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- [31] Non-invasive AAV9-mediated silencing of mutant ataxin-3 alleviates motor and neuropathological deficits in a transgenic mouse model of Machado-Joseph diseaseHUMAN GENE THERAPY, 2016, 27 (11) : A47 - A47Nobre, R. J.论文数: 0 引用数: 0 h-index: 0机构: Univ Coimbra, Ctr Neurosci & Cell Biol, Coimbra, Portugal Univ Coimbra, Inst Interdisciplinary Res, Coimbra, Portugal Univ Coimbra, Ctr Neurosci & Cell Biol, Coimbra, PortugalSaraiva, J.论文数: 0 引用数: 0 h-index: 0机构: Univ Coimbra, Ctr Neurosci & Cell Biol, Coimbra, Portugal Univ Coimbra, Ctr Neurosci & Cell Biol, Coimbra, PortugalFusco, C.论文数: 0 引用数: 0 h-index: 0机构: Univ Coimbra, Ctr Neurosci & Cell Biol, Coimbra, Portugal Univ Coimbra, Ctr Neurosci & Cell Biol, Coimbra, PortugalPaixao, S.论文数: 0 引用数: 0 h-index: 0机构: Univ Coimbra, Ctr Neurosci & Cell Biol, Coimbra, Portugal Univ Coimbra, Ctr Neurosci & Cell Biol, Coimbra, PortugalSantana, M.论文数: 0 引用数: 0 h-index: 0机构: Univ Coimbra, Ctr Neurosci & Cell Biol, Coimbra, Portugal Univ Coimbra, Inst Interdisciplinary Res, Coimbra, Portugal Univ Coimbra, Ctr Neurosci & Cell Biol, Coimbra, PortugalSena-Esteves, M.论文数: 0 引用数: 0 h-index: 0机构: Univ Massachusetts, Dept Neurol, Gene Therapy Ctr, Sch Med, Amherst, MA 01003 USA Univ Coimbra, Ctr Neurosci & Cell Biol, Coimbra, PortugalAlmeida, L. P.论文数: 0 引用数: 0 h-index: 0机构: Univ Coimbra, Ctr Neurosci & Cell Biol, Coimbra, Portugal Univ Coimbra, Fac Pharm, Coimbra, Portugal Univ Coimbra, Ctr Neurosci & Cell Biol, Coimbra, Portugal
- [32] CRISPR-Cas9-Mediated Correction of the 1.02 kb Common Deletion in CLN3 in Induced Pluripotent Stem Cells from Patients with Batten DiseaseCRISPR JOURNAL, 2018, 1 (01): : 75 - 87Burnight, Erin R.论文数: 0 引用数: 0 h-index: 0机构: Univ Iowa, Carver Coll Med, Inst Vis Res, 375 Newton Rd, Iowa City, IA 52242 USA Univ Iowa, Carver Coll Med, Dept Ophthalmol & Visual Sci, 375 Newton Rd, Iowa City, IA 52242 USA Univ Iowa, Carver Coll Med, Inst Vis Res, 375 Newton Rd, Iowa City, IA 52242 USABohrer, Laura R.论文数: 0 引用数: 0 h-index: 0机构: Univ Iowa, Carver Coll Med, Inst Vis Res, 375 Newton Rd, Iowa City, IA 52242 USA Univ Iowa, Carver Coll Med, Dept Ophthalmol & Visual Sci, 375 Newton Rd, Iowa City, IA 52242 USA Univ Iowa, Carver Coll Med, Inst Vis Res, 375 Newton Rd, Iowa City, IA 52242 USAGiacalone, Joseph C.论文数: 0 引用数: 0 h-index: 0机构: Univ Iowa, Carver Coll Med, Inst Vis Res, 375 Newton Rd, Iowa City, IA 52242 USA Univ Iowa, Carver Coll Med, Dept Ophthalmol & Visual Sci, 375 Newton Rd, Iowa City, IA 52242 USA Univ Iowa, Carver Coll Med, Inst Vis Res, 375 Newton Rd, Iowa City, IA 52242 USAKlaahsen, Darcey L.