共 34 条
- [21] CRISPR/Cas9-Mediated Correction of β-Galactocerebrosidase Gene by Stimulation of Homology-Directed Repair for the Treatment of Krabbe DiseaseMOLECULAR THERAPY, 2020, 28 (04) : 487 - 488Magrath, Justin W.论文数: 0 引用数: 0 h-index: 0机构: Tulane Univ, Sch Med, New Orlenas, LA USA Tulane Univ, Sch Med, New Orlenas, LA USABunnell, Bruce A.论文数: 0 引用数: 0 h-index: 0机构: Tulane Univ, Sch Med, New Orlenas, LA USA Tulane Univ, Sch Med, New Orlenas, LA USA
- [22] CRISPR/Cas9-mediated gene correction ofWilson disease H1069Q mutation in an iPS cell modelJOURNAL OF HEPATOLOGY, 2023, 78 : S957 - S958Zibert, Andree论文数: 0 引用数: 0 h-index: 0机构: Univ Klinikum Munster, Med Klin B, Munster, Germany Univ Klinikum Munster, Med Klin B, Munster, GermanyWeiand, Matthias论文数: 0 引用数: 0 h-index: 0机构: Univ Klinikum Munster, Med Klin B, Munster, Germany Univ Klinikum Munster, Med Klin B, Munster, GermanyNadzemova, Oksana论文数: 0 引用数: 0 h-index: 0机构: Univ Klinikum Munster, Med Klin B, Munster, Germany Univ Klinikum Munster, Med Klin B, Munster, GermanyTrebicka, Jonel论文数: 0 引用数: 0 h-index: 0机构: Univ Klinikum Munster, Med Klin B, Munster, Germany Univ Klinikum Munster, Med Klin B, Munster, GermanySandfort, Vanessa论文数: 0 引用数: 0 h-index: 0机构: Univ Klinikum Munster, Med Klin B, Munster, Germany Univ Klinikum Munster, Med Klin B, Munster, Germany
- [23] CRISPR/Cas9-Mediated In Situ Correction of LAMB3 Gene in Keratinocytes Derived from a Junctional Epidermolysis Bullosa PatientMOLECULAR THERAPY, 2018, 26 (11) : 2592 - 2603Benati, Daniela论文数: 0 引用数: 0 h-index: 0机构: Univ Modena & Reggio Emilia, Dept Life Sci, Ctr Regenerat Med, Modena, Italy Univ Modena & Reggio Emilia, Dept Life Sci, Ctr Regenerat Med, Modena, ItalyMiselli, Francesca论文数: 0 引用数: 0 h-index: 0机构: Univ Modena & Reggio Emilia, Dept Life Sci, Ctr Regenerat Med, Modena, Italy Univ Modena & Reggio Emilia, Dept Life Sci, Ctr Regenerat Med, Modena, ItalyCocchiarella, Fabienne论文数: 0 引用数: 0 h-index: 0机构: Univ Modena & Reggio Emilia, Dept Life Sci, Ctr Regenerat Med, Modena, Italy Univ Modena & Reggio Emilia, Dept Life Sci, Ctr Regenerat Med, Modena, ItalyPatrizi, Clarissa论文数: 0 引用数: 0 h-index: 0机构: Univ Modena & Reggio Emilia, Dept Life Sci, Ctr Regenerat Med, Modena, Italy Univ Modena & Reggio Emilia, Dept Life Sci, Ctr Regenerat Med, Modena, ItalyCarretero, Marta论文数: 0 引用数: 0 h-index: 0机构: CIEMAT CIBERER Ctr Biomed Res Rare Dis, Epithelial Biomed Div, Madrid, Spain Univ Carlos III Madrid, Dept Bioengn, Madrid, Spain Fdn Jimenez Diaz, Inst Invest Sanitaria, Madrid, Spain Univ Modena & Reggio Emilia, Dept Life Sci, Ctr Regenerat Med, Modena, ItalyBaldassarri, Samantha论文数: 0 引用数: 0 h-index: 0机构: Univ Modena & Reggio Emilia, Dept Life Sci, Ctr Regenerat Med, Modena, Italy Univ Modena & Reggio Emilia, Dept Life Sci, Ctr Regenerat Med, Modena, ItalyAmmendola, Virginia论文数: 0 引用数: 0 h-index: 0机构: Reithera Srl, Rome, Italy Univ Modena & Reggio Emilia, Dept Life Sci, Ctr Regenerat Med, Modena, ItalyHas, Cristina论文数: 0 引用数: 0 h-index: 0机构: Univ Med Ctr Freiburg, Dept Dermatol & Venereol, Fac Med, Freiburg, Germany Univ Modena & Reggio Emilia, Dept Life Sci, Ctr Regenerat Med, Modena, ItalyColloca, Stefano论文数: 0 引用数: 0 h-index: 0机构: Reithera Srl, Rome, Italy Univ Modena & Reggio Emilia, Dept Life Sci, Ctr Regenerat Med, Modena, ItalyDel Rio, Marcela论文数: 0 引用数: 0 h-index: 0机构: CIEMAT CIBERER Ctr Biomed Res Rare Dis, Epithelial Biomed Div, Madrid, Spain Univ Carlos III Madrid, Dept Bioengn, Madrid, Spain Fdn Jimenez Diaz, Inst Invest Sanitaria, Madrid, Spain Univ Modena & Reggio Emilia, Dept Life Sci, Ctr Regenerat Med, Modena, ItalyLarcher, Fernando论文数: 0 引用数: 0 h-index: 0机构: CIEMAT CIBERER Ctr Biomed Res Rare Dis, Epithelial Biomed Div, Madrid, Spain Univ Carlos III Madrid, Dept Bioengn, Madrid, Spain Fdn Jimenez Diaz, Inst Invest Sanitaria, Madrid, Spain Univ Modena & Reggio Emilia, Dept Life Sci, Ctr Regenerat Med, Modena, ItalyRecchia, Alessandra论文数: 0 引用数: 0 h-index: 0机构: Univ Modena & Reggio Emilia, Dept Life Sci, Ctr Regenerat Med, Modena, Italy Univ Modena & Reggio Emilia, Dept Life Sci, Ctr Regenerat Med, Modena, Italy
- [24] Reversal of Phenotypic Abnormalities by CRISPR/Cas9-Mediated Gene Correction in Huntington Disease Patient-Derived Induced Pluripotent Stem CellsSTEM CELL REPORTS, 2017, 8 (03): : 619 - 633Xu, Xiaohong论文数: 0 引用数: 0 h-index: 0机构: ASTAR, TLGM, 8A Biomed Grove,Level 5, Singapore 138648, Singapore ASTAR, TLGM, 8A Biomed Grove,Level 5, Singapore 138648, SingaporeTay, Yilin论文数: 0 引用数: 0 h-index: 0机构: ASTAR, TLGM, 8A Biomed Grove,Level 5, Singapore 138648, Singapore ASTAR, TLGM, 8A Biomed Grove,Level 5, Singapore 138648, SingaporeSim, Bernice论文数: 0 引用数: 0 h-index: 0机构: ASTAR, TLGM, 8A Biomed Grove,Level 5, Singapore 138648, Singapore ASTAR, TLGM, 8A Biomed Grove,Level 5, Singapore 138648, SingaporeYoon, Su-In论文数: 0 引用数: 0 h-index: 0机构: Nanyang Technol Univ, Lee Kong Chian Sch Med, Singapore 637553, Singapore ASTAR, TLGM, 8A Biomed Grove,Level 5, Singapore 138648, SingaporeHuang, Yihui论文数: 0 引用数: 0 h-index: 0机构: ASTAR, TLGM, 8A Biomed Grove,Level 5, Singapore 138648, Singapore ASTAR, TLGM, 8A Biomed Grove,Level 5, Singapore 138648, SingaporeOoi, Jolene论文数: 0 引用数: 0 h-index: 0机构: ASTAR, TLGM, 8A Biomed Grove,Level 5, Singapore 138648, Singapore ASTAR, TLGM, 8A Biomed Grove,Level 5, Singapore 