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- [1] Post-symptomatic scAAV9-mediated gene therapy in a murine model of severe SMAHUMAN GENE THERAPY, 2012, 23 (05) : A19 - A19Tanguy, Yannick Y. T.论文数: 0 引用数: 0 h-index: 0机构: UPMC CNRS AIM INSERM UMRS 974, Inst Myol, Biotherapies Neuromuscular Dis Team, Grp CNS Motoneuron, F-75013 Paris, France UPMC CNRS AIM INSERM UMRS 974, Inst Myol, Biotherapies Neuromuscular Dis Team, Grp CNS Motoneuron, F-75013 Paris, FranceMarais, Thibault T. M.论文数: 0 引用数: 0 h-index: 0机构: UPMC CNRS AIM INSERM UMRS 974, Inst Myol, Biotherapies Neuromuscular Dis Team, Grp CNS Motoneuron, F-75013 Paris, France UPMC CNRS AIM INSERM UMRS 974, Inst Myol, Biotherapies Neuromuscular Dis Team, Grp CNS Motoneuron, F-75013 Paris, FranceRoda, Marianne M. R.论文数: 0 引用数: 0 h-index: 0机构: UPMC CNRS AIM INSERM UMRS 974, Inst Myol, Biotherapies Neuromuscular Dis Team, Grp CNS Motoneuron, F-75013 Paris, France UPMC CNRS AIM INSERM UMRS 974, Inst Myol, Biotherapies Neuromuscular Dis Team, Grp CNS Motoneuron, F-75013 Paris, FranceAstord, Stephanie S. A.论文数: 0 引用数: 0 h-index: 0机构: UPMC CNRS AIM INSERM UMRS 974, Inst Myol, Biotherapies Neuromuscular Dis Team, Grp CNS Motoneuron, F-75013 Paris, France UPMC CNRS AIM INSERM UMRS 974, Inst Myol, Biotherapies Neuromuscular Dis Team, Grp CNS Motoneuron, F-75013 Paris, FranceBesse, Aurore A. B.论文数: 0 引用数: 0 h-index: 0机构: UPMC CNRS AIM INSERM UMRS 974, Inst Myol, Biotherapies Neuromuscular Dis Team, Grp CNS Motoneuron, F-75013 Paris, France UPMC CNRS AIM INSERM UMRS 974, Inst Myol, Biotherapies Neuromuscular Dis Team, Grp CNS Motoneuron, F-75013 Paris, FranceBarkats, Martine M. B.论文数: 0 引用数: 0 h-index: 0机构: UPMC CNRS AIM INSERM UMRS 974, Inst Myol, Biotherapies Neuromuscular Dis Team, Grp CNS Motoneuron, F-75013 Paris, France UPMC CNRS AIM INSERM UMRS 974, Inst Myol, Biotherapies Neuromuscular Dis Team, Grp CNS Motoneuron, F-75013 Paris, France
- [2] Significant Therapeutic Benefit after Post-Symptomatic Gene Therapy in a Feline Model of GM2 GangliosidosisMOLECULAR THERAPY, 2013, 21 : S145 - S145McCurdy, Victoria J.论文数: 0 引用数: 0 h-index: 0机构: Auburn Univ, Coll Vet Med, Auburn, AL 36849 USA Auburn Univ, Coll Vet Med, Auburn, AL 36849 USARandle, Ashley N.论文数: 0 引用数: 0 h-index: 0机构: Auburn Univ, Coll Vet Med, Auburn, AL 36849 USA Auburn Univ, Coll Vet Med, Auburn, AL 36849 USAGray-Edwards, Heather L.论文数: 0 引用数: 0 h-index: 0机构: Auburn Univ, Coll Vet Med, Auburn, AL 36849 USA Auburn Univ, Coll Vet Med, Auburn, AL 36849 USABradbury, Allison M.论文数: 0 引用数: 0 h-index: 0机构: Auburn Univ, Coll Vet Med, Auburn, AL 36849 USA Auburn Univ, Coll Vet Med, Auburn, AL 36849 USAJohnson, Aime K.