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An ASO therapy for Angelman syndrome that targets an evolutionarily conserved region at the start of the UBE3A-AS transcript.

Autor: Dindot SV; Department of Veterinary Pathobiology, College of Veterinary Medicine and Biomedical Sciences, Texas A&M University, College Station, TX 77843, USA.; Department of Molecular and Cellular Medicine, College of Medicine, Texas A&M University, College Station, TX 77843, USA.; GeneTx Biotherapeutics LLC, Sarasota, FL 34233, USA.; Research Department, Ultragenyx Pharmaceutical, Novato, CA 94949, USA., Christian S; Department of Veterinary Pathobiology, College of Veterinary Medicine and Biomedical Sciences, Texas A&M University, College Station, TX 77843, USA., Murphy WJ; Department of Veterinary Integrative Biosciences, College of Veterinary Medicine and Biomedical Sciences, Texas A&M University, College Station, TX 77843, USA., Berent A; GeneTx Biotherapeutics LLC, Sarasota, FL 34233, USA., Panagoulias J; GeneTx Biotherapeutics LLC, Sarasota, FL 34233, USA., Schlafer A; Department of Veterinary Pathobiology, College of Veterinary Medicine and Biomedical Sciences, Texas A&M University, College Station, TX 77843, USA., Ballard J; Texas A&M Institute for Genomic Medicine (TIGM), Texas A&M University, College Station, TX 77843, USA., Radeva K; Department of Veterinary Pathobiology, College of Veterinary Medicine and Biomedical Sciences, Texas A&M University, College Station, TX 77843, USA.; School of Biosciences and Medicine, University of Surrey, Guildford GU2 7XH, UK., Robinson R; Department of Veterinary Pathobiology, College of Veterinary Medicine and Biomedical Sciences, Texas A&M University, College Station, TX 77843, USA.; School of Biosciences and Medicine, University of Surrey, Guildford GU2 7XH, UK., Myers L; Department of Veterinary Pathobiology, College of Veterinary Medicine and Biomedical Sciences, Texas A&M University, College Station, TX 77843, USA.; School of Biosciences and Medicine, University of Surrey, Guildford GU2 7XH, UK., Jepp T; Department of Veterinary Pathobiology, College of Veterinary Medicine and Biomedical Sciences, Texas A&M University, College Station, TX 77843, USA.; School of Biosciences and Medicine, University of Surrey, Guildford GU2 7XH, UK., Shaheen H; Department of Veterinary Pathobiology, College of Veterinary Medicine and Biomedical Sciences, Texas A&M University, College Station, TX 77843, USA., Hillman P; Department of Veterinary Pathobiology, College of Veterinary Medicine and Biomedical Sciences, Texas A&M University, College Station, TX 77843, USA.; Department of Molecular and Cellular Medicine, College of Medicine, Texas A&M University, College Station, TX 77843, USA., Konganti K; Texas A&M University Institute for Genome Sciences and Society (TIGSS), Texas A&M University, College Station, TX 77843, USA., Hillhouse A; Texas A&M University Institute for Genome Sciences and Society (TIGSS), Texas A&M University, College Station, TX 77843, USA., Bredemeyer KR; Department of Veterinary Integrative Biosciences, College of Veterinary Medicine and Biomedical Sciences, Texas A&M University, College Station, TX 77843, USA., Black L; Charles River Laboratories, Reno, NV 89511, USA., Douville J; Charles River Laboratories, Montreal, Senneville, Quebec H9X 1C1, Canada.

Publikováno v: Science translational medicine [Sci Transl Med] 2023 Mar 22; Vol. 15 (688), pp. eabf4077. Date of Electronic Publication: 2023 Mar 22.

Pathologic characterization of canine multiple system degeneration in the Ibizan hound.

Autor: St Jean SC; Texas A&M University, College Station, TX, USA., Jortner BS; Texas A&M University, College Station, TX, USA., Doan RN; Boston Children's Hospital, Harvard Medical School, Boston, MA, USA., Dindot SV; Texas A&M University, College Station, TX, USA., Johnson GS; University of Missouri, Columbia, MO, USA., Bullock G; University of Missouri, Columbia, MO, USA., Whitley DB; Antech Diagnostics, Stillwater, OK, USA., Levine JM; Texas A&M University, College Station, TX, USA., Hancock SK; Virginia-Maryland College of Veterinary Medicine, Blacksburg, VA, USA., Ambrus A; Texas A&M University, College Station, TX, USA., Porter BF; Texas A&M University, College Station, TX, USA.