138648, SingaporeUtami, Kagistia Hana论文数: 0 引用数: 0 h-index: 0机构: ASTAR, TLGM, 8A Biomed Grove,Level 5, Singapore 138648, Singapore ASTAR, TLGM, 8A Biomed Grove,Level 5, Singapore 138648, SingaporeZiaei, Amin论文数: 0 引用数: 0 h-index: 0机构: ASTAR, TLGM, 8A Biomed Grove,Level 5, Singapore 138648, Singapore ASTAR, TLGM, 8A Biomed Grove,Level 5, Singapore 138648, SingaporeNg, Bryan论文数: 0 引用数: 0 h-index: 0机构: ASTAR, TLGM, 8A Biomed Grove,Level 5, Singapore 138648, Singapore ASTAR, TLGM, 8A Biomed Grove,Level 5, Singapore 138648, SingaporeRadulescu, Carola论文数: 0 引用数: 0 h-index: 0机构: ASTAR, TLGM, 8A Biomed Grove,Level 5, Singapore 138648, Singapore ASTAR, TLGM, 8A Biomed Grove,Level 5, Singapore 138648, SingaporeLow, Donovan论文数: 0 引用数: 0 h-index: 0机构: ASTAR, Singapore Immunol Network SIgN, Singapore 138648, Singapore ASTAR, TLGM, 8A Biomed Grove,Level 5, Singapore 138648, SingaporeNg, Alvin Yu Jin论文数: 0 引用数: 0 h-index: 0机构: ASTAR, Biopolis, Inst Mol & Cell Biol, Comparat Genom Lab, Singapore 138673, Singapore ASTAR, TLGM, 8A Biomed Grove,Level 5, Singapore 138648, SingaporeLoh, Marie论文数: 0 引用数: 0 h-index: 0机构: ASTAR, TLGM, 8A Biomed Grove,Level 5, Singapore 138648, Singapore ASTAR, TLGM, 8A Biomed Grove,Level 5, Singapore 138648, SingaporeVenkatesh, Byrappa论文数: 0 引用数: 0 h-index: 0机构: ASTAR, Biopolis, Inst Mol & Cell Biol, Comparat Genom Lab, Singapore 138673, Singapore Natl Univ Singapore, Yong Loo Lin Sch Med, Dept Paediat, Singapore 119228, Singapore ASTAR, TLGM, 8A Biomed Grove,Level 5, Singapore 138648, SingaporeGinhoux, Florent论文数: 0 引用数: 0 h-index: 0机构: ASTAR, Singapore Immunol Network SIgN, Singapore 138648, Singapore ASTAR, TLGM, 8A Biomed Grove,Level 5, Singapore 138648, SingaporeAugustine, George J.论文数: 0 引用数: 0 h-index: 0机构: Nanyang Technol Univ, Lee Kong Chian Sch Med, Singapore 637553, Singapore IMCB, Singapore 138673, Singapore ASTAR, TLGM, 8A Biomed Grove,Level 5, Singapore 138648, SingaporePouladi, Mahmoud A.论文数: 0 引用数: 0 h-index: 0机构: ASTAR, TLGM, 8A Biomed Grove,Level 5, Singapore 138648, Singapore Natl Univ Singapore, Yong Loo Lin Sch Med, Dept Med, Singapore 117597, Singapore ASTAR, TLGM, 8A Biomed Grove,Level 5, Singapore 138648, Singapore
- [25] Efficient CRISPR/Cas9-Mediated iIn Situ Correction of LAMB3 Gene in Keratinocytes Derived from Junctional Epidermolysis Bullosa PatientMOLECULAR THERAPY, 2018, 26 (05) : 90 - 90Benati, Daniela论文数: 0 引用数: 0 h-index: 0机构: Univ Modena & Reggio Emilia, Dept Life Sci, Modena, Italy Univ Modena & Reggio Emilia, Dept Life Sci, Modena, ItalyMiselli, Francesca论文数: 0 引用数: 0 h-index: 0机构: Univ Modena & Reggio Emilia, Dept Life Sci, Modena, Italy Univ Modena & Reggio Emilia, Dept Life