论文数: 0 引用数: 0 h-index: 0机构: Auburn Univ, Coll Vet Med, Auburn, AL 36849 USA Auburn Univ, Coll Vet Med, Auburn, AL 36849 USABeadlescomb, Patricia M.论文数: 0 引用数: 0 h-index: 0机构: Auburn Univ, Coll Vet Med, Auburn, AL 36849 USA Auburn Univ, Coll Vet Med, Auburn, AL 36849 USASena-Esteves, Miguel论文数: 0 引用数: 0 h-index: 0机构: Univ Massachusetts, Sch Med, Gene Therapy Ctr, Worcester, MA USA Auburn Univ, Coll Vet Med, Auburn, AL 36849 USAMartin, Douglas R.论文数: 0 引用数: 0 h-index: 0机构: Auburn Univ, Coll Vet Med, Auburn, AL 36849 USA Auburn Univ, Coll Vet Med, Auburn, AL 36849 USA
- [3] Respiratory And Airway Smooth Muscle Pathology In A Pompe Disease Mouse ModelAMERICAN JOURNAL OF RESPIRATORY AND CRITICAL CARE MEDICINE, 2017, 195Keeler, A. M.论文数: 0 引用数: 0 h-index: 0机构: Univ Massachusetts, Sch Med, Worcester, MA USA Univ Massachusetts, Sch Med, Worcester, MA USAZieger, M.论文数: 0 引用数: 0 h-index: 0机构: Univ Massachusetts, Sch Med, Worcester, MA USA Univ Massachusetts, Sch Med, Worcester, MA USALiu, D.论文数: 0 引用数: 0 h-index: 0机构: Univ Massachusetts, Sch Med, Worcester, MA USA Univ Massachusetts, Sch Med, Worcester, MA USASalemi, J.论文数: 0 引用数: 0 h-index: 0机构: Univ Massachusetts, Sch Med, Worcester, MA USA Univ Massachusetts, Sch Med, Worcester, MA USAZhuGe, R.论文数: 0 引用数: 0 h-index: 0机构: Univ Massachusetts, Sch Med, Worcester, MA USA Univ Massachusetts, Sch Med, Worcester, MA USAElMallah, M. K.论文数: 0 引用数: 0 h-index: 0机构: Univ Massachusetts, Sch Med, Worcester, MA USA Univ Massachusetts, Sch Med, Worcester, MA USA
- [4] Quantification of muscle pathology in infantile Pompe diseaseNEUROMUSCULAR DISORDERS, 2017, 27 (02) : 141 - 152Schaenzer, Anne论文数: 0 引用数: 0 h-index: 0机构: Justus Liebig Univ, Inst Neuropathol, Giessen, Germany Justus Liebig Univ, Inst Neuropathol, Giessen, GermanyKaiser, Ann-Kathrin论文数: 0 引用数: 0 h-index: 0机构: Justus Liebig Univ, Inst Neuropathol, Giessen, Germany Justus Liebig Univ, Inst Neuropathol, Giessen, GermanyMuehlfeld, Christian论文数: 0 引用数: 0 h-index: 0机构: Hannover Med Sch, Inst Funct & Appl Anat, Hannover, Germany Justus Liebig Univ, Inst Neuropathol, Giessen, GermanyKulessa, Martin论文数: 0 引用数: 0 h-index: 0机构: Justus Liebig Univ, Inst Neuropathol, Giessen, Germany Justus Liebig Univ, Inst Neuropathol, Giessen, GermanyPaulus, Werner论文数: 0 引用数: 0 h-index: 0机构: Univ Med Ctr, Inst Neuropathol, Munster, Germany Justus Liebig Univ, Inst Neuropathol, Giessen, Germanyvon Pein, Harald论文数: 0 引用数: 0 h-index: 0机构: Univ Med Ctr JGU Univ, Dept Neuropathol, Mainz, Germany Justus Liebig Univ, Inst Neuropathol, Giessen, GermanyRohrbach, Marianne论文数: 0 引用数: 0 