Size does matter: overcoming the adeno-associated virus packaging limit
AUTOR(ES)
Flotte, Terence R
FONTE
BioMed Central
RESUMO
Recombinant adeno-associated virus (rAAV) vectors mediate long-term gene transfer without any known toxicity. The primary limitation of rAAV has been the small size of the virion (20 nm), which only permits the packaging of 4.7 kilobases (kb) of exogenous DNA, including the promoter, the polyadenylation signal and any other enhancer elements that might be desired. Two recent reports (D Duan et al: Nat Med 2000, 6:595-598; Z Yan et al: Proc Natl Acad Sci USA 2000, 97:6716-6721) have exploited a unique feature of rAAV genomes, their ability to link together in doublets or strings, to bypass this size limitation. This technology could improve the chances for successful gene therapy of diseases like cystic fibrosis or Duchenne muscular dystrophy that lead to significant pulmonary morbidity.
ACESSO AO ARTIGO
http://www.pubmedcentral.nih.gov/articlerender.fcgi?artid=59536Documentos Relacionados
- In Vitro Packaging of Adeno-Associated Virus DNA
- Infectious Entry Pathway of Adeno-Associated Virus and Adeno-Associated Virus Vectors
- Design and Packaging of Adeno-Associated Virus Gene Targeting Vectors
- Genetics of adeno-associated virus: isolation and preliminary characterization of adeno-associated virus type 2 mutants.
- A cis-Acting Element That Directs Circular Adeno-Associated Virus Replication and Packaging