A de novo Missense Mutation in PPP2R5D Alters Dopamine Pathways and Morphology of iPSC-derived Midbrain Neurons.

Autor: Carter JL; Center for Interventional Genetics, University of California Davis Health Systems, Sacramento, CA, USA.; MIND Institute, University of California Davis Health Systems, Sacramento, CA, USA.; Stem Cell Program and Gene Therapy Center, University of California Davis Health Systems, Sacramento, CA, USA.; Institute for Regenerative Cures, University of California Davis Health Systems, Sacramento, CA, USA.; Department of Neurology, University of California Davis Health Systems, Sacramento, CA, USA., Halmai JANM; Center for Interventional Genetics, University of California Davis Health Systems, Sacramento, CA, USA.; MIND Institute, University of California Davis Health Systems, Sacramento, CA, USA.; Stem Cell Program and Gene Therapy Center, University of California Davis Health Systems, Sacramento, CA, USA.; Institute for Regenerative Cures, University of California Davis Health Systems, Sacramento, CA, USA.; Department of Neurology, University of California Davis Health Systems, Sacramento, CA, USA., Waldo JJ; Center for Interventional Genetics, University of California Davis Health Systems, Sacramento, CA, USA.; MIND Institute, University of California Davis Health Systems, Sacramento, CA, USA.; Stem Cell Program and Gene Therapy Center, University of California Davis Health Systems, Sacramento, CA, USA.; Institute for Regenerative Cures, University of California Davis Health Systems, Sacramento, CA, USA.; Department of Neurology, University of California Davis Health Systems, Sacramento, CA, USA., Vij PA; Center for Interventional Genetics, University of California Davis Health Systems, Sacramento, CA, USA.; MIND Institute, University of California Davis Health Systems, Sacramento, CA, USA.; Stem Cell Program and Gene Therapy Center, University of California Davis Health Systems, Sacramento, CA, USA.; Institute for Regenerative Cures, University of California Davis Health Systems, Sacramento, CA, USA.; Department of Neurology, University of California Davis Health Systems, Sacramento, CA, USA., Anguiano M; Center for Neuroscience, University of California Davis., Villegas IJ; Center for Interventional Genetics, University of California Davis Health Systems, Sacramento, CA, USA.; MIND Institute, University of California Davis Health Systems, Sacramento, CA, USA.; Stem Cell Program and Gene Therapy Center, University of California Davis Health Systems, Sacramento, CA, USA.; Institute for Regenerative Cures, University of California Davis Health Systems, Sacramento, CA, USA.; Department of Neurology, University of California Davis Health Systems, Sacramento, CA, USA., Du YX; Center for Interventional Genetics, University of California Davis Health Systems, Sacramento, CA, USA.; MIND Institute, University of California Davis Health Systems, Sacramento, CA, USA.; Stem Cell Program and Gene Therapy Center, University of California Davis Health Systems, Sacramento, CA, USA.; Institute for Regenerative Cures, University of California Davis Health Systems, Sacramento, CA, USA.; Department of Neurology, University of California Davis Health Systems, Sacramento, CA, USA., Nolta J; Stem Cell Program and Gene Therapy Center, University of California Davis Health Systems, Sacramento, CA, USA.; Institute for Regenerative Cures, University of California Davis Health Systems, Sacramento, CA, USA., Fink KD; Center for Interventional Genetics, University of California Davis Health Systems, Sacramento, CA, USA.; MIND Institute, University of California Davis Health Systems, Sacramento, CA, USA.; Stem Cell Program and Gene Therapy Center, University of California Davis Health Systems, Sacramento, CA, USA.; Institute for Regenerative Cures, University of California Davis Health Systems, Sacramento, CA, USA.; Department of Neurology, University of California Davis Health Systems, Sacramento, CA, USA.

Publikováno v: Stem cells (Dayton, Ohio) [Stem Cells] 2024 Oct 26. Date of Electronic Publication: 2024 Oct 26.