Sci, Modena, ItalyCocchiarella, Fabienne论文数: 0 引用数: 0 h-index: 0机构: Univ Modena & Reggio Emilia, Dept Life Sci, Modena, Italy Univ Modena & Reggio Emilia, Dept Life Sci, Modena, ItalyPatrizi, Clarissa论文数: 0 引用数: 0 h-index: 0机构: Univ Modena & Reggio Emilia, Dept Life Sci, Modena, Italy Univ Modena & Reggio Emilia, Dept Life Sci, Modena, ItalyLatella, Maria Carmela论文数: 0 引用数: 0 h-index: 0机构: Univ Modena & Reggio Emilia, Dept Life Sci, Modena, Italy Univ Modena & Reggio Emilia, Dept Life Sci, Modena, ItalyBaldassarri, Samantha论文数: 0 引用数: 0 h-index: 0机构: Univ Modena & Reggio Emilia, Dept Life Sci, Modena, Italy Univ Modena & Reggio Emilia, Dept Life Sci, Modena, ItalyPedrazzoli, Eleonora论文数: 0 引用数: 0 h-index: 0机构: Univ Modena & Reggio Emilia, Dept Life Sci, Modena, Italy Univ Modena & Reggio Emilia, Dept Life Sci, Modena, ItalyAmmendola, Virginia论文数: 0 引用数: 0 h-index: 0机构: Reithera, Rome, Italy Univ Modena & Reggio Emilia, Dept Life Sci, Modena, ItalyLarcher, Fernando论文数: 0 引用数: 0 h-index: 0机构: CIBERER, CIEMAT, Epithelial Biomed Div, Madrid, Spain Univ Modena & Reggio Emilia, Dept Life Sci, Modena, ItalyRecchia, Alessandra论文数: 0 引用数: 0 h-index: 0机构: Univ Modena & Reggio Emilia, Dept Life Sci, Modena, Italy Univ Modena & Reggio Emilia, Dept Life Sci, Modena, Italy
- [26] Generation of two isogenic human iPSC lines (ZIPi013-B-1, ZIPi013-B-2) carrying a CRISPR/Cas9-mediated deletion of TRPM4STEM CELL RESEARCH, 2024, 81Haferkamp, Undine论文数: 0 引用数: 0 h-index: 0机构: Fraunhofer Inst Translat Med & Pharmacol ITPM, Discovery Res ScreeningPort, Hamburg, Germany Fraunhofer Inst Translat Med & Pharmacol ITPM, Discovery Res ScreeningPort, Hamburg, GermanyTelugu, Narasimha论文数: 0 引用数: 0 h-index: 0机构: Max Delbruck Ctr Mol Med Helmholtz Assoc MDC, Technol Platform Pluripotent Stem Cells, Berlin, Germany Fraunhofer Inst Translat Med & Pharmacol ITPM, Discovery Res ScreeningPort, Hamburg, GermanyKrieg, Kim论文数: 0 引用数: 0 h-index: 0机构: Fraunhofer Inst Translat Med & Pharmacol ITPM, Discovery Res ScreeningPort, Hamburg, Germany Fraunhofer Inst Translat Med & Pharmacol ITPM, Discovery Res ScreeningPort, Hamburg, GermanySchaefer, Wiebe论文数: 0 引用数: 0 h-index: 0机构: Fraunhofer Inst Translat Med & Pharmacol ITPM, Discovery Res ScreeningPort, Hamburg, Germany Fraunhofer Inst Translat Med & Pharmacol ITPM, Discovery Res ScreeningPort, Hamburg, GermanyLam, Dennis论文数: 0 引用数: 0 h-index: 0机构: Fraunhofer Inst Translat Med & Pharmacol ITPM, Discovery Res ScreeningPort, Hamburg, Germany Fraunhofer Inst Translat Med & Pharmacol ITPM, Discovery Res ScreeningPort, Hamburg, GermanyBinkle-Ladisch, Lars论文数: 0 引用数: 0 h-index: 0机构: Univ Med Ctr Hamburg Eppendorf, Inst Neuroimmunol & Multiple Sclerosis, Hamburg, Germany Fraunhofer Inst Translat Med & Pharmacol ITPM, Discovery Res ScreeningPort, Hamburg, GermanyFriese, Manuel A.