AAV9-Mediated Intrastriatal Delivery of GNAO1 Reduces Hyperlocomotion in Gnao1 Heterozygous R209H Mutant Mice.
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| Title: | AAV9-Mediated Intrastriatal Delivery of GNAO1 Reduces Hyperlocomotion in Gnao1 Heterozygous R209H Mutant Mice. |
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| Authors: | Roy AJ; Department of Pharmacology and Toxicology (A.J.R., J.R.L., R.R.N.), Department of Microbiology and Molecular Genetics (A.J.R.), and Nicholas V. Perricone, M.D., Division of Dermatology, Department of Medicine (R.R.N.), Michigan State University, East Lansing, Michigan; Department of Translational Neuroscience (J.R.P., A.C.S., C.J.K., C.E.S.), Michigan State University, Grand Rapids, Michigan; Hauenstein Neuroscience Center, Mercy Health Saint Mary's, Grand Rapids, Michigan (C.E.S.); and Horae Gene Therapy Center and The Li Weibo Institute for Rare Diseases Research (Z.-T.D.O., T.M., A.R.B., M.S.-E.) and Department of Neurology (Z.-T.D.O., T.M., A.R.B., M.S.-E.), UMass Chan Medical School, Worcester, Massachusetts., Leipprandt JR; Department of Pharmacology and Toxicology (A.J.R., J.R.L., R.R.N.), Department of Microbiology and Molecular Genetics (A.J.R.), and Nicholas V. Perricone, M.D., Division of Dermatology, Department of Medicine (R.R.N.), Michigan State University, East Lansing, Michigan; Department of Translational Neuroscience (J.R.P., A.C.S., C.J.K., C.E.S.), Michigan State University, Grand Rapids, Michigan; Hauenstein Neuroscience Center, Mercy Health Saint Mary's, Grand Rapids, Michigan (C.E.S.); and Horae Gene Therapy Center and The Li Weibo Institute for Rare Diseases Research (Z.-T.D.O., T.M., A.R.B., M.S.-E.) and Department of Neurology (Z.-T.D.O., T.M., A.R.B., M.S.-E.), UMass Chan Medical School, Worcester, Massachusetts., Patterson JR; Department of Pharmacology and Toxicology (A.J.R., J.R.L., R.R.N.), Department of Microbiology and Molecular Genetics (A.J.R.), and Nicholas V. Perricone, M.D., Division of Dermatology, Department of Medicine (R.R.N.), Michigan State University, East Lansing, Michigan; Department of Translational Neuroscience (J.R.P., A.C.S., C.J.K., C.E.S.), Michigan State University, Grand Rapids, Michigan; Hauenstein Neuroscience Center, Mercy Health Saint Mary's, Grand Rapids, Michigan (C.E.S.); and Horae Gene Therapy Center and The Li Weibo Institute for Rare Diseases Research (Z.-T.D.O., T.M., A.R.B., M.S.-E.) and Department of Neurology (Z.-T.D.O., T.M., A.R.B., M.S.-E.), UMass Chan Medical School, Worcester, Massachusetts., Stoll AC; Department of Pharmacology and Toxicology (A.J.R., J.R.L., R.R.N.), Department of Microbiology and Molecular Genetics (A.J.R.), and Nicholas V. Perricone, M.D., Division of Dermatology, Department of Medicine (R.R.N.), Michigan State University, East Lansing, Michigan; Department of Translational Neuroscience (J.R.P., A.C.S., C.J.K., C.E.S.), Michigan State University, Grand Rapids, Michigan; Hauenstein Neuroscience Center, Mercy Health Saint Mary's, Grand Rapids, Michigan (C.E.S.); and Horae Gene Therapy Center and The Li Weibo Institute for Rare Diseases Research (Z.-T.D.O., T.M., A.R.B., M.S.-E.) and Department of Neurology (Z.-T.D.O., T.M., A.R.B., M.S.-E.), UMass Chan Medical School, Worcester, Massachusetts., Kemp CJ; Department of Pharmacology and Toxicology (A.J.R., J.R.L., R.R.N.), Department of Microbiology and Molecular Genetics (A.J.R.), and Nicholas V. Perricone, M.D., Division of Dermatology, Department of Medicine (R.R.N.), Michigan State University, East Lansing, Michigan; Department of Translational Neuroscience (J.R.P., A.C.S., C.J.K., C.E.S.), Michigan State University, Grand Rapids, Michigan; Hauenstein Neuroscience Center, Mercy Health Saint Mary's, Grand Rapids, Michigan (C.E.S.); and Horae Gene Therapy Center and The Li Weibo Institute for Rare Diseases Research (Z.-T.D.O., T.M., A.R.B., M.S.-E.) and Department of Neurology (Z.-T.D.O., T.M., A.R.B., M.S.-E.), UMass Chan Medical School, Worcester, Massachusetts., Oula ZD; Department of Pharmacology and Toxicology (A.J.R., J.R.L., R.R.N.), Department of Microbiology and Molecular Genetics (A.J.R.), and Nicholas V. Perricone, M.D., Division of Dermatology, Department of Medicine (R.R.N.), Michigan State University, East Lansing, Michigan; Department of Translational Neuroscience (J.R.P., A.C.S., C.J.K., C.E.S.), Michigan State University, Grand Rapids, Michigan; Hauenstein Neuroscience Center, Mercy Health Saint Mary's, Grand Rapids, Michigan (C.E.S.); and Horae Gene Therapy Center and The Li Weibo Institute for Rare Diseases Research (Z.