Transcriptional reprogramming restores UBE3A brain-wide and rescues behavioral phenotypes in an Angelman syndrome mouse model.

Autor: O'Geen H; Genome Center, UC Davis, Davis, CA, USA., Beitnere U; Genome Center, UC Davis, Davis, CA, USA., Garcia MS; Genome Center, UC Davis, Davis, CA, USA., Adhikari A; MIND Institute, UC Davis Health System, Sacramento, CA, USA; Department of Psychiatry and Behavioral Sciences, UC Davis Health System, Sacramento, CA, USA., Cameron DL; Neurology Department, Stem Cell Program and Gene Therapy Center, UC Davis Health System, Sacramento, CA, USA; MIND Institute, UC Davis Health System, Sacramento, CA, USA., Fenton TA; MIND Institute, UC Davis Health System, Sacramento, CA, USA; Department of Psychiatry and Behavioral Sciences, UC Davis Health System, Sacramento, CA, USA., Copping NA; MIND Institute, UC Davis Health System, Sacramento, CA, USA; Department of Psychiatry and Behavioral Sciences, UC Davis Health System, Sacramento, CA, USA., Deng P; Neurology Department, Stem Cell Program and Gene Therapy Center, UC Davis Health System, Sacramento, CA, USA; MIND Institute, UC Davis Health System, Sacramento, CA, USA., Lock S; Neurology Department, Stem Cell Program and Gene Therapy Center, UC Davis Health System, Sacramento, CA, USA; MIND Institute, UC Davis Health System, Sacramento, CA, USA., Halmai JANM; Neurology Department, Stem Cell Program and Gene Therapy Center, UC Davis Health System, Sacramento, CA, USA; MIND Institute, UC Davis Health System, Sacramento, CA, USA., Villegas IJ; Neurology Department, Stem Cell Program and Gene Therapy Center, UC Davis Health System, Sacramento, CA, USA; MIND Institute, UC Davis Health System, Sacramento, CA, USA., Liu J; Genome Editing and Novel Modalities (GENM), MilliporeSigma, St. Louis, MO, USA., Wang D; Genome Editing and Novel Modalities (GENM), MilliporeSigma, St. Louis, MO, USA., Fink KD; Neurology Department, Stem Cell Program and Gene Therapy Center, UC Davis Health System, Sacramento, CA, USA; MIND Institute, UC Davis Health System, Sacramento, CA, USA., Silverman JL; MIND Institute, UC Davis Health System, Sacramento, CA, USA; Department of Psychiatry and Behavioral Sciences, UC Davis Health System, Sacramento, CA, USA., Segal DJ; Genome Center, UC Davis, Davis, CA, USA; Department of Biochemistry and Molecular Medicine, UC Davis, Davis, CA, USA; MIND Institute, UC Davis Health System, Sacramento, CA, USA. Electronic address: djsegal@ucdavis.edu.

Publikováno v: Molecular therapy : the journal of the American Society of Gene Therapy [Mol Ther] 2023 Apr 05; Vol. 31 (4), pp. 1088-1105. Date of Electronic Publication: 2023 Jan 14.

An in vivo Cell-Based Delivery Platform for Zinc Finger Artificial Transcription Factors in Pre-clinical Animal Models.

