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Targeting 3′ and 5′ untranslated regions with antisense oligonucleotides to stabilize frataxin mRNA and increase protein expression |
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| Authors: | Yanjie Li Jixue Li Jun Wang David R Lynch Xiulong Shen David R Corey Darshan Parekh Balkrishen Bhat Caroline Woo Jonathan J Cherry Jill S Napierala Marek Napierala |
| Institution: | Department of Biochemistry and Molecular Genetics, University of Alabama at Birmingham, 1825 University Blvd, Birmingham, AL 35294, USA;Division of Neurology and Pediatrics, Children’s Hospital of Philadelphia, Abramson Research Center, Room 502, Philadelphia, PA 19104, USA;Department of Pharmacology and Biochemistry, University of Texas Southwestern Medical Center, Dallas, TX 75390, USA;Translate Bio, 29 Hartwell Avenue, Lexington, MA 02421, USA |
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