Expanding AAV packaging capacity with Trans-splicing or overlapping vectors: A quantitative comparison

Molecular Therapy

Volumn 4, Issue 4, 2001, Pages 383-391

  Duan, Dongsheng ^a   Yue, Yongping ^a   Engelhardt, John F ^a  

^a UNIVERSITY OF IOWA   (United States)

Author keywords

Circular intermediates; Gene therapy; Homologous recombination; Muscle; rAAV; Recombinant adeno associated virus; Splicing; Viral packaging

Indexed keywords

BETA GALACTOSIDASE; GENE PRODUCT; VIRUS DNA;

ADENO ASSOCIATED VIRUS; ANIMAL EXPERIMENT; ANIMAL MODEL; ANIMAL TISSUE; ARTICLE; COMPARATIVE STUDY; CONTROLLED STUDY; DNA PACKAGING; FIBROBLAST; GENE EXPRESSION; GENETIC CODE; HUMAN; HUMAN CELL; MOUSE; MUSCLE TISSUE; NONHUMAN; TRANS SPLICING; TRANSGENE; VIRUS RECOMBINANT; VIRUS RECOMBINATION;

ADENO-ASSOCIATED VIRUS; ANIMALIA; DNA VIRUSES; TRIXIS;

EID: 0034756451     PISSN: 15250016     EISSN: None     Source Type: Journal    
DOI: 10.1006/mthe.2001.0456     Document Type: Article

References (37)

1. Viral vectors for gene therapy: The art of turning infectious agents into vehicles of therapeutics
2. Evidence for gene transfer and expression of factor IX in haemophilia B patients treated with an AAV vector
3. Gene therapy restores vision in a canine model of childhood blindness
4. Structural analysis of adeno-associated virus transduction intermediates
5. Concatamerization of adeno-associated viral circular genomes occurs through intermolecular recombination
6. A new dual-vector approach to enhance recombinant adeno-associated virus-mediated gene expression through intermolecular cis activation
7. Trans-splicing vectors expand the utility of adeno-associated virus for gene therapy
8. Overcoming adeno-associated virus vector size limitation through viral DNA heterodimerization
9. Increasing the size of rAAV-mediated expression cassettes in vivo by intermolecular joining of two complementary vectors
10. Human gene targeting by viral vectors
11. Molecular cloning of adeno-associated virus variant genomes and generation of infectious virus by recombination in mammalian cells
12. Movement and resolution of Holliday junctions by enzymes from E coli
13. Expanding the AAV package
14. Regulation of adenovirus-mediated transgene expression by the viral E4 gene products: Requirement for E4 ORF3
15. Increased duration of transgene expression in the lung with plasmid DNA vectors harboring adenovirus E4 open reading frame 3
16. Trans-splicing vectors expand the packaging limits of adeno-associated virus for gene therapy applications
17. Transduction with recombinant adeno-associated virus for gene therapy is limited by leading-strand synthesis
18. Second-strand synthesis is a rate-limiting step for efficient transduction by recombinant adeno-associated virus vectors
19. Formation of adeno-associated virus circular genomes is differentially regulated by adenovirus E4 ORF6 and E2a gene expression
20. Two independent molecular pathways for recombinant adeno-associated virus genome conversion occur after UV-C and E4orf6 augmentation of transduction
21. Isolation and analysis of adenovirus type 5 mutants containing deletions in the gene encoding the DNA-binding protein
22. Redundant control of adenovirus late gene expression by early region 4
23. αVβ5 integrin: A co-receptor for adeno-associated virus type 2 infection
24. Human fibroblast growth factor receptor 1 is a co-receptor for infection by adeno-associated virus 2
25. Binding of adeno-associated virus type 5 to 2,3-linked sialic acid is required for gene transfer
26. Adeno-associated virus serotype 4 (aav4) and aav5 both require sialic acid binding for hemagglutination and efficient transduction but differ in sialic acid linkage specificity
27. Endosomal processing limits gene transfer to polarized airway epithelia by adeno-associated virus
28. Adeno-associated virus type 2-mediated gene transfer: Altered endocytic processing enhances transduction efficiency in murine fibroblasts
29. Recruitment of single-stranded recombinant adeno-associated virus vector genomes and intermolecular recombination are responsible for stable transduction of liver in vivo
30. Circular intermediates of recombinant adeno-associated virus have defined structural characteristics responsible for long term episomal persistence in muscle
31. Effect of DNA-dependent protein kinase on the molecular fate of the rAAV2 genome in skeletal muscle
32. Recruitment of wild-type and recombinant adeno-associated virus into adenovirus replication centers
33. Cellular and viral splicing factors can modify the splicing pattern of CFTR transcripts carrying splicing mutations
34. The adenovirus E4-ORF4 splicing enhancer protein interacts with a subset of phosphorylated SR proteins
35. Adeno-associated virus type 2-mediated gene transfer: Role of epidermal growth factor receptor protein tyrosine kinase in transgene expression
36. Production of high-titer recombinant adeno-associated virus vectors in the absence of helper adenovirus
37. Long-term gene expression and phenotypic correction using adeno-associated virus vectors in the mammalian brain