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Correction of Dystrophin Expression in Cells From Duchenne Muscular Dystrophy Patients Through Genomic Excision of Exon 51 by Zinc Finger Nucleases.
- Published in:
- Molecular Therapy, 2015, v. 23, n. 3, p. 523, doi. 10.1038/mt.2014.234
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- Article
Reading Frame Correction by Targeted Genome Editing Restores Dystrophin Expression in Cells From Duchenne Muscular Dystrophy Patients.
- Published in:
- Molecular Therapy, 2013, v. 21, n. 9, p. 1718, doi. 10.1038/mt.2013.111
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- Article
Multiplex CRISPR/Cas9-based genome engineering from a single lentiviral vector.
- Published in:
- Nucleic Acids Research, 2014, v. 42, n. 19, p. e147, doi. 10.1093/nar/gku749
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- Article
Multiplex CRISPR/Cas9-based genome editing for correction of dystrophin mutations that cause Duchenne muscular dystrophy.
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- Nature Communications, 2015, v. 6, n. 2, p. 6244, doi. 10.1038/ncomms7244
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- Article
RNA-guided gene activation by CRISPR-Cas9-based transcription factors.
- Published in:
- Nature Methods, 2013, v. 10, n. 10, p. 973, doi. 10.1038/nmeth.2600
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- Article