h-index: 0机构: Univ Childrens Hosp, Childrens Res Ctr, Div Metab, Zurich, Switzerland Justus Liebig Univ, Inst Neuropathol, Giessen, GermanyViergutz, Lara论文数: 0 引用数: 0 h-index: 0机构: Justus Liebig Univ, Inst Neuropathol, Giessen, Germany Justus Liebig Univ, Inst Neuropathol, Giessen, GermanyMengel, Eugen论文数: 0 引用数: 0 h-index: 0机构: Univ Med Ctr JGU Univ, Villa Metab, Mainz, Germany Justus Liebig Univ, Inst Neuropathol, Giessen, GermanyMarquardt, Thorsten论文数: 0 引用数: 0 h-index: 0机构: Westfal Wilhelms Univ, Dept Pediat, Munster, Germany Justus Liebig Univ, Inst Neuropathol, Giessen, GermanyNeubauer, Bernd论文数: 0 引用数: 0 h-index: 0机构: Justus Liebig Univ, Dept Child Neurol, Giessen, Germany Justus Liebig Univ, Inst Neuropathol, Giessen, GermanyAcker, Till论文数: 0 引用数: 0 h-index: 0机构: Justus Liebig Univ, Inst Neuropathol, Giessen, Germany Justus Liebig Univ, Inst Neuropathol, Giessen, GermanyHahn, Andreas论文数: 0 引用数: 0 h-index: 0机构: Justus Liebig Univ, Dept Child Neurol, Giessen, Germany Justus Liebig Univ, Inst Neuropathol, Giessen, Germany
- [5] Pompe disease gene therapyHUMAN MOLECULAR GENETICS, 2011, 20 : R61 - R68Byrne, Barry J.论文数: 0 引用数: 0 h-index: 0机构: Univ Florida, Dept Pediat, Gainesville, FL 32610 USA Univ Florida, Powell Gene Therapy Ctr, Gainesville, FL 32610 USA Univ Florida, Dept Pediat, Gainesville, FL 32610 USAFalk, Darin J.论文数: 0 引用数: 0 h-index: 0机构: Univ Florida, Dept Pediat, Gainesville, FL 32610 USA Univ Florida, Powell Gene Therapy Ctr, Gainesville, FL 32610 USA Univ Florida, Dept Pediat, Gainesville, FL 32610 USAPacak, Christina A.论文数: 0 引用数: 0 h-index: 0机构: Childrens Hosp, Dept Anesthesia, Boston, MA 02115 USA Univ Florida, Dept Pediat, Gainesville, FL 32610 USANayak, Sushrusha论文数: 0 引用数: 0 h-index: 0机构: Univ Florida, Dept Pediat, Gainesville, FL 32610 USA Univ Florida, Powell Gene Therapy Ctr, Gainesville, FL 32610 USA Univ Florida, Dept Pediat, Gainesville, FL 32610 USAHerzog, Roland W.论文数: 0 引用数: 0 h-index: 0机构: Univ Florida, Dept Pediat, Gainesville, FL 32610 USA Univ Florida, Powell Gene Therapy Ctr, Gainesville, FL 32610 USA Univ Florida, Dept Pediat, Gainesville, FL 32610 USAElder, Melissa E.论文数: 0 引用数: 0 h-index: 0机构: Univ Florida, Dept Pediat, Gainesville, FL 32610 USA Univ Florida, Dept Pediat, Gainesville, FL 32610 USACollins, Shelley W.论文数: 0 引用数: 0 h-index: 0机构: Univ Florida, Dept Pediat, Gainesville, FL 32610 USA Univ Florida, Dept Pediat, Gainesville, FL 32610 USAConlon, Thomas J.