Fanconi anemia gene editing by the CRISPR/Cas9 system.

@article{Osborn2015FanconiAG,
  title={Fanconi anemia gene editing by the CRISPR/Cas9 system.},
  author={Mark J. Osborn and Richard Gabriel and Beau R Webber and Anthony P. DeFeo and Amber N. McElroy and Jordan Jarjour and Colby Starker and John E Wagner and J. Keith Joung and Daniel F. Voytas and Christof von Kalle and Manfred Schmidt and Bruce R Blazar and Jakub Tolar},
  journal={Human gene therapy},
  year={2015},
  volume={26 2},
  pages={
          114-26
        }
}
Genome engineering with designer nucleases is a rapidly progressing field, and the ability to correct human gene mutations in situ is highly desirable. We employed fibroblasts derived from a patient with Fanconi anemia as a model to test the ability of the clustered regularly interspaced short palindromic repeats/Cas9 nuclease system to mediate gene correction. We show that the Cas9 nuclease and nickase each resulted in gene correction, but the nickase, because of its ability to preferentially… CONTINUE READING
Highly Cited
This paper has 50 citations. REVIEW CITATIONS
Related Discussions
This paper has been referenced on Twitter 2 times. VIEW TWEETS

Citations

Publications citing this paper.
Showing 1-10 of 35 extracted citations

Epigenome Editing

Methods in Molecular Biology • 2018
View 4 Excerpts
Highly Influenced

CRISPR/Cas9 Targeted Gene Editing and Cellular Engineering in Fanconi Anemia.

Stem cells and development • 2016
View 9 Excerpts
Method Support
Highly Influenced

Stem Cell Therapy for Fanconi Anemia.

Advances in experimental medicine and biology • 2018
View 2 Excerpts

Stem Cells: Biology and Engineering

Advances in Experimental Medicine and Biology • 2018

fewer than 50 Citations

0102020152016201720182019
Citations per Year
Semantic Scholar estimates that this publication has 50 citations based on the available data.

See our FAQ for additional information.

References

Publications referenced by this paper.
Showing 1-10 of 61 references

RNA-guided human genome engineering via Cas9.

Science • 2013
View 6 Excerpts
Highly Influenced

An unbiased genome-wide analysis of zinc-finger nuclease specificity

Nature Biotechnology • 2011
View 10 Excerpts
Highly Influenced

A rapid and general assay for monitoring endogenous gene modification.

Methods in molecular biology • 2010
View 5 Excerpts
Highly Influenced

Homology-directed repair of DNA nicks via pathways distinct from canonical double-strand break repair.

Proceedings of the National Academy of Sciences of the United States of America • 2014
View 7 Excerpts
Highly Influenced

Multiplex genome engineering using CRISPR/Cas systems.

Science • 2013
View 4 Excerpts
Highly Influenced

TALEN-based gene correction for epidermolysis bullosa.

Molecular therapy : the journal of the American Society of Gene Therapy • 2013
View 5 Excerpts
Highly Influenced