy show different patterns of sarcolemmal disruption J Cell Biol 1997 139: 375–385 (10.1083/jcb.139.2.375) / J Cell Biol by V Straub (1997)
Zhang Y, Chirmule N, Gao G-P, Wilson J . CD40 ligand-dependent activation of cytotoxic T lymphocytes by adeno-associated virus vectors in vivo: role of immature dendritic cells J Virol 2000 74: 8003–8010 (10.1128/JVI.74.17.8003-8010.2000) / J Virol by Y Zhang (2000)
Witmer-Pack MD et al. Tissue distribution of the DEC-205 protein that is detected by the monoclonal antibody NLDC-145 Cell Immunol 1995 163: 157–162 (10.1006/cimm.1995.1110) / Cell Immunol by MD Witmer-Pack (1995)
Khan MA . Corticosteroid therapy in Duchenne muscular dystrophy J Neurol Sci 1993 120: 8–14 (10.1016/0022-510X(93)90017-S) / J Neurol Sci by MA Khan (1993)
Pimorady-Esfahani A, Grounds MD, McMenamin PG . Macrophages and dendritic cells in normal and regenerating murine skeletal muscle Muscle Nerve 1997 20: 158–166 (10.1002/(SICI)1097-4598(199702)20:2<158::AID-MUS4>3.0.CO;2-B) / Muscle Nerve by A Pimorady-Esfahani (1997)
Hartigan-O'Connor D et al. Immune evasion by muscle-specific gene expression in dystrophic muscle Mol Ther 2001 4: 525–533 (10.1006/mthe.2001.0496) / Mol Ther by D Hartigan-O'Connor (2001)
Hohlfeld R, Engel AG . The immunobiology of muscle Immunol Today 1994 15: 269–274 (10.1016/0167-5699(94)90006-X) / Immunol Today by R Hohlfeld (1994)
Tinsley JM et al. Amelioration of the dystrophic phenotype of mdx mice using a truncated utrophin transgene Nature 1996 28: 349–353 (10.1038/384349a0) / Nature by JM Tinsley (1996)
Xiao X, Li J, Samulski RJ . Production of high-titer recombinant adeno-associated virus vectors in the absence of helper adenovirus J Virol 1998 72: 2224–2232 (10.1128/JVI.72.3.2224-2232.1998) / J Virol by X Xiao (1998)
Shield MA et al. E-box sites and a proximal regulatory region of the muscle creatine kinase gene differentially regulate expression in diverse skeletal muscles and cardiac muscle of transgenic mice Mol Cell Biol 1996 16: 5058–5068 (10.1128/MCB.16.9.5058) / Mol Cell Biol by MA Shield (1996)
Snyder R, Xiao X, Samulski RJ . Production of recombinant adeno-associated viral vectors Dracopoli N (eds); Current Protocols in Human Genetics John Wiley 1996 pp 1–2
Ishii A et al. Effective adenovirus-mediated gene expression in adult murine skeletal muscle Muscle Nerve 1999 22: 592–599 (10.1002/(SICI)1097-4598(199905)22:5<592::AID-MUS7>3.0.CO;2-2) / Muscle Nerve by A Ishii (1999)

Dates

Type	When
Created	22 years, 9 months ago (Nov. 11, 2002, 6:55 a.m.)
Deposited	3 years, 5 months ago (March 29, 2022, 10:40 p.m.)
Indexed	2 months, 2 weeks ago (June 19, 2025, 4:23 p.m.)
Issued	22 years, 9 months ago (Nov. 8, 2002)
Published	22 years, 9 months ago (Nov. 8, 2002)
Published Online	22 years, 9 months ago (Nov. 8, 2002)
Published Print	22 years, 9 months ago (Dec. 1, 2002)

Funders 0

None

BibTeX

@article{Yuasa_2002, title={Adeno-associated virus vector-mediated gene transfer into dystrophin-deficient skeletal muscles evokes enhanced immune response against the transgene product}, volume={9}, ISSN={1476-5462}, url={http://dx.doi.org/10.1038/sj.gt.3301829}, DOI={10.1038/sj.gt.3301829}, number={23}, journal={Gene Therapy}, publisher={Springer Science and Business Media LLC}, author={Yuasa, K and Sakamoto, M and Miyagoe-Suzuki, Y and Tanouchi, A and Yamamoto, H and Li, J and Chamberlain, J S and Xiao, X and Takeda, S}, year={2002}, month=nov, pages={1576–1588} }

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