论文数: 0 引用数: 0 h-index: 0机构: Univ Florida, Dept Pediat, Gainesville, FL 32610 USA Univ Florida, Powell Gene Therapy Ctr, Gainesville, FL 32610 USA Univ Florida, Dept Pediat, Gainesville, FL 32610 USAClement, Nathalie论文数: 0 引用数: 0 h-index: 0机构: Univ Florida, Dept Pediat, Gainesville, FL 32610 USA Univ Florida, Powell Gene Therapy Ctr, Gainesville, FL 32610 USA Univ Florida, Dept Pediat, Gainesville, FL 32610 USACleaver, Brian D.论文数: 0 引用数: 0 h-index: 0机构: Univ Florida, Powell Gene Therapy Ctr, Gainesville, FL 32610 USA Univ Florida, Dept Pediat, Gainesville, FL 32610 USACloutier, Denise A.论文数: 0 引用数: 0 h-index: 0机构: Univ Florida, Dept Pediat, Gainesville, FL 32610 USA Univ Florida, Powell Gene Therapy Ctr, Gainesville, FL 32610 USA Univ Florida, Dept Pediat, Gainesville, FL 32610 USAPorvasnik, Stacy L.论文数: 0 引用数: 0 h-index: 0机构: Univ Florida, Dept Pediat, Gainesville, FL 32610 USA Univ Florida, Powell Gene Therapy Ctr, Gainesville, FL 32610 USA Univ Florida, Dept Pediat, Gainesville, FL 32610 USAIslam, Saleem论文数: 0 引用数: 0 h-index: 0机构: Univ Florida, Dept Pediat, Gainesville, FL 32610 USA Univ Florida, Dept Surg, Gainesville, FL 32610 USA Univ Florida, Dept Pediat, Gainesville, FL 32610 USAElmallah, Mai K.论文数: 0 引用数: 0 h-index: 0机构: Univ Florida, Dept Pediat, Gainesville, FL 32610 USA Univ Florida, Dept Pediat, Gainesville, FL 32610 USAMartin, Anatole论文数: 0 引用数: 0 h-index: 0机构: Univ Florida, Dept Phys Therapy, Gainesville, FL 32610 USA Univ Florida, Dept Pediat, Gainesville, FL 32610 USASmith, Barbara K.论文数: 0 引用数: 0 h-index: 0机构: Univ Florida, Dept Phys Therapy, Gainesville, FL 32610 USA Univ Florida, Dept Pediat, Gainesville, FL 32610 USAFuller, David D.论文数: 0 引用数: 0 h-index: 0机构: Univ Florida, Dept Phys Therapy, Gainesville, FL 32610 USA Univ Florida, Dept Pediat, Gainesville, FL 32610 USALawson, Lee Ann论文数: 0 引用数: 0 h-index: 0机构: Univ Florida, Dept Pediat, Gainesville, FL 32610 USA Univ Florida, Powell Gene Therapy Ctr, Gainesville, FL 32610 USA Univ Florida, Dept Pediat, Gainesville, FL 32610 USAMah, Cathryn S.论文数: 0 引用数: 0 h-index: 0机构: Univ Florida, Dept Pediat, Gainesville, FL 32610 USA Univ Florida, Powell Gene Therapy Ctr, Gainesville, FL 32610 USA Univ Florida, Dept Pediat, Gainesville, FL 32610 USA
- [6] Lentivirus mediated gene therapy for murine model of Pompe diseaseJOURNAL OF INHERITED METABOLIC DISEASE, 2008, 31 : 99 - 99Kyosen, S. O.论文数: 0 引用数: 0 h-index: 0机构: Jikei Univ, Sch Med, Dept Gene Ther, Tokyo, Japan Jikei Univ, Sch Med, Dept Gene Ther, Tokyo, JapanIizuka, S.论文数: 0 引用数: 0 h-index: 0机构: Jikei Univ, Sch Med, Dept Gene Ther, Tokyo, Japan Jikei Univ, Sch Med, Dept Gene Ther, Tokyo, JapanMorita, A.论文数: 0 引用数: 0 h-index: 0机构: Jikei Univ, Sch Med, Dept Gene Ther, Tokyo, Japan Jikei Univ, Sch Med, Dept Gene Ther, Tokyo, JapanKimura, T.