共 50 条
- [31] Gene therapy for X-linked myotubular myopathy: the challenges. (vol 22, 1089, 2023)LANCET NEUROLOGY, 2024, 23 (02): : E2 - E2Voermans, N. C.论文数: 0 引用数: 0 h-index: 0Ferreiro, A.论文数: 0 引用数: 0 h-index: 0Aartsma-Rus, A.论文数: 0 引用数: 0 h-index: 0Jungbluth, H.论文数: 0 引用数: 0 h-index: 0
- [32] Moving Forward: Gene Therapy for Intervertebral Disc DegenerationNEUROSPINE, 2020, 17 (01) : 17 - 18Han, In-Bo论文数: 0 引用数: 0 h-index: 0机构: CHA Univ, Sch Med, Dept Neurosurg, CHA Bundang Med Ctr, Seongnam, South Korea CHA Univ, Sch Med, Dept Neurosurg, CHA Bundang Med Ctr, Seongnam, South Korea
- [33] Gene Therapy for Muscular Dystrophy: Moving the Field ForwardPEDIATRIC NEUROLOGY, 2014, 51 (05) : 607 - 618Al-Zaidy, Samiah论文数: 0 引用数: 0 h-index: 0机构: Nationwide Childrens Hosp, Res Inst, Dept Pediat, Ctr Gene Therapy, Columbus, OH 43205 USA Nationwide Childrens Hosp, Res Inst, Dept Pediat, Ctr Gene Therapy, Columbus, OH 43205 USARodino-Klapac, Louise论文数: 0 引用数: 0 h-index: 0机构: Nationwide Childrens Hosp, Res Inst, Dept Pediat, Ctr Gene Therapy, Columbus, OH 43205 USA Nationwide Childrens Hosp, Res Inst, Dept Pediat, Ctr Gene Therapy, Columbus, OH 43205 USAMendell, Jerry R.论文数: 0 引用数: 0 h-index: 0机构: Nationwide Childrens Hosp, Res Inst, Dept Pediat, Ctr Gene Therapy, Columbus, OH 43205 USA Nationwide Childrens Hosp, Res Inst, Dept Pediat, Ctr Gene Therapy, Columbus, OH 43205 USA
- [34] Safety and efficacy of gene replacement therapy for X-linked myotubular myopathy (ASPIRO): a multinational, open-label, dose-escalation trialLANCET NEUROLOGY, 2023, 22 (12): : 1125 - 1139Shieh, Perry B.论文数: 0 引用数: 0 h-index: 0机构: David Geffen Sch Med UCLA, Dept Neurol, Los Angeles, CA USA David Geffen UCLA Sch Med, Dept Neurol, Los Angeles, CA 90095 USA David Geffen Sch Med UCLA, Dept Neurol, Los Angeles, CA USAKuntz, Nancy L.论文数: 0 引用数: 0 h-index: 0机构: Ann & Robert H Lurie Childrens Hosp Chicago, Div Neurol, Chicago, IL USA David Geffen Sch Med UCLA, Dept Neurol, Los Angeles, CA USADowling, James J.论文数: 0 引用数: 0 h-index: 0机构: Hosp Sick Children, Div Neurol, Toronto, ON, Canada David Geffen Sch Med UCLA, Dept Neurol, Los Angeles, CA USAMueller-Felber, Wolfgang论文数: 0 引用数: 0 h-index: 0机构: Ludwig Maximilian Univ Munich, Hauner Childrens Hosp, Dept Paediat Neurol & Dev Med, Munich, Germany David Geffen Sch Med UCLA, Dept Neurol, Los Angeles, CA USABonnemann, Carsten G.论文数: 0 引用数: 0 h-index: 0机构: Neuromuscular & Neurogenet Disorders Childhood Se, NINDS, NIH, Bethesda, MD USA David Geffen Sch Med UCLA, Dept Neurol, Los Angeles, CA USASeferian, Andreea M.论文数: 0 引用数: 0 h-index: 0机构: Hop Armand Trousseau, I Mot, Paris, France David Geffen Sch Med UCLA, Dept Neurol, Los Angeles, CA USAServais, Laurent论文数: 0 引用数: 0 h-index: 0机构: Hop Armand Trousseau, I Mot, Paris, France Univ Liege, Univ Hosp Liege, Neuromuscular Reference Ctr, Dept Pediat, Liege, Belgium Univ Oxford, MDUK Oxford Neuromuscular Ctr, NIHR Oxford Biomed Res Ctr, Dept Paediat, Oxford, England David Geffen Sch Med UCLA, Dept Neurol, Los Angeles, CA USASmith, Barbara K.