DOI: 10.1038/sj.gt.3302134. Adeno-associated virus terminal repeat (TR) mutant generates self-complementary vectors to overcome the rate-limiting step to transduction in vivo

Adeno-associated virus terminal repeat (TR) mutant generates self-complementary vectors to overcome the rate-limiting step to transduction in vivo

10.1038/sj.gt.3302134

Crossref journal-article

Springer Science and Business Media LLC

Gene Therapy (297)

Bibliography

McCarty, D. M., Fu, H., Monahan, P. E., Toulson, C. E., Naik, P., & Samulski, R. J. (2003). Adeno-associated virus terminal repeat (TR) mutant generates self-complementary vectors to overcome the rate-limiting step to transduction in vivo. Gene Therapy, 10(26), 2112â2118.

Authors 6

D M McCarty (first)
H Fu (additional)
P E Monahan (additional)
C E Toulson (additional)
P Naik (additional)
R J Samulski (additional)

References 21 Referenced 450

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. (10.1038/sj.gt.3301514) / Gene Therapy by DM McCarty (2001)
Chapman MS, Rossmann MG . Single-stranded DNA–protein interactions in canine parvovirus. Structure 1995; 3: 151–162. (10.1016/S0969-2126(01)00146-0) / Structure by MS Chapman (1995)
King JA, Dubielzig R, Grimm D, Kleinschmidt JA . DNA helicase-mediated packaging of adeno-associated virus type 2 genomes into preformed capsids. EMBO J 2001; 20: 3282–3291. (10.1093/emboj/20.12.3282) / EMBO J by JA King (2001)
Ferrari FK, Samulski T, Shenk T, Samulski RJ . Second-strand synthesis is a rate limiting step for efficient transduction by recombinant adeno-associated virus vectors. J Virol 1996; 70: 3227–3234. (10.1128/jvi.70.5.3227-3234.1996) / J Virol by FK Ferrari (1996)
Fisher KJ et al. Transduction with recombinant adeno-associated virus for gene therapy is limited by leading-strand synthesis. J Virol 1996; 70: 520–532. (10.1128/jvi.70.1.520-532.1996) / J Virol by KJ Fisher (1996)
Nakai H, Storm TA, Kay MA . Recruitment of single-stranded recombinant adeno-associated virus vector genomes and intermolecular recombination are responsible for stable transduction of liver in vivo. J Virol 2000; 74: 9451–9463. (10.1128/JVI.74.20.9451-9463.2000) / J Virol by H Nakai (2000)
Snyder RO et al. Persistent and therapeutic concentrations of human factor IX in mice after hepatic gene transfer of recombinant AAV vectors. Nat Genet 1997; 16: 270–276. (10.1038/ng0797-270) / Nat Genet by RO Snyder (1997)
Miao CH et al. Nonrandom transduction of recombinant adeno-associated virus vectors in mouse hepatocytes in vivo: cell cycling does not influence hepatocyte transduction. J Virol 2000; 74: 3793–3803. (10.1128/JVI.74.8.3793-3803.2000) / J Virol by CH Miao (2000)
Yang GS et al. Virus-mediated transduction of murine retina with adeno-associated virus: effects of viral capsid and genome size. J Virol 2002; 76: 7651–7660. (10.1128/JVI.76.15.7651-7660.2002) / J Virol by GS Yang (2002)
Hirata RK, Russell DW . Design and packaging of adeno-associated virus gene targeting vectors. J Virol 2000; 74: 4612–4620. (10.1128/JVI.74.10.4612-4620.2000) / J Virol by RK Hirata (2000)
Dong JY, Fan PD, Frizzell RA . Quantitative analysis of the packaging capacity of recombinant adeno-associated virus. Hum Gene Ther 1996; 7: 2101–2112. (10.1089/hum.1996.7.17-2101) / Hum Gene Ther by JY Dong (1996)
Chen H et al. Gene transfer and expression in oligodendrocytes under the control of myelin basic protein transcriptional control region mediated by adeno-associated virus. Gene Therapy 1998; 5: 50–58. (10.1038/sj.gt.3300547) / Gene Therapy by H Chen (1998)
Chen H, McCarty DM, Bruce AT, Suzuki K . Oligodendrocyte-specific gene expression in mouse brain: use of a myelin-forming cell type-specific promoter in an adeno-associated virus. J Neurosci Res 1999; 55: 504–513. (10.1002/(SICI)1097-4547(19990215)55:4<504::AID-JNR10>3.0.CO;2-0) / J Neurosci Res by H Chen (1999)
Snyder RO et al. Features of the adeno-associated virus origin involved in substrate recognition by the viral Rep protein. J Virol 1993; 67: 6096–6104. (10.1128/jvi.67.10.6096-6104.1993) / J Virol by RO Snyder (1993)
Wang L et al. Sustained expression of therapeutic level of factor IX in hemophilia B dogs by AAV-mediated gene therapy in liver. Mol Ther 2000; 1: 154–158. (10.1006/mthe.2000.0031) / Mol Ther by L Wang (2000)
Li J, Samulski RJ, Xiao X . Role for highly regulated rep gene expression in adeno-associated virus vector production. J Virol 1997; 71: 5236–5243. (10.1128/jvi.71.7.5236-5243.1997) / J Virol by J Li (1997)
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)
Zolotukhin S et al. Recombinant adeno-associated virus purification using novel methods improves infectious titer and yield. Gene Therapy 1999; 6: 973. (10.1038/sj.gt.3300938) / Gene Therapy by S Zolotukhin (1999)
McCown TJ et al. Differential, persistent expression patterns of CNS gene transfer by an adeno-associated virus (AAV) vector. Brain Res 1996; 713: 99–107. (10.1016/0006-8993(95)01488-8) / Brain Res by TJ McCown (1996)
Fu H et al. Neurological correction of lysosomal storage in a mucopolysaccharidosis IIIB mouse model by adeno-associated virus-mediated gene delivery. Mol Ther 2002; 5: 42–49. (10.1006/mthe.2001.0514) / Mol Ther by H Fu (2002)
Franklin KBJ, Paxinos G . The Mouse Brain in Stereotaxic Coordinates. Acedemic Press: San Diego, 1997. / The Mouse Brain in Stereotaxic Coordinates by KBJ Franklin (1997)

Dates

Type	When
Created	21 years, 9 months ago (Nov. 17, 2003, 10:13 p.m.)
Deposited	3 years, 5 months ago (March 29, 2022, 10:47 p.m.)
Indexed	9 hours, 49 minutes ago (Aug. 31, 2025, 7:10 p.m.)
Issued	21 years, 9 months ago (Nov. 17, 2003)
Published	21 years, 9 months ago (Nov. 17, 2003)
Published Online	21 years, 9 months ago (Nov. 17, 2003)
Published Print	21 years, 9 months ago (Dec. 1, 2003)

Funders 0

None

BibTeX

@article{McCarty_2003, title={Adeno-associated virus terminal repeat (TR) mutant generates self-complementary vectors to overcome the rate-limiting step to transduction in vivo}, volume={10}, ISSN={1476-5462}, url={http://dx.doi.org/10.1038/sj.gt.3302134}, DOI={10.1038/sj.gt.3302134}, number={26}, journal={Gene Therapy}, publisher={Springer Science and Business Media LLC}, author={McCarty, D M and Fu, H and Monahan, P E and Toulson, C E and Naik, P and Samulski, R J}, year={2003}, month=nov, pages={2112–2118} }

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