Pre-clinical Safety and Off-Target Studies to Support Translation of AAV-Mediated RNAi Therapy for FSHD.
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| Title: | Pre-clinical Safety and Off-Target Studies to Support Translation of AAV-Mediated RNAi Therapy for FSHD. |
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| Authors: | Wallace LM; Center for Gene Therapy, The Research Institute at Nationwide Children's Hospital, Columbus, OH, USA., Saad NY; Center for Gene Therapy, The Research Institute at Nationwide Children's Hospital, Columbus, OH, USA., Pyne NK; Center for Gene Therapy, The Research Institute at Nationwide Children's Hospital, Columbus, OH, USA., Fowler AM; Center for Gene Therapy, The Research Institute at Nationwide Children's Hospital, Columbus, OH, USA., Eidahl JO; Center for Gene Therapy, The Research Institute at Nationwide Children's Hospital, Columbus, OH, USA., Domire JS; Center for Gene Therapy, The Research Institute at Nationwide Children's Hospital, Columbus, OH, USA., Griffin DA; Center for Gene Therapy, The Research Institute at Nationwide Children's Hospital, Columbus, OH, USA., Herman AC; Research Information Solutions and Innovation Infrastructure, The Research Institute at Nationwide Children's Hospital, Columbus, OH, USA., Sahenk Z; Center for Gene Therapy, The Research Institute at Nationwide Children's Hospital, Columbus, OH, USA.; Department of Pediatrics, The Ohio State University College of Medicine, Columbus, OH, USA.; Department of Neurology, The Ohio State University College of Medicine, Columbus, OH, USA., Rodino-Klapac LR; Center for Gene Therapy, The Research Institute at Nationwide Children's Hospital, Columbus, OH, USA.; Department of Pediatrics, The Ohio State University College of Medicine, Columbus, OH, USA., Harper SQ; Center for Gene Therapy, The Research Institute at Nationwide Children's Hospital, Columbus, OH, USA.; Department of Pediatrics, The Ohio State University College of Medicine, Columbus, OH, USA. |
| Source: | Molecular therapy. Methods & clinical development [Mol Ther Methods Clin Dev] 2017 Dec 24; Vol. 8, pp. 121-130. Date of Electronic Publication: 2017 Dec 24 (Print Publication: 2018). |
| Publication Type: | Journal Article |
| Journal Info: | Publisher: Cell Press Country of Publication: United States NLM ID: 101624857 Publication Model: eCollection Cited Medium: Print ISSN: 2329-0501 (Print) Linking ISSN: 23290501 NLM ISO Abbreviation: Mol Ther Methods Clin Dev Subsets: PubMed not MEDLINE |
| Database: | MEDLINE Ultimate |
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