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Items: 4

1.

CRISPR-Cas9 corrects Duchenne muscular dystrophy exon 44 deletion mutations in mice and human cells.

Min YL, Li H, Rodriguez-Caycedo C, Mireault AA, Huang J, Shelton JM, McAnally JR, Amoasii L, Mammen PPA, Bassel-Duby R, Olson EN.

Sci Adv. 2019 Mar 6;5(3):eaav4324. doi: 10.1126/sciadv.aav4324. eCollection 2019 Mar.

2.

Single-cut genome editing restores dystrophin expression in a new mouse model of muscular dystrophy.

Amoasii L, Long C, Li H, Mireault AA, Shelton JM, Sanchez-Ortiz E, McAnally JR, Bhattacharyya S, Schmidt F, Grimm D, Hauschka SD, Bassel-Duby R, Olson EN.

Sci Transl Med. 2017 Nov 29;9(418). pii: eaan8081. doi: 10.1126/scitranslmed.aan8081. Erratum in: Sci Transl Med. 2018 Jan 24;10 (425):.

3.

Postnatal genome editing partially restores dystrophin expression in a mouse model of muscular dystrophy.

Long C, Amoasii L, Mireault AA, McAnally JR, Li H, Sanchez-Ortiz E, Bhattacharyya S, Shelton JM, Bassel-Duby R, Olson EN.

Science. 2016 Jan 22;351(6271):400-3. doi: 10.1126/science.aad5725. Epub 2015 Dec 31.

4.

Prevention of muscular dystrophy in mice by CRISPR/Cas9-mediated editing of germline DNA.

Long C, McAnally JR, Shelton JM, Mireault AA, Bassel-Duby R, Olson EN.

Science. 2014 Sep 5;345(6201):1184-1188. doi: 10.1126/science.1254445. Epub 2014 Aug 14.

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