论文数: 0 引用数: 0 h-index: 0机构: Univ Iowa, Carver Coll Med, Inst Vis Res, 375 Newton Rd, Iowa City, IA 52242 USA Univ Iowa, Carver Coll Med, Dept Ophthalmol & Visual Sci, 375 Newton Rd, Iowa City, IA 52242 USA Univ Iowa, Carver Coll Med, Inst Vis Res, 375 Newton Rd, Iowa City, IA 52242 USADaggett, Heather T.论文数: 0 引用数: 0 h-index: 0机构: Univ Iowa, Carver Coll Med, Inst Vis Res, 375 Newton Rd, Iowa City, IA 52242 USA Univ Iowa, Carver Coll Med, Dept Ophthalmol & Visual Sci, 375 Newton Rd, Iowa City, IA 52242 USA Univ Iowa, Carver Coll Med, Inst Vis Res, 375 Newton Rd, Iowa City, IA 52242 USAEast, Jade S.论文数: 0 引用数: 0 h-index: 0机构: Univ Iowa, Carver Coll Med, Inst Vis Res, 375 Newton Rd, Iowa City, IA 52242 USA Univ Iowa, Carver Coll Med, Dept Ophthalmol & Visual Sci, 375 Newton Rd, Iowa City, IA 52242 USA Univ Iowa, Carver Coll Med, Inst Vis Res, 375 Newton Rd, Iowa City, IA 52242 USAMadumba, Robert A.论文数: 0 引用数: 0 h-index: 0机构: Univ Iowa, Carver Coll Med, Inst Vis Res, 375 Newton Rd, Iowa City, IA 52242 USA Univ Iowa, Carver Coll Med, Dept Ophthalmol & Visual Sci, 375 Newton Rd, Iowa City, IA 52242 USA Univ Iowa, Carver Coll Med, Inst Vis Res, 375 Newton Rd, Iowa City, IA 52242 USAWorthington, Kristan S.论文数: 0 引用数: 0 h-index: 0机构: Univ Iowa, Carver Coll Med, Inst Vis Res, 375 Newton Rd, Iowa City, IA 52242 USA Univ Iowa, Dept Biomed Engn, Iowa City, IA 52242 USA Univ Iowa, Carver Coll Med, Inst Vis Res, 375 Newton Rd, Iowa City, IA 52242 USAMullins, Robert F.论文数: 0 引用数: 0 h-index: 0机构: Univ Iowa, Carver Coll Med, Inst Vis Res, 375 Newton Rd, Iowa City, IA 52242 USA Univ Iowa, Carver Coll Med, Dept Ophthalmol & Visual Sci, 375 Newton Rd, Iowa City, IA 52242 USA Univ Iowa, Carver Coll Med, Inst Vis Res, 375 Newton Rd, Iowa City, IA 52242 USAStone, Edwin M.论文数: 0 引用数: 0 h-index: 0机构: Univ Iowa, Carver Coll Med, Inst Vis Res, 375 Newton Rd, Iowa City, IA 52242 USA Univ Iowa, Carver Coll Med, Dept Ophthalmol & Visual Sci, 375 Newton Rd, Iowa City, IA 52242 USA Univ Iowa, Carver Coll Med, Inst Vis Res, 375 Newton Rd, Iowa City, IA 52242 USATucker, Budd A.论文数: 0 引用数: 0 h-index: 0机构: Univ Iowa, Carver Coll Med, Inst Vis Res, 375 Newton Rd, Iowa City, IA 52242 USA Univ Iowa, Carver Coll Med, Dept Ophthalmol & Visual Sci, 375 Newton Rd, Iowa City, IA 52242 USA Univ Iowa, Carver Coll Med, Inst Vis Res, 375 Newton Rd, Iowa City, IA 52242 USAWiley, Luke A.论文数: 0 引用数: 0 h-index: 0机构: Univ Iowa, Carver Coll Med, Inst Vis Res, 375 Newton Rd, Iowa City, IA 52242 USA Univ Iowa, Carver Coll Med, Dept Ophthalmol & Visual Sci, 375 Newton Rd, Iowa City, IA 52242 USA Univ Iowa, Carver Coll Med, Inst Vis Res, 375 Newton Rd, Iowa City, IA 52242 USA