论文数: 0 引用数: 0 h-index: 0机构: Jikei Univ, Sch Med, Dept Urol, Tokyo, Japan Jikei Univ, Sch Med, Dept Gene Ther, Tokyo, JapanKobayashi, H.论文数: 0 引用数: 0 h-index: 0机构: Jikei Univ, Sch Med, Dept Gene Ther, Tokyo, Japan Jikei Univ, Sch Med, Dept Gene Ther, Tokyo, JapanEto, Y.论文数: 0 引用数: 0 h-index: 0机构: Jikei Univ, Sch Med, Dept Gene Ther, Tokyo, JapanIda, H.论文数: 0 引用数: 0 h-index: 0机构: Jikei Univ, Sch Med, Dept Ped, Tokyo, Japan Jikei Univ, Sch Med, Dept Gene Ther, Tokyo, JapanOhashi, T.论文数: 0 引用数: 0 h-index: 0机构: Jikei Univ, Sch Med, Dept Gene Ther, Tokyo, Japan Jikei Univ, Sch Med, Dept Gene Ther, Tokyo, Japan
- [7] A Novel Capsid for Smooth Muscle Pathology in Pompe DiseaseMOLECULAR THERAPY, 2020, 28 (04) : 492 - 492McCall, Angela L.论文数: 0 引用数: 0 h-index: 0机构: Duke Univ, Pediat, Durham, NC USA Duke Univ, Pediat, Durham, NC USAde Leon, Augusto论文数: 0 引用数: 0 h-index: 0机构: Duke Univ, Pediat, Durham, NC USA Duke Univ, Pediat, Durham, NC USAFusco, Anna F.论文数: 0 引用数: 0 h-index: 0机构: Duke Univ, Pediat, Durham, NC USA Duke Univ, Pediat, Durham, NC USABailey, Aidan论文数: 0 引用数: 0 h-index: 0机构: Duke Univ, Pediat, Durham, NC USA Duke Univ, Pediat, Durham, NC USAFanous, Marco论文数: 0 引用数: 0 h-index: 0机构: Duke Univ, Surg, Durham, NC USA Duke Univ, Pediat, Durham, NC USA论文数: 引用数: h-index:机构:ElMallah, Mai K.论文数: 0 引用数: 0 h-index: 0机构: Duke Univ, Pediat, Durham, NC USA Duke Univ, Pediat, Durham, NC USA
- [8] Assessment of Gene Therapy Treatment in the Pompe Disease Canine ModelMOLECULAR THERAPY, 2022, 30 (04) : 2 - 3Pope, Megan K.论文数: 0 引用数: 0 h-index: 0机构: Univ Florida, Powell Gene Therapy Ctr, Pediat, Gainesville, FL USA Univ Florida, Powell Gene Therapy Ctr, Pediat, Gainesville, FL USAColeman, Kirsten论文数: 0 引用数: 0 h-index: 0机构: Univ Florida, Powell Gene Therapy Ctr, Pediat, Gainesville, FL USA Univ Florida, Powell Gene Therapy Ctr, Pediat, Gainesville, FL USAWichman, Megan论文数: 0 引用数: 0 h-index: 0机构: Univ Florida, Powell Gene Therapy Ctr, Pediat, Gainesville, FL USA Univ Florida, Powell Gene Therapy Ctr, Pediat, Gainesville, FL USACloutier, Denise论文数: 0 引用数: 0 h-index: 0机构: Univ Florida, Powell Gene Therapy Ctr, Pediat, Gainesville, FL USA Univ Florida, Powell Gene Therapy Ctr, Pediat, Gainesville, FL USACousins, Christina论文数: 0 引用数: 0 h-index: 0机构: Univ Florida, Pediat, Gainesville, FL USA Univ Florida, Powell Gene Therapy Ctr, Pediat, Gainesville, FL USAMueller, Lee Ann论文数: 0 引用数: 0 h-index: 0机构: Univ Florida, Powell Gene Therapy Ctr, Pediat, Gainesville, FL USA Univ Florida, Powell Gene Therapy Ctr, Pediat, Gainesville, FL USAWhitehead, Victoria论文数: 0 引用数: 0 h-index: 0机构: ProtoKinet, Haverton, PA USA