论文数: 0 引用数: 0 h-index: 0机构: Univ Med Ctr Hamburg Eppendorf, Inst Neuroimmunol & Multiple Sclerosis, Hamburg, Germany Fraunhofer Inst Translat Med & Pharmacol ITPM, Discovery Res ScreeningPort, Hamburg, GermanyDiecke, Sebastian论文数: 0 引用数: 0 h-index: 0机构: Max Delbruck Ctr Mol Med Helmholtz Assoc MDC, Technol Platform Pluripotent Stem Cells, Berlin, Germany Fraunhofer Inst Translat Med & Pharmacol ITPM, Discovery Res ScreeningPort, Hamburg, GermanyPless, Ole论文数: 0 引用数: 0 h-index: 0机构: Fraunhofer Inst Translat Med & Pharmacol ITPM, Discovery Res ScreeningPort, Hamburg, Germany Fraunhofer Inst Translat Med & Pharmacol ITPM, Discovery Res ScreeningPort, Hamburg, Germany
- [27] CRISPR/Cas9-mediated excision of ALS/FTD-causing hexanucleotide repeat expansion in C9ORF72 rescues major disease mechanisms in vivo and in vitroNATURE COMMUNICATIONS, 2022, 13 (01)Meijboom, Katharina E.论文数: 0 引用数: 0 h-index: 0机构: Univ Massachusetts, Sch Med, Horae Gene Therapy Ctr, Worcester, MA 01605 USA Univ Massachusetts, Sch Med, Dept Neurol, Worcester, MA 01605 USA Univ Massachusetts, Sch Med, Horae Gene Therapy Ctr, Worcester, MA 01605 USAAbdallah, Abbas论文数: 0 引用数: 0 h-index: 0机构: Univ Massachusetts, Sch Med, Horae Gene Therapy Ctr, Worcester, MA 01605 USA Univ Massachusetts, Sch Med, Horae Gene Therapy Ctr, Worcester, MA 01605 USAFordham, Nicholas P.论文数: 0 引用数: 0 h-index: 0机构: Univ Massachusetts, Sch Med, Horae Gene Therapy Ctr, Worcester, MA 01605 USA Univ Massachusetts, Sch Med, Horae Gene Therapy Ctr, Worcester, MA 01605 USANagase, Hiroko论文数: 0 引用数: 0 h-index: 0机构: Univ Massachusetts, Sch Med, Horae Gene Therapy Ctr, Worcester, MA 01605 USA Univ Massachusetts, Sch Med, Horae Gene Therapy Ctr, Worcester, MA 01605 USARodriguez, Tomas论文数: 0 引用数: 0 h-index: 0机构: Univ Massachusetts, Sch Med, RNA Therapeut Inst & Program Mol Med, Worcester, MA 01605 USA Univ Massachusetts, Sch Med, Horae Gene Therapy Ctr, Worcester, MA 01605 USA论文数: 引用数: h-index:机构:Gendron, Tania F.论文数: 0 引用数: 0 h-index: 0机构: Mayo Clin, Dept Neurosci, Jacksonville, FL 32224 USA Univ Massachusetts, Sch Med, Horae Gene Therapy Ctr, Worcester, MA 01605 USAKrishnan, Gopinath论文数: 0 引用数: 0 h-index: 0机构: Univ Massachusetts, Sch Med, Dept Neurol, Worcester, MA 01605 USA Univ Massachusetts, Sch Med, Horae Gene Therapy Ctr, Worcester, MA 01605 USAEsanov, Rustam论文数: 0 引用数: 0 h-index: 0机构: Univ Miami, Miller Sch Med, Ctr Therapeut Innovat, Dept Psychiat & Behav Sci, Miami, FL 33136 USA Univ Massachusetts, Sch Med, Horae Gene Therapy Ctr, Worcester, MA 01605 USAAndrade, Nadja S.