Gene editing technology as an approach to the treatment of liver diseases

Rajagopal N. Aravalli, Clifford J. Steer

Research output: Contribution to journalReview articlepeer-review

17 Scopus citations


Introduction: More than 100 liver-related disorders are caused by mutations in a single gene. These include both inherited and acquired genetic disorders. The development of nucleic acid-based therapies for liver diseases has been severely restricted due to many undesirable side effects and methodological limitations.Areas covered: In recent years, however, efforts have been intensified to address these issues, and to develop liver-targeted approaches using novel gene editing technologies, including ZFN, TALEN, CRISPR/Cas and PITCh. While each of these methods utilizes a distinct mechanism of gene modification at the genomic level, they all are dependent on an efficient delivery system to the target site within the host cell. This review will provide an overview on current and emerging therapeutic strategies for the treatment of liver diseases.Expert opinion: Clinical trials for liver gene therapy have entered an exciting stage and are already showing promise with the development of novel technologies and delivery options. A deeper understanding of off-target effects produced by gene editing approaches and immune responses generated in host cells by gene carriers is needed to fully realize their potential.

Original languageEnglish (US)
Pages (from-to)595-608
Number of pages14
JournalExpert opinion on biological therapy
Issue number5
StatePublished - May 3 2016


  • Gene editing
  • liver
  • non-viral vectors
  • viralvectors


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