Little Vector, Big Gene Transduction: Fragmented Genome Reassembly of Adeno-associated Virus

Overview

Journal Mol Ther

Publisher Cell Press

Specialties Molecular Biology
Pharmacology

Date 2010 Jan 6

PMID 20048740

Citations 44

Authors

Matthew L Hirsch

Mavis Agbandje-Mckenna

R Jude Samulski

Affiliations

Soon will be listed here.

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References

Dong J, Wang D, van Ginkel F, Pascual D, Frizzell R . Systematic analysis of repeated gene delivery into animal lungs with a recombinant adenovirus vector. Hum Gene Ther. 1996; 7(3):319-31. DOI: 10.1089/hum.1996.7.3-319. View

Choi V, McCarty D, Samulski R . Host cell DNA repair pathways in adeno-associated viral genome processing. J Virol. 2006; 80(21):10346-56. PMC: 1641795. DOI: 10.1128/JVI.00841-06. View

Duan D, Yue Y, Engelhardt J . Expanding AAV packaging capacity with trans-splicing or overlapping vectors: a quantitative comparison. Mol Ther. 2001; 4(4):383-91. DOI: 10.1006/mthe.2001.0456. View

Lai Y, Yue Y, Duan D . Evidence for the failure of adeno-associated virus serotype 5 to package a viral genome > or = 8.2 kb. Mol Ther. 2009; 18(1):75-9. PMC: 2839223. DOI: 10.1038/mt.2009.256. View

Schultz B, Chamberlain J . Recombinant adeno-associated virus transduction and integration. Mol Ther. 2008; 16(7):1189-99. PMC: 2574934. DOI: 10.1038/mt.2008.103. View

McCarty D, Young Jr S, Samulski R . Integration of adeno-associated virus (AAV) and recombinant AAV vectors. Annu Rev Genet. 2004; 38:819-45. DOI: 10.1146/annurev.genet.37.110801.143717. View

Zhou X, Muzyczka N . In vitro packaging of adeno-associated virus DNA. J Virol. 1998; 72(4):3241-7. PMC: 109794. DOI: 10.1128/JVI.72.4.3241-3247.1998. View

Wu Z, Yang H, Colosi P . Effect of genome size on AAV vector packaging. Mol Ther. 2009; 18(1):80-6. PMC: 2839202. DOI: 10.1038/mt.2009.255. View

Arsuaga J, Tan R, Vazquez M, Sumners D, Harvey S . Investigation of viral DNA packaging using molecular mechanics models. Biophys Chem. 2002; 101-102:475-84. DOI: 10.1016/s0301-4622(02)00197-7. View

10.

Hirsch M, Storici F, Li C, Choi V, Samulski R . AAV recombineering with single strand oligonucleotides. PLoS One. 2009; 4(11):e7705. PMC: 2765622. DOI: 10.1371/journal.pone.0007705. View

11.

Maheshri N, Koerber J, Kaspar B, Schaffer D . Directed evolution of adeno-associated virus yields enhanced gene delivery vectors. Nat Biotechnol. 2006; 24(2):198-204. DOI: 10.1038/nbt1182. View

12.

Dong B, Nakai H, Xiao W . Characterization of genome integrity for oversized recombinant AAV vector. Mol Ther. 2009; 18(1):87-92. PMC: 2803017. DOI: 10.1038/mt.2009.258. View

13.

Wu Z, Asokan A, Samulski R . Adeno-associated virus serotypes: vector toolkit for human gene therapy. Mol Ther. 2006; 14(3):316-27. DOI: 10.1016/j.ymthe.2006.05.009. View

14.

Halbert C, Allen J, Miller A . Efficient mouse airway transduction following recombination between AAV vectors carrying parts of a larger gene. Nat Biotechnol. 2002; 20(7):697-701. DOI: 10.1038/nbt0702-697. View

15.

Li W, Asokan A, Wu Z, Van Dyke T, DiPrimio N, Johnson J . Engineering and Selection of Shuffled AAV Genomes: A New Strategy for Producing Targeted Biological Nanoparticles. Mol Ther. 2017; 16(7):1252-1260. DOI: 10.1038/mt.2008.100. View

16.

Inagaki K, Ma C, Storm T, Kay M, Nakai H . The role of DNA-PKcs and artemis in opening viral DNA hairpin termini in various tissues in mice. J Virol. 2007; 81(20):11304-21. PMC: 2045570. DOI: 10.1128/JVI.01225-07. View

17.

Hermonat P, Quirk J, Bishop B, Han L . The packaging capacity of adeno-associated virus (AAV) and the potential for wild-type-plus AAV gene therapy vectors. FEBS Lett. 1997; 407(1):78-84. DOI: 10.1016/s0014-5793(97)00311-6. View

18.

Grieger J, Samulski R . Packaging capacity of adeno-associated virus serotypes: impact of larger genomes on infectivity and postentry steps. J Virol. 2005; 79(15):9933-44. PMC: 1181570. DOI: 10.1128/JVI.79.15.9933-9944.2005. View

19.

King J, Dubielzig R, Grimm D, Kleinschmidt J . DNA helicase-mediated packaging of adeno-associated virus type 2 genomes into preformed capsids. EMBO J. 2001; 20(12):3282-91. PMC: 150213. DOI: 10.1093/emboj/20.12.3282. View

20.

Allocca M, Doria M, Petrillo M, Colella P, Garcia-Hoyos M, Gibbs D . Serotype-dependent packaging of large genes in adeno-associated viral vectors results in effective gene delivery in mice. J Clin Invest. 2008; 118(5):1955-64. PMC: 2298836. DOI: 10.1172/JCI34316. View