论文数: 0 引用数: 0 h-index: 0机构: Univ Miami, Miller Sch Med, Ctr Therapeut Innovat, Dept Psychiat & Behav Sci, Miami, FL 33136 USA Univ Massachusetts, Sch Med, Horae Gene Therapy Ctr, Worcester, MA 01605 USA论文数: 引用数: h-index:机构:Ramic, Melina论文数: 0 引用数: 0 h-index: 0机构: Univ Miami, Miller Sch Med, Ctr Therapeut Innovat, Dept Psychiat & Behav Sci, Miami, FL 33136 USA Univ Massachusetts, Sch Med, Horae Gene Therapy Ctr, Worcester, MA 01605 USAStephens, Zachary D.论文数: 0 引用数: 0 h-index: 0机构: Mayo Clin, Dept Quantitat Hlth Sci, Rochester, MN 55905 USA Univ Massachusetts, Sch Med, Horae Gene Therapy Ctr, Worcester, MA 01605 USA论文数: 引用数: h-index:机构:Blackwood, Meghan T.论文数: 0 引用数: 0 h-index: 0机构: Univ Massachusetts, Sch Med, Horae Gene Therapy Ctr, Worcester, MA 01605 USA Univ Massachusetts, Sch Med, Horae Gene Therapy Ctr, Worcester, MA 01605 USAKahriman, Aydan论文数: 0 引用数: 0 h-index: 0机构: Univ Massachusetts, Sch Med, Dept Neurol, Worcester, MA 01605 USA Univ Massachusetts, Sch Med, Horae Gene Therapy Ctr, Worcester, MA 01605 USAHenninger, Nils论文数: 0 引用数: 0 h-index: 0机构: Univ Massachusetts, Sch Med, Dept Neurol, Worcester, MA 01605 USA Univ Massachusetts, Sch Med, Horae Gene Therapy Ctr, Worcester, MA 01605 USAKocher, Jean-Pierre A.论文数: 0 引用数: 0 h-index: 0机构: Mayo Clin, Dept Quantitat Hlth Sci, Rochester, MN 55905 USA Univ Massachusetts, Sch Med, Horae Gene Therapy Ctr, Worcester, MA 01605 USABenatar, Michael论文数: 0 引用数: 0 h-index: 0机构: Univ Miami, Miller Sch Med, Dept Neurol, Miami, FL 33136 USA Univ Massachusetts, Sch Med, Horae Gene Therapy Ctr, Worcester, MA 01605 USABrodsky, Michael H.论文数: 0 引用数: 0 h-index: 0机构: Univ Massachusetts, Sch Med, Dept Mol Cell & Canc Biol, Worcester, MA 01605 USA Univ Massachusetts, Sch Med, Horae Gene Therapy Ctr, Worcester, MA 01605 USAPetrucelli, Leonard论文数: 0 引用数: 0 h-index: 0机构: Mayo Clin, Dept Neurosci, Jacksonville, FL 32224 USA Univ Massachusetts, Sch Med, Horae Gene Therapy Ctr, Worcester, MA 01605 USAGao, Fen-Biao论文数: 0 引用数: 0 h-index: 0机构: Univ Massachusetts, Sch Med, Dept Neurol, Worcester, MA 01605 USA Univ Massachusetts, Sch Med, Horae Gene Therapy Ctr, Worcester, MA 01605 USASontheimer, Erik J.论文数: 0 引用数: 0 h-index: 0机构: Univ Massachusetts, Sch Med, RNA Therapeut Inst & Program Mol Med, Worcester, MA 01605 USA Univ Massachusetts, Sch Med, Horae Gene Therapy Ctr, Worcester, MA 01605 USABrown, Robert H.论文数: 0 引用数: 0 h-index: 0机构: Univ Massachusetts, Sch Med, Dept Neurol, Worcester, MA 01605 USA Univ Massachusetts, Sch Med, Horae Gene Therapy Ctr, Worcester, MA 01605 USA论文数: 引用数: h-index:机构:Mueller, Christian论文数: 0 引用数: 0 h-index: 0机构: Univ Massachusetts, Sch Med, Horae Gene Therapy Ctr, Worcester, MA 01605 USA Univ Massachusetts, Sch Med, Horae Gene Therapy Ctr, Worcester, MA 01605 USA
