Engineering adeno-associated viruses for clinical gene therapy.
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.
Department of Chemical and Biomolecular Engineering, University of California, Berkeley, California 94720-3220, USA.
Melissa A
MA
Kotterman
1] Department of Chemical and Biomolecular Engineering, University of California, Berkeley, California 94720-3220, USA. [2] Department of Bioengineering, and the Department of Molecular and Cell Biology, University of California, Berkeley, California 94720-3220, USA.
David V
DV
Schaffer
D002213:Capsid / Q000737:chemistry / Q000378:metabolism; D002986:Clinical Trials as Topic; D004279:DNA, Viral / Q000235:genetics*; D000229:Dependovirus / Q000235:genetics*; D019020:Directed Molecular Evolution; D005818:Genetic Engineering; D015316:Genetic Therapy / Q000379:methods*; D005822:Genetic Vectors; D016679:Genome, Viral; D006467:Hemophilia A / Q000235:genetics / Q000473:pathology / Q000628:therapy; D006801:Humans; D008659:Metabolic Diseases / Q000235:genetics / Q000473:pathology / Q000628:therapy; D009136:Muscular Dystrophies / Q000235:genetics / Q000473:pathology / Q000628:therapy; D014764:Viral Proteins / Q000235:genetics*
D004279:DNA, Viral; D014764:Viral Proteins