论文数: 0 引用数: 0 h-index: 0机构: Univ Florida, Dept Pharmaceut, Gainesville, FL USA David Geffen Sch Med UCLA, Dept Neurol, Los Angeles, CA USAMuntoni, Francesco论文数: 0 引用数: 0 h-index: 0机构: UCL, NIHR Great Ormond St Hosp Biomed Res Ctr, Great Ormond St Inst Child Hlth, London, England David Geffen Sch Med UCLA, Dept Neurol, Los Angeles, CA USABlaschek, Astrid论文数: 0 引用数: 0 h-index: 0机构: Ludwig Maximilian Univ Munich, Hauner Childrens Hosp, Dept Paediat Neurol & Dev Med, Munich, Germany David Geffen Sch Med UCLA, Dept Neurol, Los Angeles, CA USAFoley, A. Reghan论文数: 0 引用数: 0 h-index: 0机构: Neuromuscular & Neurogenet Disorders Childhood Se, NINDS, NIH, Bethesda, MD USA David Geffen Sch Med UCLA, Dept Neurol, Los Angeles, CA USASaade, Dimah N.论文数: 0 引用数: 0 h-index: 0机构: Univ Iowa Hosp & Clin, Div Neurol, Iowa City, IA 52242 USA David Geffen Sch Med UCLA, Dept Neurol, Los Angeles, CA USANeuhaus, Sarah论文数: 0 引用数: 0 h-index: 0机构: Neuromuscular & Neurogenet Disorders Childhood Se, NINDS, NIH, Bethesda, MD USA David Geffen Sch Med UCLA, Dept Neurol, Los Angeles, CA USAAlfano, Lindsay N.论文数: 0 引用数: 0 h-index: 0机构: Nationwide Childrens Hosp, Abigail Wexner Res Inst, Columbus, OH USA David Geffen Sch Med UCLA, Dept Neurol, Los Angeles, CA USABeggs, Alan H.论文数: 0 引用数: 0 h-index: 0机构: Harvard Med Sch, Div Genet & Genom, Manton Ctr Orphan Dis Res, Boston Childrens Hosp, Boston, MA USA David Geffen Sch Med UCLA, Dept Neurol, Los Angeles, CA USABuj-Bello, Ana论文数: 0 引用数: 0 h-index: 0机构: Genethon, Evry, France Univ Evry, Univ Paris Saclay, Integrare Res unit UMR S951, INSERM, Evry, France David Geffen Sch Med UCLA, Dept Neurol, Los Angeles, CA USAChilders, Martin K.论文数: 0 引用数: 0 h-index: 0机构: Univ Washington, Inst Stem Cell & Regenerat Med, Dept Med Cardiol, Seattle, WA 98109 USA David Geffen Sch Med UCLA, Dept Neurol, Los Angeles, CA USADuong, Tina论文数: 0 引用数: 0 h-index: 0机构: Stanford Univ, Dept Neurol, Palo Alto, CA USA David Geffen Sch Med UCLA, Dept Neurol, Los Angeles, CA USAGraham, Robert J.论文数: 0 引用数: 0 h-index: 0机构: Harvard Med Sch, Boston Childrens Hosp, Div Crit Care, Dept Perioperat Med, Boston, MA USA David Geffen Sch Med UCLA, Dept Neurol, Los Angeles, CA USAJain, Minal论文数: 0 引用数: 0 h-index: 0机构: NIH, Dept Rehabil Med, Hatfield Clin Res Ctr, Bethesda, MD 20892 USA David Geffen Sch Med UCLA, Dept Neurol, Los Angeles, CA USACoats, Julie论文数: 0 引用数: 0 h-index: 0机构: Astellas Gene Therapies, San Francisco, CA USA David Geffen Sch Med UCLA, Dept Neurol, Los Angeles, CA USAMacbean, Vicky论文数: 0 引用数: 0 h-index: 0机构: Brunel Univ London, Dept Hlth Sci, London, England David Geffen Sch Med UCLA, Dept Neurol, Los Angeles, CA USAJames, Emma S.