- [28] CRISPR/Cas9-Mediated Excision of ALS/FTD-Causing Hexanucleotide Repeat Expansion in C9ORF72 Rescues Major Disease Mechanisms In Vivo and In VitroMOLECULAR THERAPY, 2021, 29 (04) : 30 - 30Meijboom, Katharina E.论文数: 0 引用数: 0 h-index: 0机构: UMASS Med Sch, Gene Therapy Ctr, Worcester, MA USA UMASS Med Sch, Gene Therapy Ctr, Worcester, MA USAAbdallah, Abbas论文数: 0 引用数: 0 h-index: 0机构: UMASS Med Sch, Gene Therapy Ctr, Worcester, MA USA UMASS Med Sch, Gene Therapy Ctr, Worcester, MA USAFordham, Nicholas论文数: 0 引用数: 0 h-index: 0机构: UMASS Med Sch, Gene Therapy Ctr, Worcester, MA USA UMASS Med Sch, Gene Therapy Ctr, Worcester, MA USANagase, Hiroko论文数: 0 引用数: 0 h-index: 0机构: UMASS Med Sch, Gene Therapy Ctr, Worcester, MA USA UMASS Med Sch, Gene Therapy Ctr, Worcester, MA USAGendron, Tania F.论文数: 0 引用数: 0 h-index: 0机构: Mayo Clin, Jacksonville, FL 32224 USA UMASS Med Sch, Gene Therapy Ctr, Worcester, MA USAKrishnan, Gopinath论文数: 0 引用数: 0 h-index: 0机构: UMASS Med Sch, Worcester, MA USA UMASS Med Sch, Gene Therapy Ctr, Worcester, MA USARodriguez, Tomas论文数: 0 引用数: 0 h-index: 0机构: UMASS Med Sch, Worcester, MA USA UMASS Med Sch, Gene Therapy Ctr, Worcester, MA USAEdraki, Alireza论文数: 0 引用数: 0 h-index: 0机构: UMASS Med Sch, Worcester, MA USA UMASS Med Sch, Gene Therapy Ctr, Worcester, MA USABlackwood, Meghan论文数: 0 引用数: 0 h-index: 0机构: UMASS Med Sch, Gene Therapy Ctr, Worcester, MA USA UMASS Med Sch, Gene Therapy Ctr, Worcester, MA USASontheimer, Erik论文数: 0 引用数: 0 h-index: 0机构: UMASS Med Sch, Worcester, MA USA UMASS Med Sch, Gene Therapy Ctr, Worcester, MA USAGao, Fen-Biao论文数: 0 引用数: 0 h-index: 0机构: UMASS Med Sch, Worcester, MA USA UMASS Med Sch, Gene Therapy Ctr, Worcester, MA USABrown, Robert H.论文数: 0 引用数: 0 h-index: 0机构: UMASS Med Sch, Worcester, MA USA UMASS Med Sch, Gene Therapy Ctr, Worcester, MA USAZeier, Zane论文数: 0 引用数: 0 h-index: 0机构: Univ Miami, Miami, FL USA UMASS Med Sch, Gene Therapy Ctr, Worcester, MA USAMueller, Christian论文数: 0 引用数: 0 h-index: 0机构: UMASS Med Sch, Gene Therapy Ctr, Worcester, MA USA UMASS Med Sch, Gene Therapy Ctr, Worcester, MA USA
- [29] CRISPR/Cas9-mediated excision of ALS/FTD-causing hexanucleotide repeat expansion in C9ORF72 rescues major disease mechanisms in vivo and in vitroNature Communications, 13Katharina E. Meijboom论文数: 0 引用数: 0 h-index: 0机构: University of Massachusetts Medical School,Horae Gene Therapy CenterAbbas Abdallah论文数: 0 引用数: 0 h-index: 0机构: University of Massachusetts Medical School,Horae Gene Therapy CenterNicholas P. Fordham论文数: 0 引用数: 0 h-index: 0机构: University of