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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
PMID24840552
show ga
Clinical gene therapy has been increasingly successful owing both to an enhanced
molecular understanding of human disease and to progressively improving gene
delivery technologies. Among these technologies, delivery vectors based on
adeno-associated viruses (AAVs) have emerged as safe and effective and, in one
recent case, have led 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.