论文数: 0 引用数: 0 h-index: 0机构: Astellas Gene Therapies, San Francisco, CA USA David Geffen Sch Med UCLA, Dept Neurol, Los Angeles, CA USALee, Jun论文数: 0 引用数: 0 h-index: 0机构: Astellas Gene Therapies, San Francisco, CA USA David Geffen Sch Med UCLA, Dept Neurol, Los Angeles, CA USAMavilio, Fulvio论文数: 0 引用数: 0 h-index: 0机构: Astellas Gene Therapies, San Francisco, CA USA Univ Modena & Reggio Emilia, Dept Life Sci, Modena, Italy David Geffen Sch Med UCLA, Dept Neurol, Los Angeles, CA USAMiller, Weston论文数: 0 引用数: 0 h-index: 0机构: Astellas Gene Therapies, San Francisco, CA USA David Geffen Sch Med UCLA, Dept Neurol, Los Angeles, CA USAVarfaj, Fatbardha论文数: 0 引用数: 0 h-index: 0机构: Astellas Gene Therapies, San Francisco, CA USA David Geffen Sch Med UCLA, Dept Neurol, Los Angeles, CA USAMurtagh, Michael论文数: 0 引用数: 0 h-index: 0机构: Astellas Gene Therapies, San Francisco, CA USA David Geffen Sch Med UCLA, Dept Neurol, Los Angeles, CA USAHan, Cong论文数: 0 引用数: 0 h-index: 0机构: Astellas Pharm Global Dev, Northbrook, IL 60062 USA David Geffen Sch Med UCLA, Dept Neurol, Los Angeles, CA USANoursalehi, Mojtaba论文数: 0 引用数: 0 h-index: 0机构: Astellas Gene Therapies, San Francisco, CA USA David Geffen Sch Med UCLA, Dept Neurol, Los Angeles, CA USALawlor, Michael W.论文数: 0 引用数: 0 h-index: 0机构: Med Coll Wisconsin, Dept Pathol & Lab Med, Milwaukee, WI 53226 USA Diverge Translat Sci Lab, Milwaukee, WI USA David Geffen Sch Med UCLA, Dept Neurol, Los Angeles, CA USAPrasad, Suyash论文数: 0 引用数: 0 h-index: 0机构: Astellas Gene Therapies, San Francisco, CA USA David Geffen Sch Med UCLA, Dept Neurol, Los Angeles, CA USARico, Salvador论文数: 0 引用数: 0 h-index: 0机构: Astellas Gene Therapies, San Francisco, CA USA David Geffen Sch Med UCLA, Dept Neurol, Los Angeles, CA USA
- [35] Gene therapy for hemophilia: Progress to date and challenges moving forwardTRANSFUSION AND APHERESIS SCIENCE, 2019, 58 (05) : 602 - 612Gollomp, Kandace L.论文数: 0 引用数: 0 h-index: 0机构: Childrens Hosp Philadelphia, Div Hematol, Philadelphia, PA 19104 USA Univ Penn, Dept Pediat, Philadelphia, PA 19104 USA Childrens Hosp Philadelphia, Div Hematol, Philadelphia, PA 19104 USADoshi, Bhavya S.论文数: 0 引用数: 0 h-index: 0机构: Childrens Hosp Philadelphia, Div Hematol, Philadelphia, PA 19104 USA Univ Penn, Dept Pediat, Philadelphia, PA 19104 USA Childrens Hosp Philadelphia, Div Hematol, Philadelphia, PA 19104 USAArruda, Valder R.论文数: 0 引用数: 0 h-index: 0机构: Childrens Hosp Philadelphia, Div Hematol, Philadelphia, PA 19104 USA Univ Penn, Dept Pediat, Philadelphia, PA 19104 USA Childrens Hosp Philadelphia, Raymond G Perelman Ctr Cellular & Mol Therapeut, Philadelphia, PA 19104 USA Childrens Hosp Philadelphia, Div Hematol, Philadelphia, PA 19104 USA
