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Viral Vectors for Gene Therapy: Translational and Clinical Outlook

Kotterman, Melissa A., Chalberg, Thomas W., Schaffer, David V.·2015·Annual Review of Biomedical Engineering
DOI 10.1146/annurev-bioeng-071813-104938
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Abstract

In a range of human trials, viral vectors have emerged as safe and effective delivery vehicles for clinical gene therapy, particularly for monogenic recessive disorders, but there has also been early work on some idiopathic diseases. These successes have been enabled by research and development efforts focusing on vectors that combine low genotoxicity and immunogenicity with highly efficient delivery, including vehicles based on adeno-associated virus and lentivirus, which are increasingly enabling clinical success. However, numerous delivery challenges must be overcome to extend this success to many diseases; these challenges include developing techniques to evade preexisting immunity, to ensure more efficient transduction of therapeutically relevant cell types, to target delivery, and to ensure genomic maintenance. Fortunately, vector-engineering efforts are demonstrating promise in the development of next-generation gene therapy vectors that can overcome these barriers. This review highlights key historical trends in clinical gene therapy, the recent clinical successes of viral-based gene therapy, and current research that may enable future clinical application.

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Metadata

Authors
Kotterman, M. A., Chalberg, T. W., & Schaffer, D. V.
Affiliations
  • 4D Molecular Therapeutics, San Francisco, California 94107;
  • Avalanche Biotechnologies, Inc., Menlo Park, California 94025;
Venue
Annual Review of Biomedical Engineering
Publisher
Annual Reviews
Published
2015-12-07
Volume
17
Issue
1
Pages
63-89
ISSN
  • 1523-9829
  • 1545-4274
DOI
10.1146/annurev-bioeng-071813-104938
Cited by
424
References
33
Language
en

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