Tento výsledek nelze pro nepřihlášené uživatele zobrazit.
K zobrazení výsledku je třeba se přihlásit.

Tento výsledek nelze pro nepřihlášené uživatele zobrazit.
K zobrazení výsledku je třeba se přihlásit.

Retraction Note: Rescue of the spinal muscular atrophy phenotype in a mouse model by early postnatal delivery of SMN.

Autor: Foust KD; Center for Gene Therapy, The Research Institute at Nationwide Children's Hospital, Columbus, Ohio, USA., Wang X; Wright State University, Dayton, Ohio, USA., McGovern VL; Department of Molecular and Cellular Biochemistry, The Ohio State University, Columbus, Ohio, USA., Braun L; Center for Gene Therapy, The Research Institute at Nationwide Children's Hospital, Columbus, Ohio, USA., Bevan AK; Center for Gene Therapy, The Research Institute at Nationwide Children's Hospital, Columbus, Ohio, USA.; Integrated Biomedical Graduate Program, The Ohio State University, Columbus, Ohio, USA., Haidet AM; Center for Gene Therapy, The Research Institute at Nationwide Children's Hospital, Columbus, Ohio, USA.; Integrated Biomedical Graduate Program, The Ohio State University, Columbus, Ohio, USA., Le TT; Department of Molecular and Cellular Biochemistry, The Ohio State University, Columbus, Ohio, USA., Morales PR; The Mannheimer Foundation, Inc., Homestead, Florida, USA., Rich MM; Wright State University, Dayton, Ohio, USA., Burghes AHM; Department of Molecular and Cellular Biochemistry, The Ohio State University, Columbus, Ohio, USA.; Integrated Biomedical Graduate Program, The Ohio State University, Columbus, Ohio, USA., Kaspar BK; Center for Gene Therapy, The Research Institute at Nationwide Children's Hospital, Columbus, Ohio, USA. Brian.Kaspar@NationwideChildrens.org.; Department of Molecular and Cellular Biochemistry, The Ohio State University, Columbus, Ohio, USA. Brian.Kaspar@NationwideChildrens.org.; Integrated Biomedical Graduate Program, The Ohio State University, Columbus, Ohio, USA. Brian.Kaspar@NationwideChildrens.org.

Publikováno v: Nature biotechnology [Nat Biotechnol] 2022 Nov; Vol. 40 (11), pp. 1692.

Biodistribution of onasemnogene abeparvovec DNA, mRNA and SMN protein in human tissue.

Autor: Thomsen G; Novartis Gene Therapies, Bannockburn, IL, USA., Burghes AHM; Department of Neurology, The Ohio State University, Columbus, OH, USA. burghes.1@osu.edu.; Department of Biological Chemistry and Pharmacology, The Ohio State University, Columbus, OH, USA. burghes.1@osu.edu., Hsieh C; Novartis Gene Therapies, Bannockburn, IL, USA., Do J; Novartis Gene Therapies, Bannockburn, IL, USA., Chu BTT; Novartis Gene Therapies, Bannockburn, IL, USA., Perry S; Novartis Gene Therapies, Bannockburn, IL, USA., Barkho B; Novartis Gene Therapies, Bannockburn, IL, USA., Kaufmann P; Novartis Gene Therapies, Bannockburn, IL, USA., Sproule DM; Novartis Gene Therapies, Bannockburn, IL, USA., Feltner DE; Novartis Gene Therapies, Bannockburn, IL, USA., Chung WK; Departments of Pediatrics and Medicine, Columbia University, New York, NY, USA., McGovern VL; Department of Biological Chemistry and Pharmacology, The Ohio State University, Columbus, OH, USA., Hevner RF; Department of Pathology, University of California, San Diego, CA, USA., Conces M; Department of Pathology and Laboratory Medicine, Nationwide Children's Hospital, Columbus, OH, USA.; Department of Pathology, The Ohio State University, Columbus, OH, USA., Pierson CR; Department of Pathology and Laboratory Medicine, Nationwide Children's Hospital, Columbus, OH, USA.; Department of Pathology, The Ohio State University, Columbus, OH, USA., Scoto M; National Institute for Health Research, Great Ormond Street Institute of Child Health Biomedical Research Centre, University College London, London, UK.; Great Ormond Street Hospital Trust, London, UK., Muntoni F; National Institute for Health Research, Great Ormond Street Institute of Child Health Biomedical Research Centre, University College London, London, UK.; Great Ormond Street Hospital Trust, London, UK., Mendell JR; Department of Neurology, The Ohio State University, Columbus, OH, USA.; Center for Gene Therapy, Nationwide Children's Hospital, Columbus, OH, USA.; Department of Pediatrics, The Ohio State University, Columbus, OH, USA., Foust KD; Novartis Gene Therapies, Bannockburn, IL, USA.

Publikováno v: Nature medicine [Nat Med] 2021 Oct; Vol. 27 (10), pp. 1701-1711. Date of Electronic Publication: 2021 Oct 04.

Intragenic complementation of amino and carboxy terminal SMN missense mutations can rescue Smn null mice.

Autor: McGovern VL; Department of Biological Chemistry and Pharmacology, The Ohio State University, Columbus, OH 43210, USA., Kray KM; Department of Biological Chemistry and Pharmacology, The Ohio State University, Columbus, OH 43210, USA., Arnold WD; Department of Neurology, The Ohio State University, Columbus, OH 43210, USA., Duque SI; Department of Biological Chemistry and Pharmacology, The Ohio State University, Columbus, OH 43210, USA., Iyer CC; Department of Biological Chemistry and Pharmacology, The Ohio State University, Columbus, OH 43210, USA., Massoni-Laporte A; Department of Biological Chemistry and Pharmacology, The Ohio State University, Columbus, OH 43210, USA., Workman E; Department of Biological Chemistry and Pharmacology, The Ohio State University, Columbus, OH 43210, USA., Patel A; Department of Biological Chemistry and Pharmacology, The Ohio State University, Columbus, OH 43210, USA., Battle DJ; Department of Biological Chemistry, The Ohio State University, Columbus, OH 43210, USA., Burghes AHM; Department of Biological Chemistry and Pharmacology, The Ohio State University, Columbus, OH 43210, USA.; Department of Neurology, The Ohio State University, Columbus, OH 43210, USA.; Department of Molecular Genetics, The Ohio State University, Columbus, OH 43210, USA.

Publikováno v: Human molecular genetics [Hum Mol Genet] 2020 Nov 01; Vol. 29 (21), pp. 3493-3503.