Skip to main page content
U.S. flag

An official website of the United States government

Dot gov

The .gov means it’s official.
Federal government websites often end in .gov or .mil. Before sharing sensitive information, make sure you’re on a federal government site.

Https

The site is secure.
The https:// ensures that you are connecting to the official website and that any information you provide is encrypted and transmitted securely.

Access keys NCBI Homepage MyNCBI Homepage Main Content Main Navigation
Comment
. 2019 Mar 15;115(4):e39-e40.
doi: 10.1093/cvr/cvz038.

Therapeutic gene editing, making a point

Anke M Smits  1
Affiliations
Comment

Therapeutic gene editing, making a point

Anke M Smits. Cardiovasc Res. .
No abstract available

PubMed Disclaimer

Comment on

  • In utero CRISPR-mediated therapeutic editing of metabolic genes.
    Rossidis AC, Stratigis JD, Chadwick AC, Hartman HA, Ahn NJ, Li H, Singh K, Coons BE, Li L, Lv W, Zoltick PW, Alapati D, Zacharias W, Jain R, Morrisey EE, Musunuru K, Peranteau WH. Rossidis AC, et al. Nat Med. 2018 Oct;24(10):1513-1518. doi: 10.1038/s41591-018-0184-6. Epub 2018 Oct 8. Nat Med. 2018. PMID: 30297903 Free PMC article.
  • Treatment of a metabolic liver disease by in vivo genome base editing in adult mice.
    Villiger L, Grisch-Chan HM, Lindsay H, Ringnalda F, Pogliano CB, Allegri G, Fingerhut R, Häberle J, Matos J, Robinson MD, Thöny B, Schwank G. Villiger L, et al. Nat Med. 2018 Oct;24(10):1519-1525. doi: 10.1038/s41591-018-0209-1. Epub 2018 Oct 8. Nat Med. 2018. PMID: 30297904

References

    1. Kosicki M, Tomberg K, Bradley A.. Repair of double-strand breaks induced by CRISPR–Cas9 leads to large deletions and complex rearrangements. Nat Biotechnol 2018;36:765–772. - PMC - PubMed
    1. Villiger L, Grisch-Chan HM, Lindsay H, Ringnalda F, Pogliano CB, Allegri G, Fingerhut R, Häberle J, Matos J, Robinson MD, Thöny B, Schwank G.. Treatment of a metabolic liver disease by in vivo genome base editing in adult mice. Nat Med 2018;24:1519–1525. - PubMed
    1. Rossidis AC, Stratigis JD, Chadwick AC, Hartman HA, Ahn NJ, Li H, Singh K, Coons BE, Li L, Lv W, Zoltick PW, Alapati D, Zacharias W, Jain R, Morrisey EE, Musunuru K, Peranteau WH.. In utero CRISPR-mediated therapeutic editing of metabolic genes. Nat Med 2018;24:1513–1518. - PMC - PubMed
    1. Gaudelli NM, Komor AC, Rees HA, Packer MS, Badran AH, Bryson DI, Liu DR.. Programmable base editing of T to G C in genomic DNA without DNA cleavage. Nature 2017;551:464–471. - PMC - PubMed
    1. Komor AC, Kim YB, Packer MS, Zuris JA, Liu DR.. Programmable editing of a target base in genomic DNA without double-stranded DNA cleavage. Nature 2016;533:420–424. - PMC - PubMed

Publication types

MeSH terms