Autor: Deng P; Department of Neurology, University of California Davis School of Medicine, Sacramento, CA, United States.; Stem Cell Program and Gene Therapy Center, University of California, Davis, Sacramento, CA, United States.; Department of Biochemistry and Molecular Medicine, Genome Center, University of California, Davis, Davis, CA, United States.; Department of Psychiatry and Behavioral Sciences, MIND Institute, University of California Davis School of Medicine, Sacramento, CA, United States., Halmai JANM; Department of Neurology, University of California Davis School of Medicine, Sacramento, CA, United States.; Stem Cell Program and Gene Therapy Center, University of California, Davis, Sacramento, CA, United States.; Department of Psychiatry and Behavioral Sciences, MIND Institute, University of California Davis School of Medicine, Sacramento, CA, United States., Beitnere U; Department of Biochemistry and Molecular Medicine, Genome Center, University of California, Davis, Davis, CA, United States., Cameron D; Department of Neurology, University of California Davis School of Medicine, Sacramento, CA, United States.; Stem Cell Program and Gene Therapy Center, University of California, Davis, Sacramento, CA, United States.; Department of Psychiatry and Behavioral Sciences, MIND Institute, University of California Davis School of Medicine, Sacramento, CA, United States., Martinez ML; Departments of Pediatrics and Cell Biology and Human Anatomy, School of Medicine, Gene Therapy Center, and California National Primate Research Center, University of California, Davis, Davis, CA, United States., Lee CC; Departments of Pediatrics and Cell Biology and Human Anatomy, School of Medicine, Gene Therapy Center, and California National Primate Research Center, University of California, Davis, Davis, CA, United States., Waldo JJ; Department of Neurology, University of California Davis School of Medicine, Sacramento, CA, United States.; Stem Cell Program and Gene Therapy Center, University of California, Davis, Sacramento, CA, United States.; Department of Psychiatry and Behavioral Sciences, MIND Institute, University of California Davis School of Medicine, Sacramento, CA, United States., Thongphanh K; Department of Neurology, University of California Davis School of Medicine, Sacramento, CA, United States.; Stem Cell Program and Gene Therapy Center, University of California, Davis, Sacramento, CA, United States., Adhikari A; Department of Psychiatry and Behavioral Sciences, MIND Institute, University of California Davis School of Medicine, Sacramento, CA, United States., Copping N; Department of Psychiatry and Behavioral Sciences, MIND Institute, University of California Davis School of Medicine, Sacramento, CA, United States., Petkova SP; Department of Psychiatry and Behavioral Sciences, MIND Institute, University of California Davis School of Medicine, Sacramento, CA, United States., Lee RD; Department of Psychiatry and Behavioral Sciences, MIND Institute, University of California Davis School of Medicine, Sacramento, CA, United States., Lock S; Department of Neurology, University of California Davis School of Medicine, Sacramento, CA, United States.; Stem Cell Program and Gene Therapy Center, University of California, Davis, Sacramento, CA, United States.; Department of Psychiatry and Behavioral Sciences, MIND Institute, University of California Davis School of Medicine, Sacramento, CA, United States., Palomares M; Department of Biochemistry and Molecular Medicine, Genome Center, University of California, Davis, Davis, CA, United States., O'Geen H; Department of Biochemistry and Molecular Medicine, Genome Center, University of California, Davis, Davis, CA, United States., Carter J; Department of Neurology, University of California Davis School of Medicine, Sacramento, CA, United States.; Stem Cell Program and Gene Therapy Center, University of California, Davis, Sacramento, CA, United States.; Department of Psychiatry and Behavioral Sciences, MIND Institute, University of California Davis School of Medicine, Sacramento, CA, United States., Gonzalez CE; Department of Neurology, University of California Davis School of Medicine, Sacramento, CA, United States.; Stem Cell Program and Gene Therapy Center, University of California, Davis, Sacramento, CA, United States.; Department of Psychiatry and Behavioral Sciences, MIND Institute, University of California Davis School of Medicine, Sacramento, CA, United States., Buchanan FKB; Department of Neurology, University of California Davis School of Medicine, Sacramento, CA, United States.; Stem Cell Program and Gene Therapy Center, University of California, Davis, Sacramento, CA, United States.; Department of Psychiatry and Behavioral Sciences, MIND Institute, University of California Davis School of Medicine, Sacramento, CA, United States., Anderson JD; Department of Otolaryngology, University of California, Davis, Davis, CA, United States., Fierro FA; Stem Cell Program and Gene Therapy Center, University of California, Davis, Sacramento, CA, United States., Nolta JA; Stem Cell Program and Gene Therapy Center, University of California, Davis, Sacramento, CA, United States., Tarantal AF; Departments of Pediatrics and Cell Biology and Human Anatomy, School of Medicine, Gene Therapy Center, and California National Primate Research Center, University of California, Davis, Davis, CA, United States., Silverman JL; Department of Psychiatry and Behavioral Sciences, MIND Institute, University of California Davis School of Medicine, Sacramento, CA, United States., Segal DJ; Department of Biochemistry and Molecular Medicine, Genome Center, University of California, Davis, Davis, CA, United States., Fink KD; Department of Neurology, University of California Davis School of Medicine, Sacramento, CA, United States.; Stem Cell Program and Gene Therapy Center, University of California, Davis, Sacramento, CA, United States.; Department of Psychiatry and Behavioral Sciences, MIND Institute, University of California Davis School of Medicine, Sacramento, CA, United States.

