In an adult mouse model of tyrosinaemia, a base editor correcting an A-to-G splice-site mutation in the Fah gene restores the translation of the functional enzyme, promoting the repopulation of the liver with the corrected cells.
This is a preview of subscription content, access via your institution
Access options
Access Nature and 54 other Nature Portfolio journals
Get Nature+, our best-value online-access subscription
$29.99 / 30 days
cancel any time
Subscribe to this journal
Receive 12 digital issues and online access to articles
$99.00 per year
only $8.25 per issue
Buy this article
- Purchase on Springer Link
- Instant access to full article PDF
Prices may be subject to local taxes which are calculated during checkout
References
Yang, Y. et al. Nat. Biotechnol. 34, 334–338 (2016).
Komor, A. C., Kim, Y. B., Packer, M. S., Zuris, J. A. & Liu, D. R. Nature 533, 420–424 (2016).
Nishida, K. et al. Science 353, aaf8729 (2016).
Gaudelli, N. M. et al. Nature 551, 464–471 (2017).
Chadwick, A. C., Wang, X. & Musunuru, K. Arterioscler. Thromb. Vasc. Biol. 9, 1741–1747 (2017).
Rossidis, A. C. et al. Nat. Med. 24, 1513–1518 (2018).
Villiger, L. et al. Nat. Med. 24, 1519–1525 (2018).
Ryu, S.-M. et al. Nat. Biotechnol. 36, 536–539 (2018).
Song, C.-Q. et al. Nat. Biomed. Eng. https://doi.org/10.1038/s41551-019-0357-8 (2019).
Yin, H. et al. Nat. Biotechnol. 32, 551–553 (2014).
Yin, H. et al. Nat. Biotechnol. 34, 328–333 (2016).
Zuo, E. et al. Science 364, 289–292 (2019).
Jin, S. et al. Science 364, 292–295 (2019).
Grünewald, J. et al. Nature 569, 433–437 (2019).
Grünewald, J. et al. Nat. Biotechnol. 37, 1041–1048 (2019).
Author information
Authors and Affiliations
Corresponding author
Ethics declarations
Competing interests
K. M. is a co-founder and advisor of Verve Therapeutics, and an advisor of Variant Bio.
Rights and permissions
About this article
Cite this article
Musunuru, K. Correcting tyrosinaemia via a point mutation. Nat Biomed Eng 4, 14–15 (2020). https://doi.org/10.1038/s41551-019-0489-x
Published:
Issue Date:
DOI: https://doi.org/10.1038/s41551-019-0489-x