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Academic Journal

miRNA-Mediated Knockdown of ATXN3 Alleviates Molecular Disease Hallmarks in a Mouse Model for Spinocerebellar Ataxia Type 3.

  • Authors : Nobre RJ; Center for Neuroscience and Cell Biology (CNC), Molecular Therapy of Brain Disorders Group, University of Coimbra, Coimbra, Portugal.; Center for Innovative Biomedicine and Biotechnology (CIBB), Vectors, Gene and Cell Therapy Group, University of Coimbra, Coimbra, Portugal.

Subjects: Ataxin-3*/Ataxin-3*/Ataxin-3*/genetics ; Machado-Joseph Disease*/Machado-Joseph Disease*/Machado-Joseph Disease*/therapy ; MicroRNAs*/MicroRNAs*/MicroRNAs*/genetics

  • Source: Nucleic acid therapeutics [Nucleic Acid Ther] 2022 Jun; Vol. 32 (3), pp. 194-205. Date of Electronic Publication: 2021 Dec 07.Publisher: Mary Ann Liebert, Inc Country of Publication: United States NLM ID: 101562758 Publication Model: Print-Electronic

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Academic Journal

CRISPR/Cas9-Targeted Deletion of Polyglutamine in Spinocerebellar Ataxia Type 3-Derived Induced Pluripotent Stem Cells.

  • Authors : Ouyang S; Key Laboratory for Major Obstetric Diseases of Guangdong Province, Key Laboratory of Reproduction and Genetics of Guangdong Higher Education Institutes, The Third Affiliated Hospital of Guangzhou Medical University , Guangzhou City, People's Republic of China .; Xie Y

Subjects: CRISPR-Cas Systems* ; Gene Deletion*; Ataxin-3/Ataxin-3/Ataxin-3/*genetics

  • Source: Stem cells and development [Stem Cells Dev] 2018 Jun 01; Vol. 27 (11), pp. 756-770. Date of Electronic Publication: 2018 May 18.Publisher: Mary Ann Liebert, Inc Country of Publication: United States NLM ID: 101197107 Publication Model: Print-Electronic

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