Journal
EXPERT OPINION ON BIOLOGICAL THERAPY
Volume 11, Issue 3, Pages 315-327Publisher
INFORMA HEALTHCARE
DOI: 10.1517/14712598.2011.548799
Keywords
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Funding
- National Natural Science Foundation of China [30800093]
- Natural Science Foundation of Zhejiang Province in China [Y2090935]
- Erasmus Medical Center
- Liver Research Foundation (SLO) Rotterdam
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Areas covered: This review provides a summary of current literature on AAV-mediated gene therapies for both inherited and acquired liver diseases and outlines different strategies to overcome current clinical limitations. The unique properties of AAV over other viral vectors are highlighted as well as the current challenges which are faced for wide-ranging clinical application. Expert opinion: Despite the extensive positive results from animal models, successful application in clinical settings is hampered by immunological barriers. However, immune suppression and other strategies can be employed to overcome these limitations. Given some of their unique advantages, AAV vectors are currently the most obvious candidate for hepatic gene therapy applications, however, serotype-related issues of immune reactivity still represent a formidable barrier for clinical success.
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