-T.D.O., T.M., A.R.B., M.S.-E.) and Department of Neurology (Z.-T.D.O., T.M., A.R.B., M.S.-E.), UMass Chan Medical School, Worcester, Massachusetts., Mola T; Department of Pharmacology and Toxicology (A.J.R., J.R.L., R.R.N.), Department of Microbiology and Molecular Genetics (A.J.R.), and Nicholas V. Perricone, M.D., Division of Dermatology, Department of Medicine (R.R.N.), Michigan State University, East Lansing, Michigan; Department of Translational Neuroscience (J.R.P., A.C.S., C.J.K., C.E.S.), Michigan State University, Grand Rapids, Michigan; Hauenstein Neuroscience Center, Mercy Health Saint Mary's, Grand Rapids, Michigan (C.E.S.); and Horae Gene Therapy Center and The Li Weibo Institute for Rare Diseases Research (Z.-T.D.O., T.M., A.R.B., M.S.-E.) and Department of Neurology (Z.-T.D.O., T.M., A.R.B., M.S.-E.), UMass Chan Medical School, Worcester, Massachusetts., Batista AR; Department of Pharmacology and Toxicology (A.J.R., J.R.L., R.R.N.), Department of Microbiology and Molecular Genetics (A.J.R.), and Nicholas V. Perricone, M.D., Division of Dermatology, Department of Medicine (R.R.N.), Michigan State University, East Lansing, Michigan; Department of Translational Neuroscience (J.R.P., A.C.S., C.J.K., C.E.S.), Michigan State University, Grand Rapids, Michigan; Hauenstein Neuroscience Center, Mercy Health Saint Mary's, Grand Rapids, Michigan (C.E.S.); and Horae Gene Therapy Center and The Li Weibo Institute for Rare Diseases Research (Z.-T.D.O., T.M., A.R.B., M.S.-E.) and Department of Neurology (Z.-T.D.O., T.M., A.R.B., M.S.-E.), UMass Chan Medical School, Worcester, Massachusetts., Sortwell CE; Department of Pharmacology and Toxicology (A.J.R., J.R.L., R.R.N.), Department of Microbiology and Molecular Genetics (A.J.R.), and Nicholas V. Perricone, M.D., Division of Dermatology, Department of Medicine (R.R.N.), Michigan State University, East Lansing, Michigan; Department of Translational Neuroscience (J.R.P., A.C.S., C.J.K., C.E.S.), Michigan State University, Grand Rapids, Michigan; Hauenstein Neuroscience Center, Mercy Health Saint Mary's, Grand Rapids, Michigan (C.E.S.); and Horae Gene Therapy Center and The Li Weibo Institute for Rare Diseases Research (Z.-T.D.O., T.M., A.R.B., M.S.-E.) and Department of Neurology (Z.-T.D.O., T.M., A.R.B., M.S.-E.), UMass Chan Medical School, Worcester, Massachusetts., Sena-Esteves M; Department of Pharmacology and Toxicology (A.J.R., J.R.L., R.R.N.), Department of Microbiology and Molecular Genetics (A.J.R.), and Nicholas V. Perricone, M.D., Division of Dermatology, Department of Medicine (R.R.N.), Michigan State University, East Lansing, Michigan; Department of Translational Neuroscience (J.R.P., A.C.S., C.J.K., C.E.S.), Michigan State University, Grand Rapids, Michigan; Hauenstein Neuroscience Center, Mercy Health Saint Mary's, Grand Rapids, Michigan (C.E.S.); and Horae Gene Therapy Center and The Li Weibo Institute for Rare Diseases Research (Z.-T.D.O., T.M., A.R.B., M.S.-E.) and Department of Neurology (Z.-T.D.O., T.M., A.R.B., M.S.-E.), UMass Chan Medical School, Worcester, Massachusetts., Neubig RR; Department of Pharmacology and Toxicology (A.J.R., J.R.L., R.R.N.), Department of Microbiology and Molecular Genetics (A.J.R.), and Nicholas V. Perricone, M.D., Division of Dermatology, Department of Medicine (R.R.N.), Michigan State University, East Lansing, Michigan; Department of Translational Neuroscience (J.R.P., A.C.S., C.J.K., C.E.S.), Michigan State University, Grand Rapids, Michigan; Hauenstein Neuroscience Center, Mercy Health Saint Mary's, Grand Rapids, Michigan (C.E.S.); and Horae Gene Therapy Center and The Li Weibo Institute for Rare Diseases Research (Z.-T.D.O., T.M., A.R.B., M.S.-E.) and Department of Neurology (Z.-T.D.O., T.M., A.R.B., M.S.-E.), UMass Chan Medical School, Worcester, Massachusetts RNeubig@msu.edu. |
| Source: | The Journal of pharmacology and experimental therapeutics [J Pharmacol Exp Ther] 2024 Jul 18; Vol. 390 (2), pp. 250-259. Date of Electronic Publication: 2024 Jul 18. |
| Publication Type: | Journal Article; Research Support, Non-U.S. Gov't |
| Journal Info: | Publisher: Elsevier Country of Publication: Netherlands NLM ID: 0376362 Publication Model: Electronic Cited Medium: Internet ISSN: 1521-0103 (Electronic) Linking ISSN: 00223565 NLM ISO Abbreviation: J Pharmacol Exp Ther Subsets: MEDLINE |
| Database: | MEDLINE Ultimate |
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