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- [41] Antisense oligomer mediated retention of SMN2 intron and exon 7 leads to a novel SMN transcript and increased functional SMN protein in Spinal Muscular Atrophy fibroblastsNEUROMUSCULAR DISORDERS, 2011, 21 (9-10) : 685 - 685Mitrpant, C.论文数: 0 引用数: 0 h-index: 0机构: Univ Western Australia, Ctr Neuromuscular & Neurol Disorders, Perth, WA 6009, Australia Univ Western Australia, Ctr Neuromuscular & Neurol Disorders, Perth, WA 6009, AustraliaPrice, L.论文数: 0 引用数: 0 h-index: 0机构: Univ Western Australia, Ctr Neuromuscular & Neurol Disorders, Perth, WA 6009, Australia Univ Western Australia, Ctr Neuromuscular & Neurol Disorders, Perth, WA 6009, AustraliaJohnsen, R. D.论文数: 0 引用数: 0 h-index: 0机构: Univ Western Australia, Ctr Neuromuscular & Neurol Disorders, Perth, WA 6009, Australia Univ Western Australia, Ctr Neuromuscular & Neurol Disorders, Perth, WA 6009, AustraliaBurghes, A. H.论文数: 0 引用数: 0 h-index: 0机构: Ohio State Univ, Dept Mol & Cellular Biochem, Columbus, OH 43210 USA Univ Western Australia, Ctr Neuromuscular & Neurol Disorders, Perth, WA 6009, AustraliaFletcher, S.论文数: 0 引用数: 0 h-index: 0机构: Univ Western Australia, Ctr Neuromuscular & Neurol Disorders, Perth, WA 6009, Australia Univ Western Australia, Ctr Neuromuscular & Neurol Disorders, Perth, WA 6009, AustraliaWilton, S. D.论文数: 0 引用数: 0 h-index: 0机构: Univ Western Australia, Ctr Neuromuscular & Neurol Disorders, Perth, WA 6009, Australia Univ Western Australia, Ctr Neuromuscular & Neurol Disorders, Perth, WA 6009, Australia
- [42] Optimization of base editors for the functional correction of SMN2 as a treatment for spinal muscular atrophyNATURE BIOMEDICAL ENGINEERING, 2024, 8 (02) : 118 - +Alves, Christiano R. R.论文数: 0 引用数: 0 h-index: 0机构: Massachusetts Gen Hosp, Ctr Genom Med, Boston, MA 02114 USA Massachusetts Gen Hosp, Dept Neurol, Boston, MA 02114 USA Harvard Med Sch, Dept Neurol, Boston, MA 02115 USA Massachusetts Gen Hosp, Ctr Genom Med, Boston, MA 02114 USAHa, Leillani L.论文数: 0 引用数: 0 h-index: 0机构: Massachusetts Gen Hosp, Ctr Genom Med, Boston, MA 02114 USA Massachusetts Gen Hosp, Dept Pathol, Boston, MA 02114 USA Massachusetts Gen Hosp, Ctr Genom Med, Boston, MA 02114 USAYaworski, Rebecca论文数: 0 引用数: 0 h-index: 0机构: Ottawa Hosp Res Inst, Ottawa, ON, Canada Univ Ottawa, Dept Cellular & Mol Med, Ottawa, ON, Canada Univ Ottawa, Ctr Neuromuscular Dis, Ottawa, ON, Canada Massachusetts Gen Hosp, Ctr Genom Med, Boston, MA 02114 USASutton, Emma R.论文数: 0 引用数: 0 h-index: 0机构: Ottawa Hosp Res Inst, Ottawa, ON, Canada Univ Ottawa, Dept Cellular & Mol Med, Ottawa, ON, Canada Univ Ottawa, Ctr Neuromuscular Dis, Ottawa, ON, Canada Massachusetts Gen Hosp, Ctr Genom Med, Boston, MA 02114 USALazzarotto, Cicera R.