- [36] INCEPTUS Natural History, Run-in Study for Gene Replacement Clinical Trial in X-Linked Myotubular MyopathyJOURNAL OF NEUROMUSCULAR DISEASES, 2022, 9 (04) : 503 - 516Dowling, James J.论文数: 0 引用数: 0 h-index: 0机构: Hosp Sick Children, 555 Univ Ave, Toronto, ON M5G 1X8, Canada Hosp Sick Children, 555 Univ Ave, Toronto, ON M5G 1X8, CanadaMueller-Felber, Wolfgang论文数: 0 引用数: 0 h-index: 0机构: Klinikum Univ Munchen, Dr V Haunersches Kinderspital, Munich, Germany Hosp Sick Children, 555 Univ Ave, Toronto, ON M5G 1X8, CanadaSmith, Barbara K.论文数: 0 引用数: 0 h-index: 0机构: Univ Florida, Gainesville, FL USA Hosp Sick Children, 555 Univ Ave, Toronto, ON M5G 1X8, CanadaBonnemann, Carsten G.论文数: 0 引用数: 0 h-index: 0机构: NINDS, NIH, Bethesda, MD USA Hosp Sick Children, 555 Univ Ave, Toronto, ON M5G 1X8, CanadaKuntz, Nancy L.论文数: 0 引用数: 0 h-index: 0机构: Ann & Robert H Lurie Childrens Hosp Chicago, Chicago, IL 60611 USA Hosp Sick Children, 555 Univ Ave, Toronto, ON M5G 1X8, CanadaMuntoni, Francesco论文数: 0 引用数: 0 h-index: 0机构: UCL, Natl Inst Hlth Res NIHR, Great Ormond St GOS Hosp, Biomed Res Ctr,Inst Child Hlth, London, England Hosp Sick Children, 555 Univ Ave, Toronto, ON M5G 1X8, CanadaServais, Laurent论文数: 0 引用数: 0 h-index: 0机构: Hop Armand Trousseau, I Mot, Paris, France Univ Hosp Liege, Reference Ctr Neuromuscular Dis, Dept Pediat, Div Child Neurol, Liege, Belgium Univ Liege, Liege, Belgium Hosp Sick Children, 555 Univ Ave, Toronto, ON M5G 1X8, CanadaAlfano, Lindsay N.论文数: 0 引用数: 0 h-index: 0机构: Nationwide Childrens Hosp, Columbus, OH USA Hosp Sick Children, 555 Univ Ave, Toronto, ON M5G 1X8, CanadaBeggs, Alan H.论文数: 0 引用数: 0 h-index: 0机构: Harvard Med Sch, Boston Childrens Hosp, Boston, MA USA Hosp Sick Children, 555 Univ Ave, Toronto, ON M5G 1X8, CanadaBilder, Deborah A.论文数: 0 引用数: 0 h-index: 0机构: Univ Utah, Salt Lake City, UT USA Hosp Sick Children, 555 Univ Ave, Toronto, ON M5G 1X8, CanadaBlaschek, Astrid论文数: 0 引用数: 0 h-index: 0机构: Klinikum Univ Munchen, Dr V Haunersches Kinderspital, Munich, Germany Hosp Sick Children, 555 Univ Ave, Toronto, ON M5G 1X8, Canada论文数: 引用数: h-index:机构:Graham, Robert J.论文数: 0 引用数: 0 h-index: 0机构: Harvard Med Sch, Boston Childrens Hosp, Boston, MA USA Hosp Sick Children, 555 Univ Ave, Toronto, ON M5G 1X8, CanadaJain, Minal论文数: 0 引用数: 0 h-index: 0机构: NIH, Hatfield Clin Res Ctr, Bethesda, MD USA Hosp Sick Children, 555 Univ Ave, Toronto, ON M5G 1X8, CanadaLawlor, Michael W.论文数: 0 引用数: 0 h-index: 0机构: Med Coll Wisconsin, Milwaukee, WI 53226 USA Hosp Sick Children, 555 Univ Ave, Toronto, ON M5G 1X8, CanadaLee, Jun论文数: 0 引用数: 0 h-index: 0机构: Astellas Gene Therapies, San Francisco, CA USA Hosp Sick Children, 555 Univ Ave, Toronto, ON M5G 1X8, CanadaCoats, Julie论文数: 0 引用数: 0 h-index: 0机构: Astellas Gene Therapies, San Francisco, CA USA Hosp Sick Children, 555 Univ Ave, Toronto, ON M5G 1X8, CanadaLilien, Charlotte论文数: 0 引用数: 0 h-index: 0机构: Hop Armand Trousseau, I Mot, Paris, France Hosp Sick Children, 555 Univ Ave, Toronto, ON M5G 1X8, CanadaLowes, Linda P.论文数: 0 引用数: 0 h-index: 0机构: Nationwide Childrens Hosp, Columbus, OH USA Hosp Sick Children, 555 Univ Ave, Toronto, ON M5G 1X8, CanadaMacBean, Victoria论文数: 0 引用数: 0 h-index: 0机构: Brunel Univ London, London, England Kings Coll London, London, England Hosp Sick Children, 555 Univ Ave, Toronto, ON M5G 1X8, CanadaNeuhaus, Sarah论文数: 0 引用数: 0 h-index: 0机构: NINDS, NIH, Bethesda, MD USA Hosp Sick Children, 555 Univ Ave, Toronto, ON M5G 1X8, CanadaNoursalehi, Mojtaba论文数: 0 引用数: 0 h-index: 0机构: Astellas Gene Therapies, San Francisco, CA USA Hosp Sick Children, 555 Univ Ave, Toronto, ON M5G 1X8, Canada论文数: 引用数: h-index:机构:Finlay, Caroline论文数: 0 引用数: 0 h-index: 0机构: Astellas Gene Therapies, San Francisco, CA USA Univ Louisville, Louisville, KY 40292 USA Hosp Sick Children, 555 Univ Ave, Toronto, ON M5G 1X8, CanadaChristensen, Sarah论文数: 0 引用数: 0 h-index: 0机构: Astellas Gene Therapies, San Francisco, CA USA Univ Louisville, Louisville, KY 40292 USA Hosp Sick Children, 555 Univ Ave, Toronto, ON M5G 1X8, CanadaRafferty, Gerrard论文数: 0 引用数: 0 h-index: 0机构: Kings Coll London, London, England Hosp Sick Children, 555 Univ Ave, Toronto, ON M5G 1X8, CanadaSeferian, Andreea M.论文数: 0 引用数: 0 h-index: 0机构: Hop Armand Trousseau, I Mot, Paris, France Hosp Sick Children, 555 Univ Ave, Toronto, ON M5G 1X8, CanadaTsuchiya, Etsuko论文数: 0 引用数: 0 h-index: 0机构: Hosp Sick Children, 555 Univ Ave, Toronto, ON M5G 1X8, Canada Hosp Sick Children, 555 Univ Ave, Toronto, ON M5G 1X8, CanadaJames, Emma S.论文数: 0 引用数: 0 h-index: 0机构: Astellas Gene Therapies, San Francisco, CA USA Univ Louisville, Louisville, KY 40292 USA Hosp Sick Children, 555 Univ Ave, Toronto, ON M5G 1X8, CanadaMiller, Weston论文数: 0 引用数: 0 h-index: 0机构: Astellas Gene Therapies, San Francisco, CA USA Hosp Sick Children, 555 Univ Ave, Toronto, ON M5G 1X8, CanadaSepulveda, Bryan论文数: 0 引用数: 0 h-index: 0机构: Astellas Gene Therapies, San Francisco, CA USA Hosp Sick Children, 555 Univ Ave, Toronto, ON M5G 1X8, CanadaVila, Maria Candida论文数: 0 引用数: 0 h-index: 0机构: Astellas Gene Therapies, San Francisco, CA USA Hosp Sick Children, 555 Univ Ave, Toronto, ON M5G 1X8, CanadaPrasad, Suyash论文数: 0 引用数: 0 h-index: 0机构: Astellas Gene Therapies, San Francisco, CA USA Hosp Sick Children, 555 Univ Ave, Toronto, ON M5G 1X8, CanadaRico, Salvador论文数: 0 引用数: 0 h-index: 0机构: Astellas Gene Therapies, San Francisco, CA USA Hosp Sick Children, 555 Univ Ave, Toronto, ON M5G 1X8, CanadaShieh, Perry B.论文数: 0 引用数: 0 h-index: 0机构: Univ Calif Los Angeles, Los Angeles, CA USA Hosp Sick Children, 555 Univ Ave, Toronto, ON M5G 1X8, Canada
- [37] Systemic AAV8-Mediated Gene Therapy Drives Whole-Body Correction of Myotubular Myopathy in DogsMOLECULAR THERAPY, 2017, 25 (04) : 839 - 854Mack, David L.论文数: 0 引用数: 0 h-index: 0机构: Univ Washington, Dept Rehabil Med, Seattle, WA 98104 USA Univ Washington, Sch Med, lnst Stem Cell & Regenerat Med, Seattle, WA 98107 USA Univ Washington, Dept Rehabil Med, Seattle, WA 98104 USAPoulard, Karine论文数: 0 引用数: 0 h-index: 0机构: Genethon, F-91000 Evry, France INSEAD, UMR S951, F-91002 Evry, France Univ Washington, Dept Rehabil Med, Seattle, WA 98104 USAGoddard, Melissa A.