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CRISPR/Cas9-Mediated Genome Editing as a Therapeutic Approach for Leber Congenital Amaurosis 10.
Ruan, Guo-Xiang; Barry, Elizabeth; Yu, Dan; Lukason, Michael; Cheng, Seng H; Scaria, Abraham.
Affiliation
  • Ruan GX; Rare Diseases, Sanofi Genzyme, Framingham, MA 01701, USA. Electronic address: guoxiang.ruan@genzyme.com.
  • Barry E; Rare Diseases, Sanofi Genzyme, Framingham, MA 01701, USA.
  • Yu D; Rare Diseases, Sanofi Genzyme, Framingham, MA 01701, USA.
  • Lukason M; Rare Diseases, Sanofi Genzyme, Framingham, MA 01701, USA.
  • Cheng SH; Rare Diseases, Sanofi Genzyme, Framingham, MA 01701, USA.
  • Scaria A; Rare Diseases, Sanofi Genzyme, Framingham, MA 01701, USA.
Mol Ther ; 25(2): 331-341, 2017 02 01.
Article in En | MEDLINE | ID: mdl-28109959
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Full text: 1 Collection: 01-internacional Database: MEDLINE Main subject: Leber Congenital Amaurosis / CRISPR-Cas Systems / Gene Editing Limits: Animals Language: En Journal: Mol Ther Journal subject: BIOLOGIA MOLECULAR / TERAPEUTICA Year: 2017 Document type: Article Country of publication:
Fulltext
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Full text: 1 Collection: 01-internacional Database: MEDLINE Main subject: Leber Congenital Amaurosis / CRISPR-Cas Systems / Gene Editing Limits: Animals Language: En Journal: Mol Ther Journal subject: BIOLOGIA MOLECULAR / TERAPEUTICA Year: 2017 Document type: Article Country of publication: