AAV-mediated gene therapy for liver diseases: the prime candidate for clinical application?

Luc van der Laan, YG Wang, Hugo Tilanus, HLA Janssen, Qiuwei Pan

Research output: Contribution to journalArticleAcademicpeer-review

23 Citations (Scopus)


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.
Original languageUndefined/Unknown
Pages (from-to)315-327
Number of pages13
JournalExpert Opinion on Biological Therapy
Issue number3
Publication statusPublished - 2011

Research programs

  • EMC MM-03-47-02-A
  • EMC MM-04-20-02-A
  • EMC MM-04-47-07

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