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Viral Vectors for Gene Therapy: Translational and Clinical Outlook
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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“For CRISPR/Cas9 delivery, however, host genome integrations could lead to unwanted off-target insertional mutagenesis and do not benefit genome surgery [46,47].”
From Viral Delivery Systems for CRISPR (2019) · cites this paper as [47]
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Cited by
Sorted by citations
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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
- Cited by
- 424
- References
- 33
- Language
- en
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