论文数: 0 引用数: 0 h-index: 0机构: Univ Washington, Sch Med, lnst Stem Cell & Regenerat Med, Seattle, WA 98107 USA Univ Washington, Dept Rehabil Med, Seattle, WA 98104 USALatoumerie, Virginie论文数: 0 引用数: 0 h-index: 0机构: Genethon, F-91000 Evry, France INSEAD, UMR S951, F-91002 Evry, France Univ Washington, Dept Rehabil Med, Seattle, WA 98104 USASnyder, Jessica M.论文数: 0 引用数: 0 h-index: 0机构: Univ Washington, Dept Comparat Med, Seattle, WA 98195 USA Univ Washington, Dept Rehabil Med, Seattle, WA 98104 USAGrange, Robert W.论文数: 0 引用数: 0 h-index: 0机构: Virginia Polytech Inst & State Univ, Dept Human Nutr Foods & Exercise, Blacksburg, VA 24061 USA Univ Washington, Dept Rehabil Med, Seattle, WA 98104 USAElverman, Matthew R.论文数: 0 引用数: 0 h-index: 0机构: Univ Washington, Sch Med, lnst Stem Cell & Regenerat Med, Seattle, WA 98107 USA Univ Washington, Dept Rehabil Med, Seattle, WA 98104 USADenard, Jerome论文数: 0 引用数: 0 h-index: 0机构: Genethon, F-91000 Evry, France Univ Washington, Dept Rehabil Med, Seattle, WA 98104 USAVeron, Philippe论文数: 0 引用数: 0 h-index: 0机构: Genethon, F-91000 Evry, France INSEAD, UMR S951, F-91002 Evry, France Univ Washington, Dept Rehabil Med, Seattle, WA 98104 USABuscara, Laurine论文数: 0 引用数: 0 h-index: 0机构: Genethon, F-91000 Evry, France INSEAD, UMR S951, F-91002 Evry, France Univ Washington, Dept Rehabil Med, Seattle, WA 98104 USALe Bec, Christine论文数: 0 引用数: 0 h-index: 0机构: Genethon, F-91000 Evry, France Univ Washington, Dept Rehabil Med, Seattle, WA 98104 USAHogrel, Jean-Yves论文数: 0 引用数: 0 h-index: 0机构: Inst Myol, Neuromuscular Physiol & Evaluat Lab, F-75651 Paris, France Univ Washington, Dept Rehabil Med, Seattle, WA 98104 USABrezovec, Annie G.论文数: 0 引用数: 0 h-index: 0机构: Univ Washington, Dept Rehabil Med, Seattle, WA 98104 USAMeng, Hui论文数: 0 引用数: 0 h-index: 0机构: Med Coll Wisconsin, Dept Pathol & Lab Med, Div Pediat Pathol, Milwaukee, WI 53226 USA Univ Washington, Dept Rehabil Med, Seattle, WA 98104 USAYang, Lin论文数: 0 引用数: 0 h-index: 0机构: Univ Florida, Dept Biomed Engn, Gainesville, FL 32611 USA Univ Washington, Dept Rehabil Med, Seattle, WA 98104 USALiu, Fujun论文数: 0 引用数: 0 h-index: 0机构: Univ Florida, Dept Biomed Engn, Gainesville, FL 32611 USA Univ Washington, Dept Rehabil Med, Seattle, WA 98104 USAO'Callaghan, Michael论文数: 0 引用数: 0 h-index: 0机构: Audentes Therapeut, San Francisco, CA 94108 USA Univ Washington, Dept Rehabil Med, Seattle, WA 98104 USAGopal, Nikhil论文数: 0 引用数: 0 h-index: 0机构: Univ Washington, Dept Biomed Informat & Med Educ, Seattle, WA 98019 USA Univ Washington, Dept Rehabil Med, Seattle, WA 98104 USAKelly, Valerie E.论文数: 0 引用数: 0 h-index: 0机构: Univ Washington, Dept Rehabil Med, Seattle, WA 98104 USA Univ Washington, Dept Rehabil Med, Seattle, WA 98104 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 Washington, Dept Rehabil Med, Seattle, WA 98104 USAStrande, Jennifer L.