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10.1038/nrg3742

http://scihub22266oqcxt.onion/10.1038/nrg3742
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C4393649!4393649!24840552
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suck abstract from ncbi


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pmid24840552      Nat+Rev+Genet 2014 ; 15 (7): 445-51
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  • Engineering adeno-associated viruses for clinical gene therapy #MMPMID24840552
  • Kotterman MA; Schaffer DV
  • Nat Rev Genet 2014[Jul]; 15 (7): 445-51 PMID24840552show ga
  • Clinical gene therapy has been increasingly successful, due both to an enhanced molecular understanding of human disease and to progressively improving gene delivery technologies. Among the latter, delivery vectors based on adeno-associated virus (AAV) have emerged as safe and effective ? in one recent case leading to regulatory approval. Although shortcomings in viral vector properties will render extension of such successes to many other human diseases challenging, new approaches to engineer and improve AAV vectors and their genetic cargo are increasingly helping to overcome these barriers.
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