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Knock-in mouse models for CMTX1 show a loss of function phenotype in the peripheral nervous system.

Autor: Abrams CK; Department of Neurology and Rehabilitation, College of Medicine, University of Illinois at Chicago, 912 South Wood Street, Chicago, IL 60657, USA; Richard and Loan Hill Department of Biomedical Engineering, University of Illinois at Chicago, USA. Electronic address: cabrams1@uic.edu., Lancaster E; Department of Neurology, The Perelman School of Medicine, University of Pennsylvania, Philadelphia, PA 19104, USA.. Electronic address: eoh@pennmedicine.upenn.edu., Li JJ; Department of Neurology, The Perelman School of Medicine, University of Pennsylvania, Philadelphia, PA 19104, USA.. Electronic address: LiJian@pennmedicine.upenn.edu., Dungan G; Department of Neurology and Rehabilitation, College of Medicine, University of Illinois at Chicago, 912 South Wood Street, Chicago, IL 60657, USA., Gong D; Richard and Loan Hill Department of Biomedical Engineering, University of Illinois at Chicago, USA. Electronic address: gong9@uic.edu., Scherer SS; Department of Neurology, The Perelman School of Medicine, University of Pennsylvania, Philadelphia, PA 19104, USA.. Electronic address: sscherer@pennmedicine.upenn.edu., Freidin MM; Department of Neurology and Rehabilitation, College of Medicine, University of Illinois at Chicago, 912 South Wood Street, Chicago, IL 60657, USA. Electronic address: mfreidin@uic.edu.

Publikováno v: Experimental neurology [Exp Neurol] 2023 Feb; Vol. 360, pp. 114277. Date of Electronic Publication: 2022 Nov 18.

AAV1.NT-3 gene therapy for X-linked Charcot-Marie-Tooth neuropathy type 1.

Autor: Ozes B; Center for Gene Therapy, The Abigail Wexner Research Institute at Nationwide Children's Hospital, Columbus, OH, USA., Myers M; Center for Gene Therapy, The Abigail Wexner Research Institute at Nationwide Children's Hospital, Columbus, OH, USA., Moss K; Center for Gene Therapy, The Abigail Wexner Research Institute at Nationwide Children's Hospital, Columbus, OH, USA., Mckinney J; Department of Pediatrics and Neurology, Nationwide Children's Hospital and The Ohio State University, Columbus, OH, USA., Ridgley A; Center for Gene Therapy, The Abigail Wexner Research Institute at Nationwide Children's Hospital, Columbus, OH, USA., Chen L; Center for Gene Therapy, The Abigail Wexner Research Institute at Nationwide Children's Hospital, Columbus, OH, USA., Bai S; Department of Biomedical Informatics, The Ohio State University College of Medicine, Columbus, OH, USA.; Biostatistics Resource at Nationwide Children's Hospital, Columbus, OH, USA., Abrams CK; Department of Neurology and Rehabilitation, University of Illinois at Chicago, Chicago, IL, USA., Freidin MM; Department of Neurology and Rehabilitation, University of Illinois at Chicago, Chicago, IL, USA., Mendell JR; Center for Gene Therapy, The Abigail Wexner Research Institute at Nationwide Children's Hospital, Columbus, OH, USA.; Department of Pediatrics and Neurology, Nationwide Children's Hospital and The Ohio State University, Columbus, OH, USA., Sahenk Z; Center for Gene Therapy, The Abigail Wexner Research Institute at Nationwide Children's Hospital, Columbus, OH, USA. zarife.sahenk@nationwidechildrens.org.; Department of Pediatrics and Neurology, Nationwide Children's Hospital and The Ohio State University, Columbus, OH, USA. zarife.sahenk@nationwidechildrens.org.; Department of Pathology and Laboratory Medicine, Nationwide Children's Hospital, Columbus, OH, USA. zarife.sahenk@nationwidechildrens.org.

Publikováno v: Gene therapy [Gene Ther] 2022 Apr; Vol. 29 (3-4), pp. 127-137. Date of Electronic Publication: 2021 Feb 04.