Univ Florida, Powell Gene Therapy Ctr, Pediat, Gainesville, FL USAMeena, Naresh论文数: 0 引用数: 0 h-index: 0机构: NIH, Bldg 10, Bethesda, MD 20892 USA Univ Florida, Powell Gene Therapy Ctr, Pediat, Gainesville, FL USARaben, Nina论文数: 0 引用数: 0 h-index: 0机构: NIH, Bldg 10, Bethesda, MD 20892 USA Univ Florida, Powell Gene Therapy Ctr, Pediat, Gainesville, FL USACorti, Manuela论文数: 0 引用数: 0 h-index: 0机构: Univ Florida, Powell Gene Therapy Ctr, Pediat, Gainesville, FL USA Univ Florida, Powell Gene Therapy Ctr, Pediat, Gainesville, FL USAByrne, Barry论文数: 0 引用数: 0 h-index: 0机构: Univ Florida, Powell Gene Therapy Ctr, Pediat, Gainesville, FL USA Univ Florida, Powell Gene Therapy Ctr, Pediat, Gainesville, FL USA
- [9] Intravital imaging of muscle damage and response to therapy in a model of Pompe diseaseMOLECULAR GENETICS AND METABOLISM, 2024, 141 (02)Meena, Naresh Kumar论文数: 0 引用数: 0 h-index: 0机构: NIH, Bethesda, MD USA NIH, Bethesda, MD USANg, Yeap论文数: 0 引用数: 0 h-index: 0机构: NCI, Bethesda, MD USA NIH, Bethesda, MD USAWeigert, Roberto论文数: 0 引用数: 0 h-index: 0机构: NCI, Bethesda, MD USA NIH, Bethesda, MD USAPuertollano, Rosa论文数: 0 引用数: 0 h-index: 0机构: NIH, Bethesda, MD USA NIH, Bethesda, MD USARaben, Nina论文数: 0 引用数: 0 h-index: 0机构: NIH, Bethesda, MD USA NIH, Bethesda, MD USA
- [10] Intravital imaging of muscle damage and response to therapy in a model of Pompe diseaseCLINICAL AND TRANSLATIONAL MEDICINE, 2024, 14 (03):Meena, Naresh K.论文数: 0 引用数: 0 h-index: 0机构: NHLBI, Cell & Dev Biol Ctr, NIH, Bethesda, MD USA NHLBI, Cell & Dev Biol Ctr, NIH, Bethesda, MD USANg, Yeap论文数: 0 引用数: 0 h-index: 0机构: NCI, Intravital Microscopy Core, Ctr Canc Res, NIH, Bethesda, MD USA NCI, Lab Cellular & Mol Biol, Ctr Canc Res, NIH, Bethesda, MD USA NHLBI, Cell & Dev Biol Ctr, NIH, Bethesda, MD USARandazzo, Davide论文数: 0 引用数: 0 h-index: 0机构: NIAMSD, Light Imaging Sect, Off Sci & Technol, NIH, Bethesda, MD USA NHLBI, Cell & Dev Biol Ctr, NIH, Bethesda, MD USAWeigert, Roberto论文数: 0 引用数: 0 h-index: 0机构: NCI, Intravital Microscopy Core, Ctr Canc Res, NIH, Bethesda, MD USA NCI, Lab Cellular & Mol Biol, Ctr Canc Res, NIH, Bethesda, MD USA NCI, CCR, NIH, Bldg 37,Room 2050-B, Bethesda, MD 20892 USA NHLBI, Cell & Dev Biol Ctr, NIH, Bethesda, MD USAPuertollano, Rosa论文数: 0 引用数: 0 h-index: 0机构: NHLBI, Cell & Dev Biol Ctr, NIH, Bethesda, MD USA NHLBI, NIH, 50 South Dr,Room 3537, Bethesda, MD 20892 USA NHLBI, Cell & Dev Biol Ctr, NIH, Bethesda, MD USARaben, Nina论文数: 0 引用数: 0 h-index: 0机构: NHLBI, Cell & Dev Biol Ctr, NIH, Bethesda, MD USA NHLBI, NIH, 50 South Dr,Room 3533, Bethesda, MD 20892 USA NHLBI, Cell & Dev Biol Ctr, NIH, Bethesda, MD USA