Gersbach CA - Search Results

1

Full-length dystrophin restoration via targeted exon integration by AAV-CRISPR in a humanized mouse model of Duchenne muscular dystrophy.

Pickar-Oliver A, Gough V, Bohning JD, Liu S, Robinson-Hamm JN, Daniels H, Majoros WH, Devlin G, Asokan A, Gersbach CA. Pickar-Oliver A, et al. Among authors: gersbach ca. Mol Ther. 2021 Nov 3;29(11):3243-3257. doi: 10.1016/j.ymthe.2021.09.003. Epub 2021 Sep 10. Mol Ther. 2021. PMID: 34509668 Free PMC article.

2

Advances in targeted genome editing.

Perez-Pinera P, Ousterout DG, Gersbach CA. Perez-Pinera P, et al. Among authors: gersbach ca. Curr Opin Chem Biol. 2012 Aug;16(3-4):268-77. doi: 10.1016/j.cbpa.2012.06.007. Epub 2012 Jul 20. Curr Opin Chem Biol. 2012. PMID: 22819644 Free PMC article. Review.

3

Reading frame correction by targeted genome editing restores dystrophin expression in cells from Duchenne muscular dystrophy patients.

Ousterout DG, Perez-Pinera P, Thakore PI, Kabadi AM, Brown MT, Qin X, Fedrigo O, Mouly V, Tremblay JP, Gersbach CA. Ousterout DG, et al. Among authors: gersbach ca. Mol Ther. 2013 Sep;21(9):1718-26. doi: 10.1038/mt.2013.111. Epub 2013 Jun 4. Mol Ther. 2013. PMID: 23732986 Free PMC article.

4

Multiplex CRISPR/Cas9-based genome engineering from a single lentiviral vector.

Kabadi AM, Ousterout DG, Hilton IB, Gersbach CA. Kabadi AM, et al. Among authors: gersbach ca. Nucleic Acids Res. 2014 Oct 29;42(19):e147. doi: 10.1093/nar/gku749. Epub 2014 Aug 13. Nucleic Acids Res. 2014. PMID: 25122746 Free PMC article.

5

Correction of dystrophin expression in cells from Duchenne muscular dystrophy patients through genomic excision of exon 51 by zinc finger nucleases.

Ousterout DG, Kabadi AM, Thakore PI, Perez-Pinera P, Brown MT, Majoros WH, Reddy TE, Gersbach CA. Ousterout DG, et al. Among authors: gersbach ca. Mol Ther. 2015 Mar;23(3):523-32. doi: 10.1038/mt.2014.234. Epub 2014 Dec 10. Mol Ther. 2015. PMID: 25492562 Free PMC article.

6

Multiplex CRISPR/Cas9-based genome editing for correction of dystrophin mutations that cause Duchenne muscular dystrophy.

Ousterout DG, Kabadi AM, Thakore PI, Majoros WH, Reddy TE, Gersbach CA. Ousterout DG, et al. Among authors: gersbach ca. Nat Commun. 2015 Feb 18;6:6244. doi: 10.1038/ncomms7244. Nat Commun. 2015. PMID: 25692716 Free PMC article.

7

Genome-wide specificity of DNA binding, gene regulation, and chromatin remodeling by TALE- and CRISPR/Cas9-based transcriptional activators.

Polstein LR, Perez-Pinera P, Kocak DD, Vockley CM, Bledsoe P, Song L, Safi A, Crawford GE, Reddy TE, Gersbach CA. Polstein LR, et al. Among authors: gersbach ca. Genome Res. 2015 Aug;25(8):1158-69. doi: 10.1101/gr.179044.114. Epub 2015 May 29. Genome Res. 2015. PMID: 26025803 Free PMC article.

8

In vivo genome editing improves muscle function in a mouse model of Duchenne muscular dystrophy.

Nelson CE, Hakim CH, Ousterout DG, Thakore PI, Moreb EA, Castellanos Rivera RM, Madhavan S, Pan X, Ran FA, Yan WX, Asokan A, Zhang F, Duan D, Gersbach CA. Nelson CE, et al. Among authors: gersbach ca. Science. 2016 Jan 22;351(6271):403-7. doi: 10.1126/science.aad5143. Epub 2015 Dec 31. Science. 2016. PMID: 26721684 Free PMC article.

9

Genome-editing Technologies for Gene and Cell Therapy.

Maeder ML, Gersbach CA. Maeder ML, et al. Among authors: gersbach ca. Mol Ther. 2016 Mar;24(3):430-46. doi: 10.1038/mt.2016.10. Epub 2016 Jan 12. Mol Ther. 2016. PMID: 26755333 Free PMC article. Review.

10

In Vivo Zinc Finger Nuclease-mediated Targeted Integration of a Glucose-6-phosphatase Transgene Promotes Survival in Mice With Glycogen Storage Disease Type IA.

Landau DJ, Brooks ED, Perez-Pinera P, Amarasekara H, Mefferd A, Li S, Bird A, Gersbach CA, Koeberl DD. Landau DJ, et al. Among authors: gersbach ca. Mol Ther. 2016 Apr;24(4):697-706. doi: 10.1038/mt.2016.35. Epub 2016 Feb 11. Mol Ther. 2016. PMID: 26865405 Free PMC article.

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