Publikováno v: Frontiers in molecular neuroscience [Front Mol Neurosci] 2022 Jan 27; Vol. 14, pp. 789913. Date of Electronic Publication: 2022 Jan 27 (Print Publication: 2021).

Artificial escape from XCI by DNA methylation editing of the CDKL5 gene.

Autor: Halmai JANM; Department of Neurology, University of California Davis School of Medicine, Sacramento, CA 95817, USA.; Stem Cell Program and Gene Therapy Center, University of California, Davis, Sacramento, CA, USA., Deng P; Department of Neurology, University of California Davis School of Medicine, Sacramento, CA 95817, USA.; Stem Cell Program and Gene Therapy Center, University of California, Davis, Sacramento, CA, USA.; Genome Center and Department of Biochemistry and Molecular Medicine, University of California, Davis, CA 95616, USA., Gonzalez CE; Department of Neurology, University of California Davis School of Medicine, Sacramento, CA 95817, USA.; Stem Cell Program and Gene Therapy Center, University of California, Davis, Sacramento, CA, USA., Coggins NB; Genome Center and Department of Biochemistry and Molecular Medicine, University of California, Davis, CA 95616, USA., Cameron D; Department of Neurology, University of California Davis School of Medicine, Sacramento, CA 95817, USA.; Stem Cell Program and Gene Therapy Center, University of California, Davis, Sacramento, CA, USA., Carter JL; Department of Neurology, University of California Davis School of Medicine, Sacramento, CA 95817, USA.; Stem Cell Program and Gene Therapy Center, University of California, Davis, Sacramento, CA, USA., Buchanan FKB; Department of Neurology, University of California Davis School of Medicine, Sacramento, CA 95817, USA.; Stem Cell Program and Gene Therapy Center, University of California, Davis, Sacramento, CA, USA., Waldo JJ; Department of Neurology, University of California Davis School of Medicine, Sacramento, CA 95817, USA.; Stem Cell Program and Gene Therapy Center, University of California, Davis, Sacramento, CA, USA., Lock SR; Stem Cell Program and Gene Therapy Center, University of California, Davis, Sacramento, CA, USA., Anderson JD; Department of Otolaryngology, University of California, Davis, Davis, CA, USA., O'Geen H; Genome Center and Department of Biochemistry and Molecular Medicine, University of California, Davis, CA 95616, USA., Segal DJ; Genome Center and Department of Biochemistry and Molecular Medicine, University of California, Davis, CA 95616, USA., Nolta J; Stem Cell Program and Gene Therapy Center, University of California, Davis, Sacramento, CA, USA., Fink KD; Department of Neurology, University of California Davis School of Medicine, Sacramento, CA 95817, USA.; Stem Cell Program and Gene Therapy Center, University of California, Davis, Sacramento, CA, USA.

Publikováno v: Nucleic acids research [Nucleic Acids Res] 2020 Mar 18; Vol. 48 (5), pp. 2372-2387.