Ontology highlight
ABSTRACT:
SUBMITTER: Kotterman MA
PROVIDER: S-EPMC4393649 | biostudies-other | 2014 Jul
REPOSITORIES: biostudies-other
Kotterman Melissa A MA Schaffer David V DV
Nature reviews. Genetics 20140520 7
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 approac ...[more]