Publikováno v: Veterinary pathology [Vet Pathol] 2022 Jan; Vol. 59 (1), pp. 132-137. Date of Electronic Publication: 2021 Sep 07.

Regulation of autism-relevant behaviors by cerebellar-prefrontal cortical circuits.

Autor: Kelly E; Department of Neurology and Neurotherapeutics, University of Texas Southwestern Medical Center, Dallas, TX, USA.; Department of Neuroscience, University of Texas Southwestern Medical Center, Dallas, TX, USA., Meng F; Department of Neurology and Neurotherapeutics, University of Texas Southwestern Medical Center, Dallas, TX, USA., Fujita H; Departments of Otolaryngology-Head and Neck Surgery, Neurology and Neuroscience, Johns Hopkins University School of Medicine, Baltimore, MD, USA., Morgado F; Department of Medical Biophysics, University of Toronto, Toronto, ON, Canada.; Mouse Imaging Centre, Toronto Hospital for Sick Children, Toronto, ON, Canada., Kazemi Y; Department of Neurology and Neurotherapeutics, University of Texas Southwestern Medical Center, Dallas, TX, USA., Rice LC; Department of Neuroscience, Center for Behavioral Neuroscience, American University, Washington, DC, USA., Ren C; Department of Neurology and Neurotherapeutics, University of Texas Southwestern Medical Center, Dallas, TX, USA., Escamilla CO; Department of Neurology and Neurotherapeutics, University of Texas Southwestern Medical Center, Dallas, TX, USA., Gibson JM; Department of Neurology and Neurotherapeutics, University of Texas Southwestern Medical Center, Dallas, TX, USA.; Department of Neuroscience, University of Texas Southwestern Medical Center, Dallas, TX, USA., Sajadi S; Department of Neurology and Neurotherapeutics, University of Texas Southwestern Medical Center, Dallas, TX, USA., Pendry RJ; Department of Neuroscience, University of Texas Southwestern Medical Center, Dallas, TX, USA., Tan T; Department of Neurology and Neurotherapeutics, University of Texas Southwestern Medical Center, Dallas, TX, USA., Ellegood J; Mouse Imaging Centre, Toronto Hospital for Sick Children, Toronto, ON, Canada., Basson MA; Centre for Craniofacial and Regenerative Biology and MRC Centre for Neurodevelopmental Disorders, King's College London, London, UK., Blakely RD; Department of Biomedical Science, Charles E. Schmidt College of Medicine and Brain Institute, Florida Atlantic University, Jupiter, Florida, USA., Dindot SV; Department of Veterinary Pathobiology, College of Veterinary Medicine & Biomedical Sciences, Texas A&M University, College Station, TX, USA., Golzio C; Institut de Génétique et de Biologie Moléculaire et Cellulaire; Centre National de la Recherche Scientifique; Institut National de la Santé et de la Recherche Médicale; Université de Strasbourg, Illkirch, France., Hahn MK; Department of Biomedical Science, Charles E. Schmidt College of Medicine and Brain Institute, Florida Atlantic University, Jupiter, Florida, USA., Katsanis N; ACT-GeM, Department of Human Genetics at Stanley Manne Children's Research Institute; Department of Pediatrics and Cellular and Molecular Biology, Northwestern University Feinberg School of Medicine, Chicago, IL, USA., Robins DM; Department of Human Genetics, University of Michigan Medical School, Ann Arbor, MI, USA., Silverman JL; MIND Institute and Department of Psychiatry and Behavioral Sciences, University of California, Davis, Davis, CA, USA., Singh KK; Department of Biochemistry and Biomedical Sciences, McMaster University, Hamilton, ON, Canada., Wevrick R; Department of Medical Genetics, University of Alberta, Edmonton, AB, Canada., Taylor MJ; Department of Medical Imaging and Psychology, University of Toronto; Diagnostic Imaging, Hospital for Sick Children, Toronto, ON, USA., Hammill C; Mouse Imaging Centre, Toronto Hospital for Sick Children, Toronto, ON, Canada., Anagnostou E; Department of Pediatrics, University of Toronto, Holland Bloorview Kids Rehabilitation Hospital, Toronto, ON, USA., Pfeiffer BE; Department of Neuroscience, University of Texas Southwestern Medical Center, Dallas, TX, USA., Stoodley CJ; Department of Neuroscience, Center for Behavioral Neuroscience, American University, Washington, DC, USA., Lerch JP; Department of Medical Biophysics, University of Toronto, Toronto, ON, Canada.; Mouse Imaging Centre, Toronto Hospital for Sick Children, Toronto, ON, Canada.; Wellcome Centre for Integrative Neuroimaging, FMRIB, Nuffield Department of Clinical Neuroscience, University of Oxford, Oxford, UK., du Lac S; Departments of Otolaryngology-Head and Neck Surgery, Neurology and Neuroscience, Johns Hopkins University School of Medicine, Baltimore, MD, USA., Tsai PT; Department of Neurology and Neurotherapeutics, University of Texas Southwestern Medical Center, Dallas, TX, USA. peter.tsai@utsouthwestern.edu.; Department of Neuroscience, University of Texas Southwestern Medical Center, Dallas, TX, USA. peter.tsai@utsouthwestern.edu.; Departments of Psychiatry and Pediatrics, University of Texas Southwestern Medical Center, Dallas, TX, USA. peter.tsai@utsouthwestern.edu.

