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

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RR0102c01_Flotte.qxd Commentary Size does matter: overcoming the adeno-associated virus packaging limit Terence R Flotte University of Florida, Gainesville, Florida, USA Abstract 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. Keywords: adeno-associated virus, cystic fibrosis, gene therapy Received: 6 June 2000 Revisions requested: 19 June 2000 Revisions received: 20 June 2000 Accepted: 20 June 2000 Published: 5 July 2000 Respir Res 2000, 1:16–18 The electronic version of this article can be found online at © Current Science Ltd (Print ISSN 1465-9921; Online ISSN 1465-993X) CF = cystic fibrosis; CFTR = cystic fibrosis transmembrane conductance regulator; ITR = inverted terminal repeat; kb = kilobases; rAAV = recombi- nant adeno-associated virus. Recombinant adeno-associated virus (rAAV) vectors have some important advantages for gene therapy because they mediate stable transgene expression in terminally dif- ferentiated cells without inducing significant inflammatory toxicity [1–3]. For many years the use of rAAV was some- what limited by inefficient production methods, but this problem has recently been addressed by several groups [4–7], so tha

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