论文数: 0 引用数: 0 h-index: 0机构: St Jude Childrens Res Hosp, Dept Hematol, Memphis, TN USA Massachusetts Gen Hosp, Ctr Genom Med, Boston, MA 02114 USAChristie, Kathleen A.论文数: 0 引用数: 0 h-index: 0机构: Massachusetts Gen Hosp, Ctr Genom Med, Boston, MA 02114 USA Massachusetts Gen Hosp, Dept Pathol, Boston, MA 02114 USA Harvard Med Sch, Dept Pathol, Boston, MA 02115 USA Massachusetts Gen Hosp, Ctr Genom Med, Boston, MA 02114 USAReilly, Aoife论文数: 0 引用数: 0 h-index: 0机构: Ottawa Hosp Res Inst, Ottawa, ON, Canada Univ Ottawa, Dept Cellular & Mol Med, Ottawa, ON, Canada Univ Ottawa, Ctr Neuromuscular Dis, Ottawa, ON, Canada Massachusetts Gen Hosp, Ctr Genom Med, Boston, MA 02114 USABeauvais, Ariane论文数: 0 引用数: 0 h-index: 0机构: Ottawa Hosp Res Inst, Ottawa, ON, Canada Univ Ottawa, Dept Cellular & Mol Med, Ottawa, ON, Canada Univ Ottawa, Ctr Neuromuscular Dis, Ottawa, ON, Canada Massachusetts Gen Hosp, Ctr Genom Med, Boston, MA 02114 USADoll, Roman M.论文数: 0 引用数: 0 h-index: 0机构: Massachusetts Gen Hosp, Ctr Genom Med, Boston, MA 02114 USA Massachusetts Gen Hosp, Dept Pathol, Boston, MA 02114 USA Heidelberg Univ, Mol Biosci Canc Biol Program, Heidelberg, Germany German Canc Res Ctr, Heidelberg, Germany Massachusetts Gen Hosp, Ctr Genom Med, Boston, MA 02114 USAde la Cruz, Demitri论文数: 0 引用数: 0 h-index: 0机构: Massachusetts Gen Hosp, Dept Neurol, Mol Neurogenet Unit, Charlestown, MA USA Harvard Med Sch, Program Neurosci, Boston, MA USA Massachusetts Gen Hosp, Ctr Genom Med, Boston, MA 02114 USAMaguire, Casey A.论文数: 0 引用数: 0 h-index: 0机构: Massachusetts Gen Hosp, Dept Neurol, Mol Neurogenet Unit, Charlestown, MA USA Harvard Med Sch, Program Neurosci, Boston, MA USA Massachusetts Gen Hosp, Ctr Genom Med, Boston, MA 02114 USASwoboda, Kathryn J.论文数: 0 引用数: 0 h-index: 0机构: Massachusetts Gen Hosp, Ctr Genom Med, Boston, MA 02114 USA Massachusetts Gen Hosp, Dept Neurol, Boston, MA 02114 USA Harvard Med Sch, Dept Neurol, Boston, MA 02115 USA Massachusetts Gen Hosp, Ctr Genom Med, Boston, MA 02114 USATsai, Shengdar Q.论文数: 0 引用数: 0 h-index: 0机构: St Jude Childrens Res Hosp, Dept Hematol, Memphis, TN USA Massachusetts Gen Hosp, Ctr Genom Med, Boston, MA 02114 USAKothary, Rashmi论文数: 0 引用数: 0 h-index: 0机构: Ottawa Hosp Res Inst, Ottawa, ON, Canada Univ Ottawa, Dept Cellular & Mol Med, Ottawa, ON, Canada Univ Ottawa, Ctr Neuromuscular Dis, Ottawa, ON, Canada Univ Ottawa, Dept Med, Ottawa, ON, Canada Massachusetts Gen Hosp, Ctr Genom Med, Boston, MA 02114 USAKleinstiver, Benjamin P.论文数: 0 引用数: 0 h-index: 0机构: Massachusetts Gen Hosp, Ctr Genom Med, Boston, MA 02114 USA Massachusetts Gen Hosp, Dept Pathol, Boston, MA 02114 USA Harvard Med Sch, Dept Pathol, Boston, MA 02115 USA Massachusetts Gen Hosp, Ctr Genom Med, Boston, MA 02114 USA
