Perez-Pinera P - Search Results

1

CRISPR base editing of cis-regulatory elements enables the perturbation of neurodegeneration-linked genes.

Lim CKW, McCallister TX, Saporito-Magriña C, McPheron GD, Krishnan R, Zeballos C MA, Powell JE, Clark LV, Perez-Pinera P, Gaj T. Lim CKW, et al. Mol Ther. 2022 Dec 7;30(12):3619-3631. doi: 10.1016/j.ymthe.2022.08.008. Epub 2022 Aug 13. Mol Ther. 2022. PMID: 35965414 Free PMC article.

2

Treatment of a Mouse Model of ALS by In Vivo Base Editing.

Lim CKW, Gapinske M, Brooks AK, Woods WS, Powell JE, Zeballos C MA, Winter J, Perez-Pinera P, Gaj T. Lim CKW, et al. Mol Ther. 2020 Apr 8;28(4):1177-1189. doi: 10.1016/j.ymthe.2020.01.005. Epub 2020 Jan 14. Mol Ther. 2020. PMID: 31991108 Free PMC article.

3

The continuously evolving CRISPR barcoding toolbox.

Gaj T, Perez-Pinera P. Gaj T, et al. Genome Biol. 2018 Sep 25;19(1):143. doi: 10.1186/s13059-018-1541-y. Genome Biol. 2018. PMID: 30253789 Free PMC article.

4

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. 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.

5

Directed Evolution of CRISPR-Cas9 Base Editors.

Winter J, Perez-Pinera P. Winter J, et al. Trends Biotechnol. 2019 Nov;37(11):1151-1153. doi: 10.1016/j.tibtech.2019.09.005. Epub 2019 Oct 14. Trends Biotechnol. 2019. PMID: 31623959

6

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. 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.

7

CRISPR-SKIP: programmable gene splicing with single base editors.

Gapinske M, Luu A, Winter J, Woods WS, Kostan KA, Shiva N, Song JS, Perez-Pinera P. Gapinske M, et al. Genome Biol. 2018 Aug 15;19(1):107. doi: 10.1186/s13059-018-1482-5. Genome Biol. 2018. PMID: 30107853 Free PMC article.

8

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. 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.

9

Putting Non-coding RNA on Display with CRISPR.

Perez-Pinera P, Jones MF, Lal A, Lu TK. Perez-Pinera P, et al. Mol Cell. 2015 Jul 16;59(2):146-8. doi: 10.1016/j.molcel.2015.07.002. Mol Cell. 2015. PMID: 26186289 Free PMC article.

10

Targeted Gene Activation Using RNA-Guided Nucleases.

Brown A, Woods WS, Perez-Pinera P. Brown A, et al. Methods Mol Biol. 2017;1468:235-50. doi: 10.1007/978-1-4939-4035-6_16. Methods Mol Biol. 2017. PMID: 27662880

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