Publikováno v: Nature neuroscience [Nat Neurosci] 2020 Sep; Vol. 23 (9), pp. 1102-1110. Date of Electronic Publication: 2020 Jul 13.

Generation of a Novel Rat Model of Angelman Syndrome with a Complete Ube3a Gene Deletion.

Autor: Dodge A; Department of Molecular Pharmacology and Physiology, University of South Florida, Tampa, Florida., Peters MM; Department of Molecular Pharmacology and Physiology, University of South Florida, Tampa, Florida., Greene HE; Department of Molecular Pharmacology and Physiology, University of South Florida, Tampa, Florida., Dietrick C; Department of Molecular Pharmacology and Physiology, University of South Florida, Tampa, Florida., Botelho R; Department of Molecular Pharmacology and Physiology, University of South Florida, Tampa, Florida., Chung D; Department of Molecular Pharmacology and Physiology, University of South Florida, Tampa, Florida., Willman J; Department of Molecular Pharmacology and Physiology, University of South Florida, Tampa, Florida., Nenninger AW; Department of Molecular Pharmacology and Physiology, University of South Florida, Tampa, Florida., Ciarlone S; Department of Molecular Pharmacology and Physiology, University of South Florida, Tampa, Florida.; PTC Therapeutics Inc., Plainfield, 07080, New Jersey., Kamath SG; Department of Molecular Pharmacology and Physiology, University of South Florida, Tampa, Florida., Houdek P; Department of Neurohumoral Regulations, Institute of Physiology, Czech Academy of Sciences, Prague, Czech Republic., Sumová A; Department of Neurohumoral Regulations, Institute of Physiology, Czech Academy of Sciences, Prague, Czech Republic., Anderson AE; Department of Pediatrics, Baylor College of Medicine, Houston, Texas., Dindot SV; Department of Veterinary Pathobiology, Texas A&M, College Station, Texas., Berg EL; School of Medicine, MIND Institute, Department of Psychiatry and Behavioral Sciences, University of California - Davis, Sacramento, California., O'Geen H; Genome Center and MIND Institute, University of California - Davis, Davis, California., Segal DJ; Genome Center and MIND Institute, University of California - Davis, Davis, California., Silverman JL; School of Medicine, MIND Institute, Department of Psychiatry and Behavioral Sciences, University of California - Davis, Sacramento, California., Weeber EJ; Department of Molecular Pharmacology and Physiology, University of South Florida, Tampa, Florida.; PTC Therapeutics Inc., Plainfield, 07080, New Jersey., Nash KR; Department of Molecular Pharmacology and Physiology, University of South Florida, Tampa, Florida.

Publikováno v: Autism research : official journal of the International Society for Autism Research [Autism Res] 2020 Mar; Vol. 13 (3), pp. 397-409. Date of Electronic Publication: 2020 Jan 21.