- [43] Targeting RNA structure in SMN2 reverses spinal muscular atrophy molecular phenotypesNATURE COMMUNICATIONS, 2018, 9Garcia-Lopez, Amparo论文数: 0 引用数: 0 h-index: 0机构: Univ Lausanne, Sch Pharmaceut Sci, Pharmaceut Biochem Grp, Rue Michel Servet 1, CH-1211 Geneva, Switzerland Univ Geneva, Rue Michel Servet 1, CH-1211 Geneva, Switzerland Univ Lausanne, Sch Pharmaceut Sci, Pharmaceut Biochem Grp, Rue Michel Servet 1, CH-1211 Geneva, SwitzerlandTessaro, Francesca论文数: 0 引用数: 0 h-index: 0机构: Univ Lausanne, Sch Pharmaceut Sci, Pharmaceut Biochem Grp, Rue Michel Servet 1, CH-1211 Geneva, Switzerland Univ Geneva, Rue Michel Servet 1, CH-1211 Geneva, Switzerland Univ Lausanne, Sch Pharmaceut Sci, Pharmaceut Biochem Grp, Rue Michel Servet 1, CH-1211 Geneva, SwitzerlandJonker, Hendrik R. A.论文数: 0 引用数: 0 h-index: 0机构: Goethe Univ Frankfurt, Ctr Biomol Magnet Resonance BMRZ, Inst Organ Chem & Chem Biol, Max von Laue Str 7, D-60438 Frankfurt, Germany Univ Lausanne, Sch Pharmaceut Sci, Pharmaceut Biochem Grp, Rue Michel Servet 1, CH-1211 Geneva, Switzerland论文数: 引用数: h-index:机构:论文数: 引用数: h-index:机构:论文数: 引用数: h-index:机构:Berntenis, Nikolaos论文数: 0 引用数: 0 h-index: 0机构: F Hoffmann La Roche, Roche Innovat Ctr Basel, Pharmaceut Res & Early Dev, Grenzacherstr 124, CH-4070 Basel, Switzerland Univ Lausanne, Sch Pharmaceut Sci, Pharmaceut Biochem Grp, Rue Michel Servet 1, CH-1211 Geneva, SwitzerlandSchmucki, Roland论文数: 0 引用数: 0 h-index: 0机构: F Hoffmann La Roche, Roche Innovat Ctr Basel, Pharmaceut Res & Early Dev, Grenzacherstr 124, CH-4070 Basel, Switzerland Univ Lausanne, Sch Pharmaceut Sci, Pharmaceut Biochem Grp, Rue Michel Servet 1, CH-1211 Geneva, SwitzerlandHatje, Klas论文数: 0 引用数: 0 h-index: 0机构: F Hoffmann La Roche, Roche Innovat Ctr Basel, Pharmaceut Res & Early Dev, Grenzacherstr 124, CH-4070 Basel, Switzerland Univ Lausanne, Sch Pharmaceut Sci, Pharmaceut Biochem Grp, Rue Michel Servet 1, CH-1211 Geneva, SwitzerlandPetermann, Olivier论文数: 0 引用数: 0 h-index: 0机构: Univ Lausanne, Sch Pharmaceut Sci, Pharmaceut Biochem Grp, Rue Michel Servet 1, CH-1211 Geneva, Switzerland Univ Geneva, Rue Michel Servet 1, CH-1211 Geneva, Switzerland Univ Lausanne, Sch Pharmaceut Sci, Pharmaceut Biochem Grp, Rue Michel Servet 1, CH-1211 Geneva, SwitzerlandChiriano, Gianpaolo论文数: 0 引用数: 0 h-index: 0机构: Univ Lausanne, Sch Pharmaceut Sci, Pharmaceut Biochem Grp, Rue Michel Servet 1, CH-1211 Geneva, Switzerland Univ Geneva, Rue Michel Servet 1, CH-1211 Geneva, Switzerland Univ Lausanne, Sch Pharmaceut Sci, Pharmaceut Biochem Grp, Rue Michel Servet 1, CH-1211 Geneva, SwitzerlandPerozzo, Remo论文数: 0 引用数: 0 h-index: 0机构: Univ Lausanne, Sch Pharmaceut Sci, Pharmaceut Biochem Grp, Rue Michel Servet 1, CH-1211 Geneva, Switzerland Univ Geneva, Rue Michel Servet 1, CH-1211 Geneva, Switzerland Univ Lausanne, Sch Pharmaceut Sci, Pharmaceut Biochem Grp, Rue Michel Servet 1, CH-1211 Geneva, SwitzerlandSciarra, Daniel论文数: 0 引用数: 0 h-index: 0机构: Univ Lausanne, Sch Pharmaceut Sci, Pharmaceut Biochem Grp, Rue Michel Servet 1, CH-1211 Geneva, Switzerland Univ Geneva, Rue Michel Servet 1, CH-1211 Geneva, Switzerland Univ Lausanne, Sch Pharmaceut Sci, Pharmaceut Biochem Grp, Rue Michel Servet 1, CH-1211 Geneva, Switzerland论文数: 引用数: h-index:机构:Faustino, Ignacio论文数: 0 引用数: 0 h-index: 0机构: BIST, IRB, Joint BSC IRB Res Program Computat Biol, Baldiri Reixac 10, Barcelona 08028, Spain Univ Lausanne, Sch Pharmaceut Sci, Pharmaceut Biochem Grp, Rue Michel Servet 1, CH-1211 Geneva, SwitzerlandFournet, Guy论文数: 0 引用数: 0 h-index: 0机构: Univ Claude Bernard Lyon 1, UMR CNRS 5246, ICBMS, 43 Bd 11 Novembre 1918, F-69622 Villeurbanne, France Univ Lausanne, Sch Pharmaceut Sci, Pharmaceut Biochem Grp, Rue Michel Servet 1, CH-1211 Geneva, SwitzerlandOrozco, Modesto论文数: 0 引用数: 0 h-index: 0机构: BIST, IRB, Joint BSC IRB Res Program Computat Biol, Baldiri Reixac 10, Barcelona 08028, Spain Univ Lausanne, Sch Pharmaceut Sci, Pharmaceut Biochem Grp, Rue Michel Servet 1, CH-1211 Geneva, SwitzerlandArtero, Ruben论文数: 0 引用数: 0 h-index: 0机构: Incliva Hlth Res Inst, Translat Genom Grp, Menendez Pelayo 4, Valencia 46010, Spain Univ Valencia, Dept Genet, Dr Moliner 50, E-46100 Burjassot, Spain Univ Valencia, Interdisciplinary Res Struct Biotechnol & Biomed, Dr Moliner 50, E-46100 Burjassot, Spain Univ Lausanne, Sch Pharmaceut Sci, Pharmaceut Biochem Grp, Rue Michel Servet 1, CH-1211 Geneva, SwitzerlandMetzger, Friedrich论文数: 0 引用数: 0 h-index: 0机构: F Hoffmann La Roche, Roche Innovat Ctr Basel, Pharmaceut Res & Early Dev, Grenzacherstr 124, CH-4070 Basel, Switzerland Univ Lausanne, Sch Pharmaceut Sci, Pharmaceut Biochem Grp, Rue Michel Servet 1, CH-1211 Geneva, SwitzerlandEbeling, Martin论文数: 0 引用数: 0 h-index: 0机构: F Hoffmann La Roche, Roche Innovat Ctr Basel, Pharmaceut Res & Early Dev, Grenzacherstr 124, CH-4070 Basel, Switzerland Univ Lausanne, Sch Pharmaceut Sci, Pharmaceut Biochem Grp, Rue Michel Servet 1, CH-1211 Geneva, SwitzerlandGoekjian, Peter论文数: 0 引用数: 0 h-index: 0机构: Univ Claude Bernard Lyon 1, UMR CNRS 5246, ICBMS, 43 Bd 11 Novembre 1918, F-69622 Villeurbanne, France Univ Lausanne, Sch Pharmaceut Sci, Pharmaceut Biochem Grp, Rue Michel