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U1 snRNA-mediated gene therapeutic correction of splice defects caused by an exceptionally mild BBS mutation
Authors:Schmid Fabian  Glaus Esther  Barthelmes Daniel  Fliegauf Manfred  Gaspar Harald  Nürnberg Gudrun  Nürnberg Peter  Omran Heymut  Berger Wolfgang  Neidhardt John
Affiliation:University of Zurich, Institute of Medical Molecular Genetics, Schorenstrasse 16, Schwerzenbach, Switzerland.
Abstract:
Keywords:retinitis pigmentosa  splice defect  gene therapy  BBS1  U1 snRNA  splicing
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