- [33] In Vivo Modelling of ATP1A3 G316S-Induced Ataxia in C-elegans Using CRISPR/Cas9-Mediated Homologous Recombination Reveals Dominant Loss of Function DefectsPLOS ONE, 2016, 11 (12):Sorkac, Altar论文数: 0 引用数: 0 h-index: 0机构: Brown Univ, Dept Neurosci, Providence, RI 02912 USA Brown Univ, Dept Neurosci, Providence, RI 02912 USAAlcantara, Ivan C.论文数: 0 引用数: 0 h-index: 0机构: Brown Univ, Dept Neurosci, Providence, RI 02912 USA Brown Univ, Dept Neurosci, Providence, RI 02912 USAHart, Anne C.论文数: 0 引用数: 0 h-index: 0机构: Brown Univ, Dept Neurosci, Providence, RI 02912 USA Brown Univ, Dept Neurosci, Providence, RI 02912 USA
- [34] CRISPR-Cas9-Mediated Correction of SLC12A3 Gene Mutation Rescues the Gitelman's Disease Phenotype in a Patient-Derived Kidney Organoid SystemINTERNATIONAL JOURNAL OF MOLECULAR SCIENCES, 2023, 24 (03)论文数: 引用数: h-index:机构:Fang, Xianying论文数: 0 引用数: 0 h-index: 0机构: Catholic Univ Korea, Transplantat Res Ctr Coll Med, Seoul 06591, South Korea Catholic Univ Korea, Transplantat Res Ctr Coll Med, Seoul 06591, South KoreaCui, Sheng论文数: 0 引用数: 0 h-index: 0机构: Catholic Univ Korea, Transplantat Res Ctr Coll Med, Seoul 06591, South Korea Catholic Univ Korea, Transplantat Res Ctr Coll Med, Seoul 06591, South KoreaLee, Hanbi论文数: 0 引用数: 0 h-index: 0机构: Catholic Univ Korea, Transplantat Res Ctr Coll Med, Seoul 06591, South Korea Catholic Univ Korea, Seoul St Marys Hosp, Coll Med, Dept Internal Med,Div Nephrol, Seoul St, Seoul 06591, South Korea Catholic Univ Korea, Transplantat Res Ctr Coll Med, Seoul 06591, South KoreaShin, Yoo Jin论文数: 0 引用数: 0 h-index: 0机构: Catholic Univ Korea, Transplantat Res Ctr Coll Med, Seoul 06591, South Korea Catholic Univ Korea, Transplantat Res Ctr Coll Med, Seoul 06591, South KoreaKo, Eun Jeong论文数: 0 引用数: 0 h-index: 0机构: Catholic Univ Korea, Transplantat Res Ctr Coll Med, Seoul 06591, South Korea Catholic Univ Korea, Seoul St Marys Hosp, Coll Med, Dept Internal Med,Div Nephrol, Seoul St, Seoul 06591, South Korea Catholic Univ Korea, Transplantat Res Ctr Coll Med, Seoul 06591, South KoreaLee, Kang In论文数: 0 引用数: 0 h-index: 0机构: Tool Gen Inc, R&D Ctr, Seoul 06591, South Korea Catholic Univ Korea, Transplantat Res Ctr Coll Med, Seoul 06591, South KoreaLee, Jae Young论文数: 0 引用数: 0 h-index: 0机构: Tool Gen Inc, R&D Ctr, Seoul 06591, South Korea Catholic Univ Korea, Transplantat Res Ctr Coll Med, Seoul 06591, South Korea论文数: 引用数: h-index:机构:Yang, Chul Woo论文数: 0 引用数: 0 h-index: 0机构: Catholic Univ Korea, Transplantat Res Ctr Coll Med, Seoul 06591, South Korea Catholic Univ Korea, Seoul St Marys Hosp, Coll Med, Dept Internal Med,Div Nephrol, Seoul St, Seoul 06591, South Korea Catholic Univ Korea, Transplantat Res Ctr Coll Med, Seoul 06591, South Korea