Massachusetts Medical School,Horae Gene Therapy CenterHiroko Nagase论文数: 0 引用数: 0 h-index: 0机构: University of Massachusetts Medical School,Horae Gene Therapy CenterTomás Rodriguez论文数: 0 引用数: 0 h-index: 0机构: University of Massachusetts Medical School,Horae Gene Therapy CenterCarolyn Kraus论文数: 0 引用数: 0 h-index: 0机构: University of Massachusetts Medical School,Horae Gene Therapy CenterTania F. Gendron论文数: 0 引用数: 0 h-index: 0机构: University of Massachusetts Medical School,Horae Gene Therapy CenterGopinath Krishnan论文数: 0 引用数: 0 h-index: 0机构: University of Massachusetts Medical School,Horae Gene Therapy CenterRustam Esanov论文数: 0 引用数: 0 h-index: 0机构: University of Massachusetts Medical School,Horae Gene Therapy CenterNadja S. Andrade论文数: 0 引用数: 0 h-index: 0机构: University of Massachusetts Medical School,Horae Gene Therapy CenterMatthew J. Rybin论文数: 0 引用数: 0 h-index: 0机构: University of Massachusetts Medical School,Horae Gene Therapy CenterMelina Ramic论文数: 0 引用数: 0 h-index: 0机构: University of Massachusetts Medical School,Horae Gene Therapy CenterZachary D. Stephens论文数: 0 引用数: 0 h-index: 0机构: University of Massachusetts Medical School,Horae Gene Therapy CenterAlireza Edraki论文数: 0 引用数: 0 h-index: 0机构: University of Massachusetts Medical School,Horae Gene Therapy CenterMeghan T. Blackwood论文数: 0 引用数: 0 h-index: 0机构: University of Massachusetts Medical School,Horae Gene Therapy CenterAydan Kahriman论文数: 0 引用数: 0 h-index: 0机构: University of Massachusetts Medical School,Horae Gene Therapy CenterNils Henninger论文数: 0 引用数: 0 h-index: 0机构: University of Massachusetts Medical School,Horae Gene Therapy CenterJean-Pierre A. Kocher论文数: 0 引用数: 0 h-index: 0机构: University of Massachusetts Medical School,Horae Gene Therapy CenterMichael Benatar论文数: 0 引用数: 0 h-index: 0机构: University of Massachusetts Medical School,Horae Gene Therapy CenterMichael H. Brodsky论文数: 0 引用数: 0 h-index: 0机构: University of Massachusetts Medical School,Horae Gene Therapy CenterLeonard Petrucelli论文数: 0 引用数: 0 h-index: 0机构: University of Massachusetts Medical School,Horae Gene Therapy CenterFen-Biao Gao论文数: 0 引用数: 0 h-index: 0机构: University of Massachusetts Medical School,Horae Gene Therapy CenterErik J. Sontheimer论文数: 0 引用数: 0 h-index: 0机构: University of Massachusetts Medical School,Horae Gene Therapy CenterRobert H. Brown论文数: 0 引用数: 0 h-index: 0机构: University of Massachusetts Medical School,Horae Gene Therapy CenterZane Zeier论文数: 0 引用数: 0 h-index: 0机构: University of Massachusetts Medical School,Horae Gene Therapy CenterChristian Mueller论文数: 0 引用数: 0 h-index: 0机构: University of Massachusetts Medical School,Horae Gene Therapy Center
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