Size does matter: overcoming the adeno-associated virus packaging limit

Respiratory Research

Terence R Flotte¹ 

[1] University of Florida, Gainesville, Florida, USA

关键词: gene therapy;    cystic fibrosis;    adeno-associated virus;   

Others  :  1227493
DOI  :  10.1186/rr6

 received in 2000-06-06, accepted in 2000-06-20,  发布年份 2000

【 摘 要 】

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.

【 授权许可】

   
2000 Current Science Ltd

【 图 表 】

Figure 1.

Figure 2.

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