Servet 1, CH-1211 Geneva, SwitzerlandJoseph, Benoit论文数: 0 引用数: 0 h-index: 0机构: Univ Claude Bernard Lyon 1, UMR CNRS 5246, ICBMS, 43 Bd 11 Novembre 1918, F-69622 Villeurbanne, France Univ Lausanne, Sch Pharmaceut Sci, Pharmaceut Biochem Grp, Rue Michel Servet 1, CH-1211 Geneva, SwitzerlandSchwalbe, Harald论文数: 0 引用数: 0 h-index: 0机构: Goethe Univ Frankfurt, Ctr Biomol Magnet Resonance BMRZ, Inst Organ Chem & Chem Biol, Max von Laue Str 7, D-60438 Frankfurt, Germany Univ Lausanne, Sch Pharmaceut Sci, Pharmaceut Biochem Grp, Rue Michel Servet 1, CH-1211 Geneva, SwitzerlandScapozza, Leonardo论文数: 0 引用数: 0 h-index: 0机构: Univ Lausanne, Sch Pharmaceut Sci, Pharmaceut Biochem Grp, Rue Michel Servet 1, CH-1211 Geneva, Switzerland Univ Geneva, Rue Michel Servet 1, CH-1211 Geneva, Switzerland Univ Lausanne, Sch Pharmaceut Sci, Pharmaceut Biochem Grp, Rue Michel Servet 1, CH-1211 Geneva, Switzerland
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- [50] Optimization of base editors for the functional correction of SMN2 as a treatment for spinal muscular atrophyNature Biomedical Engineering, 2024, 8 : 118 - 131Christiano R. R. Alves论文数: 0 引用数: 0 h-index: 0机构: Massachusetts General Hospital,Center for Genomic MedicineLeillani L. Ha论文数: 0 引用数: 0 h-index: 0机构: Massachusetts General Hospital,Center for Genomic MedicineRebecca Yaworski论文数: 0 引用数: 0 h-index: 0机构: Massachusetts General Hospital,Center for Genomic MedicineEmma R. Sutton论文数: 0 引用数: 0 h-index: 0机构: Massachusetts General Hospital,Center for Genomic MedicineCicera R. Lazzarotto论文数: 0 引用数: 0 h-index: 0机构: Massachusetts General Hospital,Center for Genomic MedicineKathleen A. Christie论文数: 0 引用数: 0 h-index: 0机构: Massachusetts General Hospital,Center for Genomic MedicineAoife Reilly论文数: 0 引用数: 0 h-index: 0机构: Massachusetts General Hospital,Center for Genomic MedicineAriane Beauvais论文数: 0 引用数: 0 h-index: 0机构: Massachusetts General Hospital,Center for Genomic MedicineRoman M. Doll论文数: 0 引用数: 0 h-index: 0机构: Massachusetts General Hospital,Center for Genomic MedicineDemitri de la Cruz论文数: 0 引用数: 0 h-index: 0机构: Massachusetts General Hospital,Center for Genomic MedicineCasey A. Maguire论文数: 0 引用数: 0 h-index: 0机构: Massachusetts General Hospital,Center for Genomic MedicineKathryn J. Swoboda论文数: 0 引用数: 0 h-index: 0机构: Massachusetts General Hospital,Center for Genomic MedicineShengdar Q. Tsai论文数: 0 引用数: 0 h-index: 0机构: Massachusetts General Hospital,Center for Genomic MedicineRashmi Kothary论文数: 0 引用数: 0 h-index: 0机构: Massachusetts General Hospital,Center for Genomic MedicineBenjamin P. Kleinstiver论文数: 0 引用数: 0 h-index: 0机构: Massachusetts General Hospital,Center for Genomic Medicine