CRISPR/Cas9 β-globin gene targeting in human haematopoietic stem cells-Reference-Cited by-同舟云学术

CRISPR/Cas9 β-globin gene targeting in human haematopoietic stem cells

Published:2016-11-07 Issue:7629 Volume:539 Page:384-389
ISSN:0028-0836
Container-title:Nature
language:en
Short-container-title:Nature

Author:

Dever Daniel P.,Bak Rasmus O.,Reinisch Andreas,Camarena Joab,Washington Gabriel,Nicolas Carmencita E.,Pavel-Dinu Mara,Saxena Nivi,Wilkens Alec B.,Mantri Sruthi,Uchida Nobuko,Hendel Ayal,Narla Anupama,Majeti Ravindra,Weinberg Kenneth I.,Porteus Matthew H.

Publisher

Springer Science and Business Media LLC

Subject

Multidisciplinary

Link

https://www.nature.com/articles/nature20134.pdf

Reference48 articles.

1. Naldini, L. Ex vivo gene transfer and correction for cell-based therapies. Nat. Rev. Genet. 12, 301–315 (2011)

2. Porteus, M. Genome editing: a new approach to human therapeutics. Annu. Rev. Pharmacol. Toxicol. 56, 163–190 (2016)

3. Smithies, O., Gregg, R. G., Boggs, S. S., Koralewski, M. A. & Kucherlapati, R. S. Insertion of DNA sequences into the human chromosomal β-globin locus by homologous recombination. Nature 317, 230–234 (1985)

4. Porteus, M. H. & Baltimore, D. Chimeric nucleases stimulate gene targeting in human cells. Science 300, 763 (2003)

5. Rouet, P., Smih, F. & Jasin, M. Expression of a site-specific endonuclease stimulates homologous recombination in mammalian cells. Proc. Natl Acad. Sci. USA 91, 6064–6068 (1994)

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