Journal
JOURNAL OF HUMAN GENETICS
Volume 63, Issue 2, Pages 157-164Publisher
NATURE PUBLISHING GROUP
DOI: 10.1038/s10038-017-0352-4
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Funding
- JSPS KAKENHI [15K21762]
- Leona M. and Harry B. Helmsley Charitable Trust [2012-PG-MED002]
- G. Harold and Leila Y. Mathers Charitable Foundation
- NIH [R01HL123755]
- McKnight Foundation
- Moxie Foundation
- Fundacion Dr. Pedro Guillen
- Universidad Catolica San Antonio de Murcia (UCAM)
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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.
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