论文数: 0 引用数: 0 h-index: 0机构: Med Coll Wisconsin, Dept Med, Milwaukee, WI 53226 USA Med Coll Wisconsin, Dept Cell Biol, Neurobiol & Anat, Milwaukee, WI 53226 USA Med Coll Wisconsin, Cardiovasc Ctr, Milwaukee, WI 53226 USA Univ Washington, Dept Rehabil Med, Seattle, WA 98104 USAMavilio, Fulvio论文数: 0 引用数: 0 h-index: 0机构: Genethon, F-91000 Evry, France INSEAD, UMR S951, F-91002 Evry, France Univ Washington, Dept Rehabil Med, Seattle, WA 98104 USABeggs, Alan H.论文数: 0 引用数: 0 h-index: 0机构: Harvard Med Sch, Boston Childrens Hosp, Manton Ctr Orphan Dis Res, Boston, MA 02115 USA Univ Washington, Dept Rehabil Med, Seattle, WA 98104 USAMingozzi, Federico论文数: 0 引用数: 0 h-index: 0机构: Genethon, F-91000 Evry, France INSEAD, UMR S951, F-91002 Evry, France Univ Paris 06, Inst Myol, F-75005 Paris, France Univ Washington, Dept Rehabil Med, Seattle, WA 98104 USALawlor, Michael W.论文数: 0 引用数: 0 h-index: 0机构: Med Coll Wisconsin, Dept Pathol & Lab Med, Div Pediat Pathol, Milwaukee, WI 53226 USA Univ Washington, Dept Rehabil Med, Seattle, WA 98104 USABuj-Bello, Ana论文数: 0 引用数: 0 h-index: 0机构: Genethon, F-91000 Evry, France INSEAD, UMR S951, F-91002 Evry, France Univ Washington, Dept Rehabil Med, Seattle, WA 98104 USAChilders, Martin K.论文数: 0 引用数: 0 h-index: 0机构: Univ Washington, Sch Med, lnst Stem Cell & Regenerat Med, Seattle, WA 98107 USA Univ Washington, Dept Rehabil Med, Seattle, WA 98104 USA
- [38] Restricting MTM1 Transgene Expression to Skeletal Muscle in AAV-Mediated Gene Therapy for Myotubular MyopathyMOLECULAR THERAPY, 2013, 21 : S231 - S232Joubert, Romain论文数: 0 引用数: 0 h-index: 0机构: Genethon, Dept Res & Dev, Evry, France Genethon, Dept Res & Dev, Evry, FranceMoal, Christelle论文数: 0 引用数: 0 h-index: 0机构: Genethon, Dept Res & Dev, Evry, France Genethon, Dept Res & Dev, Evry, FranceVignaud, Alban论文数: 0 引用数: 0 h-index: 0机构: Genethon, Dept Res & Dev, Evry, France Genethon, Dept Res & Dev, Evry, FranceMartin, Samia论文数: 0 引用数: 0 h-index: 0机构: Genethon, Dept Res & Dev, Evry, France Genethon, Dept Res & Dev, Evry, FranceRichard, Isabelle论文数: 0 引用数: 0 h-index: 0机构: Genethon, Dept Res & Dev, Evry, France Genethon, Dept Res & Dev, Evry, FranceMoullier, Philippe论文数: 0 引用数: 0 h-index: 0机构: Genethon, Dept Res & Dev, Evry, France Genethon, Dept Res & Dev, Evry, FranceBeggs, Alan H.论文数: 0 引用数: 0 h-index: 0机构: Harvard Univ, Sch Med, Div Genet, Boston, MA USA Harvard Univ, Sch Med, Childrens Hosp Boston, Program Genom,Manton Ctr Orphan Dis Res, Boston, MA USA Genethon, Dept Res & Dev, Evry, FranceChilders, Martin K.论文数: 0 引用数: 0 h-index: 0机构: Univ Washington, Dept Rehabil Med, Inst Stem Cell & Regenerat Med, Seattle, WA 98195 USA Genethon, Dept Res & Dev, Evry, FranceBuj-Bello, Anna论文数: 0 引用数: 0 h-index: 0机构: Genethon, Dept Res & Dev, Evry, France Genethon, Dept Res & Dev, Evry, France
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