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J Hum Genet. 2018 Feb;63(2):157-164. doi: 10.1038/s10038-017-0352-4. Epub 2017 Nov 13.

In vivo genome editing via the HITI method as a tool for gene therapy.

Author information

1
Institute for Advanced Co-Creation Studies, Osaka University, Suita, Japan. ksuzuki@chem.es.osaka-u.ac.jp.
2
Graduate School of Engineering Science, Osaka University, Toyonaka, Japan. ksuzuki@chem.es.osaka-u.ac.jp.
3
Gene Expression Laboratory, Salk Institute for Biological Studies, La Jolla, USA. ksuzuki@chem.es.osaka-u.ac.jp.
4
Gene Expression Laboratory, Salk Institute for Biological Studies, La Jolla, USA.

Abstract

Using genome-editing technologies to correct specific mutations represents a potentially transformative new approach for treating genetic disorders. Despite rapid advances in the field of genome editing, it is still unclear whether the long-standing goal of in vivo targeted transgene integration is feasible. This is primarily because current tools are inefficient. In particular, current technologies are incapable of targeted gene knock-in in non-dividing cells, the major building blocks of adult tissues. This poses a significant barrier for developing therapeutic strategies to treat a broad range of devastating genetic disorders. Recently, our group has developed a unique CRISPR/Cas9-based strategy, termed homology-independent targeted insertion (HITI), which enables targeted gene insertion in non-dividing cells, both in vitro and in vivo. This review will summarize current progress in developing this technology, and discuss the potential impact of HITI-based gene-correction therapies.

PMID:
29215090
DOI:
10.1038/s10038-017-0352-4
[Indexed for MEDLINE]

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