GABAergic interneurons contribute to the fatal seizure phenotype of CLN2 disease mice.

Autor: Takahashi K, Rensing NR, Eultgen EM, Wang SH, Nelvagal HR, Le SQ, Roberts MS, Doray B, Han EB, Dickson PI, Wong M, Sands MS, Cooper JD

Publikováno v: BioRxiv : the preprint server for biology [bioRxiv] 2024 Mar 30. Date of Electronic Publication: 2024 Mar 30.

Cross-species efficacy of enzyme replacement therapy for CLN1 disease in mice and sheep.

Autor: Nelvagal HR; Department of Pediatrics, Washington University in St. Louis, School of Medicine, St. Louis, Missouri, USA., Eaton SL; The Roslin Institute and Royal (Dick) School of Veterinary Studies, University of Edinburgh, Easter Bush Campus, Easter Bush, Scotland, United Kingdom., Wang SH; Department of Pediatrics, Washington University in St. Louis, School of Medicine, St. Louis, Missouri, USA., Eultgen EM; Department of Pediatrics, Washington University in St. Louis, School of Medicine, St. Louis, Missouri, USA., Takahashi K; Department of Pediatrics, Washington University in St. Louis, School of Medicine, St. Louis, Missouri, USA., Le SQ; Department of Pediatrics, Washington University in St. Louis, School of Medicine, St. Louis, Missouri, USA., Nesbitt R; Department of Medicine, Washington University in St. Louis, School of Medicine, St .Louis, Missouri, USA., Dearborn JT; Department of Medicine, Washington University in St. Louis, School of Medicine, St .Louis, Missouri, USA., Siano N; Discovery Science Division, Amicus Therapeutics Inc., Philadelphia, Pennsylvania, USA., Puhl AC; Collaborations Pharmaceuticals Inc., Lab 3510, Raleigh, North Carolina, USA., Dickson PI; Department of Pediatrics, Washington University in St. Louis, School of Medicine, St. Louis, Missouri, USA.; Department of Genetics, Washington University in St. Louis, School of Medicine, St. Louis, Missouri, USA., Thompson G; Centre for Clinical Brain Sciences, University of Edinburgh, Chancellor's Building, Edinburgh, Scotland, United Kingdom.; Department of Clinical Neurosciences, NHS Lothian, Edinburgh, Scotland, United Kingdom., Murdoch F; The Roslin Institute and Royal (Dick) School of Veterinary Studies, University of Edinburgh, Easter Bush Campus, Easter Bush, Scotland, United Kingdom., Brennan PM; Centre for Clinical Brain Sciences, University of Edinburgh, Chancellor's Building, Edinburgh, Scotland, United Kingdom.; Department of Clinical Neurosciences, NHS Lothian, Edinburgh, Scotland, United Kingdom., Gray M; The Roslin Institute and Royal (Dick) School of Veterinary Studies, University of Edinburgh, Easter Bush Campus, Easter Bush, Scotland, United Kingdom.; The Large Animal Research and Imaging Facility (LARIF), Royal (Dick) School of Veterinary Studies, University of Edinburgh, Easter Bush Campus, Easter Bush, Scotland, United Kingdom., Greenhalgh SN; The Roslin Institute and Royal (Dick) School of Veterinary Studies, University of Edinburgh, Easter Bush Campus, Easter Bush, Scotland, United Kingdom.; The Large Animal Research and Imaging Facility (LARIF), Royal (Dick) School of Veterinary Studies, University of Edinburgh, Easter Bush Campus, Easter Bush, Scotland, United Kingdom., Tennant P; The Roslin Institute and Royal (Dick) School of Veterinary Studies, University of Edinburgh, Easter Bush Campus, Easter Bush, Scotland, United Kingdom.; The Large Animal Research and Imaging Facility (LARIF), Royal (Dick) School of Veterinary Studies, University of Edinburgh, Easter Bush Campus, Easter Bush, Scotland, United Kingdom., Gregson R; The Roslin Institute and Royal (Dick) School of Veterinary Studies, University of Edinburgh, Easter Bush Campus, Easter Bush, Scotland, United Kingdom.; The Large Animal Research and Imaging Facility (LARIF), Royal (Dick) School of Veterinary Studies, University of Edinburgh, Easter Bush Campus, Easter Bush, Scotland, United Kingdom., Clutton E; The Roslin Institute and Royal (Dick) School of Veterinary Studies, University of Edinburgh, Easter Bush Campus, Easter Bush, Scotland, United Kingdom.; The Large Animal Research and Imaging Facility (LARIF), Royal (Dick) School of Veterinary Studies, University of Edinburgh, Easter Bush Campus, Easter Bush, Scotland, United Kingdom., Nixon J; The Roslin Institute and Royal (Dick) School of Veterinary Studies, University of Edinburgh, Easter Bush Campus, Easter Bush, Scotland, United Kingdom.; The Large Animal Research and Imaging Facility (LARIF), Royal (Dick) School of Veterinary Studies, University of Edinburgh, Easter Bush Campus, Easter Bush, Scotland, United Kingdom., Proudfoot C; The Roslin Institute and Royal (Dick) School of Veterinary Studies, University of Edinburgh, Easter Bush Campus, Easter Bush, Scotland, United Kingdom.; The Large Animal Research and Imaging Facility (LARIF), Royal (Dick) School of Veterinary Studies, University of Edinburgh, Easter Bush Campus, Easter Bush, Scotland, United Kingdom., Guido S; The Roslin Institute and Royal (Dick) School of Veterinary Studies, University of Edinburgh, Easter Bush Campus, Easter Bush, Scotland, United Kingdom., Lillico SG; The Roslin Institute and Royal (Dick) School of Veterinary Studies, University of Edinburgh, Easter Bush Campus, Easter Bush, Scotland, United Kingdom., Whitelaw CBA; The Roslin Institute and Royal (Dick) School of Veterinary Studies, University of Edinburgh, Easter Bush Campus, Easter Bush, Scotland, United Kingdom., Lu JY; Department of Internal Medicine, University of Texas Southwestern Medical Center, Dallas, Texas, USA., Hofmann SL; Department of Internal Medicine, University of Texas Southwestern Medical Center, Dallas, Texas, USA., Ekins S; Collaborations Pharmaceuticals Inc., Lab 3510, Raleigh, North Carolina, USA., Sands MS; Department of Medicine, Washington University in St. Louis, School of Medicine, St .Louis, Missouri, USA.; Department of Genetics, Washington University in St. Louis, School of Medicine, St. Louis, Missouri, USA., Wishart TM; The Roslin Institute and Royal (Dick) School of Veterinary Studies, University of Edinburgh, Easter Bush Campus, Easter Bush, Scotland, United Kingdom., Cooper JD; Department of Pediatrics, Washington University in St. Louis, School of Medicine, St. Louis, Missouri, USA.; Department of Genetics, Washington University in St. Louis, School of Medicine, St. Louis, Missouri, USA.; Department of Neurology, Washington University in St. Louis, School of Medicine, St. Louis, Missouri, USA.

Publikováno v: The Journal of clinical investigation [J Clin Invest] 2022 Oct 17; Vol. 132 (20). Date of Electronic Publication: 2022 Oct 17.