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Spell Checking Nature: Versatility of CRISPR/Cas9 for Developing Treatments for Inherited Disorders

Wojtal, Daria, Kemaladewi, Dwi U., Malam, Zeenat, Abdullah, Sarah, Wong, Tatianna W.Y., Hyatt, Elzbieta, Baghestani, Zahra, Pereira, Sergio +11 more·2016·The American Journal of Human Genetics
DOI 10.1016/j.ajhg.2015.11.012
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  • CRISPR/Cas9 targeting exon duplications in the human dystrophin gene in vitro led to production of full-length dystrophin in myotubules of an individual with DMD.

    “Other studies have used CRISPR/Cas9 to target duplication of exons in the human dystrophin gene in vitro and have shown that this approach can lead to production of full-length dystrophin in the myotubules of an individual with DMD. 8”

    From What is CRISPR/Cas9? (2016) · cites this paper as [8]

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Authors
Wojtal, D., Kemaladewi, D. U., Malam, Z., Abdullah, S., Wong, T. W. Y., Hyatt, E., Baghestani, Z., Pereira, S., Stavropoulos, J., Mouly, V., Mamchaoui, K., Muntoni, F., Voit, T., Gonorazky, H. D., Dowling, J. J., Wilson, M. D., Mendoza-Londono, R., Ivakine, E. A., & Cohn, R. D.
Venue
The American Journal of Human Genetics
Publisher
Elsevier BV
Published
2016-01
Volume
98
Issue
1
Pages
90-101
ISSN
0002-9297
DOI
10.1016/j.ajhg.2015.11.012
Cited by
103
References
11
Language
en
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