Cited by 549Hemophilia Treatment and ResearchCRISPR and Genetic EngineeringVirus-based gene therapy research

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In vivo genome editing restores haemostasis in a mouse model of haemophilia

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Li, Hojun, Haurigot, Virginia, Doyon, Yannick, Li, Tianjian, Wong, Sunnie Y., Bhagwat, Anand S., Malani, Nirav, Anguela, Xavier M., Sharma, Rajiv, Ivanciu, Lacramiora, Murphy, Samuel L., Finn, Jonathan D., Khazi, Fayaz R., Zhou, Shangzhen, Paschon, David E., Rebar, Edward J., Bushman, Frederic D., Gregory, Philip D., Holmes, Michael C., High, Katherine A.·2011·Nature

DOI 10.1038/nature10177PMC PMC3152293

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Abstract

Editing of the human genome to correct disease-causing mutations is a promising approach for the treatment of genetic disorders. Genome editing improves on simple gene-replacement strategies by effecting in situ correction of a mutant gene, thus restoring normal gene function under the control of endogenous regulatory elements and reducing risks associated with random insertion into the genome. Gene-specific targeting has historically been limited to mouse embryonic stem cells. The development of zinc finger nucleases (ZFNs) has permitted efficient genome editing in transformed and primary cells that were previously thought to be intractable to such genetic manipulation. In vitro, ZFNs have been shown to promote efficient genome editing via homology-directed repair by inducing a site-specific double-strand break (DSB) at a target locus, but it is unclear whether ZFNs can induce DSBs and stimulate genome editing at a clinically meaningful level in vivo. Here we show that ZFNs are able to induce DSBs efficiently when delivered directly to mouse liver and that, when co-delivered with an appropriately designed gene-targeting vector, they can stimulate gene replacement through both homology-directed and homology-independent targeted gene insertion at the ZFN-specified locus. The level of gene targeting achieved was sufficient to correct the prolonged clotting times in a mouse model of haemophilia B, and remained persistent after induced liver regeneration. Thus, ZFN-driven gene correction can be achieved in vivo, raising the possibility of genome editing as a viable strategy for the treatment of genetic disease.

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Self-citation
Identification of Novel Mutations Controlling Cerebral Cortex Malformations Caused by ENU-Induced Mutagenesis in the Mouse
Borisova, E. V., Epifanova, E. A., Tutukova, S., Belousova, I. I., Zhidkova, N., Rusanova, A. M., Salina, V. A., Turovsky, E. A., Turovskaya, M. V., Tarabykin, V. S., et al. (2018) · Современные технологии в медицине · doi:10.17691/stm2018.10.3.8 · Cited by 5
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Authors: Li, H., Haurigot, V., Doyon, Y., Li, T., Wong, S. Y., Bhagwat, A. S., Malani, N., Anguela, X. M., Sharma, R., Ivanciu, L., Murphy, S. L., Finn, J. D., Khazi, F. R., Zhou, S., Paschon, D. E., Rebar, E. J., Bushman, F. D., Gregory, P. D., Holmes, M. C., & High, K. A.
Affiliations: Children's Hospital of Philadelphia (US)
Sangamo BioSciences (United States) (US)
University of Pennsylvania (US)
Corresponding author: Holmes, Michael C.
High, Katherine A.
ORCID: Li, Hojun
Haurigot, Virginia
Doyon, Yannick
Wong, Sunnie Y.
Bhagwat, Anand S.
Malani, Nirav
Anguela, Xavier M.
Sharma, Rajiv
Finn, Jonathan D.
Rebar, Edward J.
Bushman, Frederic D.
Gregory, Philip D.
Holmes, Michael C.
High, Katherine A.
Venue: Nature
Publisher: Springer Science and Business Media LLC
Published: 2011-06-26
Volume: 475
Issue: 7355
Pages: 217-221
ISSN: 0028-0836 (ISSN-L)
1476-4687
DOI: 10.1038/nature10177
PMC: PMC3152293
OpenAlex: W2038866304
Cited by: 548
References: 29
Citations by year: 2026: 6 · 2025: 10 · 2024: 12 · 2023: 13 · 2022: 22 · 2021: 19 · 2020: 34 · 2019: 33 · 2018: 48 · 2017: 54 · 2016: 66 · 2015: 62 · 2014: 66 · 2013: 44 · 2012: 40
FWCI: 35.43
Type: journal article
Language: en
Open access: green
www.ncbi.nlm.nih.gov
Licence: www.springer.com/tdm (tdm)
Locations: Nature (publishedVersion)
PubMed (publishedVersion)
Europe PMC (PubMed Central) (acceptedVersion)
PubMed Central (acceptedVersion)
RePEc: Research Papers in Economics (submittedVersion)
Funders: Howard Hughes Medical Institute
National Heart, Lung, and Blood Institute (P01HL064190, T32HL007150)
Topics: Health Sciences › Medicine › Hematology › Hemophilia Treatment and Research
Life Sciences › Biochemistry, Genetics and Molecular Biology › Molecular Biology › CRISPR and Genetic Engineering
Life Sciences › Biochemistry, Genetics and Molecular Biology › Genetics › Virus-based gene therapy research
Keywords: zinc finger nucleases, genome editing, hemophilia B, in vivo gene targeting, DNA double-strand breaks, homology-directed repair, gene replacement, liver regeneration, genetic disease treatment
MeSH terms: Animals, Base Sequence, Hemophilia B, Disease Models, Animal, DNA Repair, Endonucleases, Exons, Factor IX, Genetic Vectors, Hemostasis, Humans, Introns, Liver, Liver Regeneration, Mice, Inbred C57BL, Mutation, Phenotype, Genetic Therapy, Zinc Fingers, Genome, Sequence Homology, Gene Targeting, Cell Line, Tumor, Mice, DNA Breaks, Double-Stranded, HEK293 Cells

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In vivo genome editing restores haemostasis in a mouse model of haemophilia

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