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- [31] Safe and Effective Gene Therapy for Murine Wiskott-Aldrich Syndrome Using an Insulated Lentiviral VectorMOLECULAR THERAPY-METHODS & CLINICAL DEVELOPMENT, 2017, 4 : 1 - 16Singh, Swati论文数: 0 引用数: 0 h-index: 0机构: Seattle Childrens Res Inst, Ctr Immun & Immunotherapies, 1900 Ninth Ave, Seattle, WA 98101 USA Seattle Childrens Res Inst, Program Cell & Gene Therapy, Seattle, WA 98101 USA Seattle Childrens Res Inst, Ctr Immun & Immunotherapies, 1900 Ninth Ave, Seattle, WA 98101 USAKhan, Iram论文数: 0 引用数: 0 h-index: 0机构: Seattle Childrens Res Inst, Ctr Immun & Immunotherapies, 1900 Ninth Ave, Seattle, WA 98101 USA Seattle Childrens Res Inst, Program Cell & Gene Therapy, Seattle, WA 98101 USA Seattle Childrens Res Inst, Ctr Immun & Immunotherapies, 1900 Ninth Ave, Seattle, WA 98101 USAKhim, Socheath论文数: 0 引用数: 0 h-index: 0机构: Seattle Childrens Res Inst, Ctr Immun & Immunotherapies, 1900 Ninth Ave, Seattle, WA 98101 USA Seattle Childrens Res Inst, Program Cell & Gene Therapy, Seattle, WA 98101 USA Seattle Childrens Res Inst, Ctr Immun & Immunotherapies, 1900 Ninth Ave, Seattle, WA 98101 USASeymour, Brenda论文数: 0 引用数: 0 h-index: 0机构: Seattle Childrens Res Inst, Ctr Immun & Immunotherapies, 1900 Ninth Ave, Seattle, WA 98101 USA Seattle Childrens Res Inst, Program Cell & Gene Therapy, Seattle, WA 98101 USA Seattle Childrens Res Inst, Ctr Immun & Immunotherapies, 1900 Ninth Ave, Seattle, WA 98101 USASommer, Karen论文数: 0 引用数: 0 h-index: 0机构: Seattle Childrens Res Inst, Ctr Immun & Immunotherapies, 1900 Ninth Ave, Seattle, WA 98101 USA Seattle Childrens Res Inst, Program Cell & Gene Therapy, Seattle, WA 98101 USA Seattle Childrens Res Inst, Ctr Immun & Immunotherapies, 1900 Ninth Ave, Seattle, WA 98101 USAWielgosz, Matthew论文数: 0 引用数: 0 h-index: 0机构: St Jude Childrens Res Hosp, Dept Hematol, Memphis, TN 38105 USA Seattle Childrens Res Inst, Ctr Immun & Immunotherapies, 1900 Ninth Ave, Seattle, WA 98101 USANorgaard, Zachary论文数: 0 引用数: 0 h-index: 0机构: Fred Hutchinson Canc Res Ctr, Clin Res Div, Seattle, WA 98109 USA Seattle Childrens Res Inst, Ctr Immun & Immunotherapies, 1900 Ninth Ave, Seattle, WA 98101 USAKiem, Hans-Peter论文数: 0 引用数: 0 h-index: 0机构: Fred Hutchinson Canc Res Ctr, Clin Res Div, Seattle, WA 98109 USA Univ Washington, Dept Pathol, Seattle, WA 98105 USA Seattle Childrens Res Inst, Ctr Immun & Immunotherapies, 1900 Ninth Ave, Seattle, WA 98101 USAAdair, Jennifer论文数: 0 引用数: 0 h-index: 0机构: Fred Hutchinson Canc Res Ctr, Clin Res Div, Seattle, WA 98109 USA Univ Washington, Dept Med Oncol, Seattle, WA 98105 USA Seattle Childrens Res Inst, Ctr Immun & Immunotherapies, 1900 Ninth Ave, Seattle, WA 98101 USALiggitt, Denny论文数: 0 引用数: 0 h-index: 0机构: Univ Washington, Dept Comparat Med, Seattle, WA 98105 USA Seattle Childrens Res Inst, Ctr Immun & Immunotherapies, 1900 Ninth Ave, Seattle, WA 98101 USANienhuis, Arthur论文数: 0 引用数: 0 h-index: 0机构: St Jude Childrens Res Hosp, Dept Hematol, Memphis, TN 38105 USA Seattle Childrens Res Inst, Ctr Immun & Immunotherapies, 1900 Ninth Ave, Seattle, WA 98101 USARawlings, David J.论文数: 0 引用数: 0 h-index: 0机构: Seattle Childrens Res Inst, Ctr Immun & Immunotherapies, 1900 Ninth Ave, Seattle, WA 98101 USA Seattle Childrens Res Inst, Program Cell & Gene Therapy, Seattle, WA 98101 USA Univ Washington, Dept Pediat, Seattle, WA 98105 USA Univ Washington, Dept Immunol, Seattle, WA 98105 USA Seattle Childrens Res Inst, Ctr Immun & Immunotherapies, 1900 Ninth Ave, Seattle, WA 98101 USA
- [32] Development of a rabbit model of Wiskott-Aldrich syndromeFASEB JOURNAL, 2021, 35 (02):Zhou, Juanjuan论文数: 0 引用数: 0 h-index: 0机构: Univ Sci & Technol China, Sch Life Sci, Hefei, Peoples R China Chinese Acad Sci, South China Inst Stem Cell Biol & Regenerat Med, Guangzhou Inst Biomed, CAS Key Lab Regenerat Biol,Guangdong Prov Key Lab, 90 Kaiyuan Ave,Guangzhou Sci Pk, Guangzhou 510530, Peoples R China Chinese Acad Sci, South China Inst Stem Cell Biol & Regenerat Med, Guangzhou Inst Hlth, CAS Key Lab Regenerat Biol,Guangdong Prov Key Lab, 90 Kaiyuan Ave,Guangzhou Sci Pk, Guangzhou 510530, Peoples R China Univ Sci & Technol China, Sch Life Sci, Hefei, Peoples R ChinaYan, Quanmei论文数: 0 引用数: 0 h-index: 0机构: Chinese Acad Sci, South China Inst Stem Cell Biol & Regenerat Med, Guangzhou Inst Biomed, CAS Key Lab Regenerat Biol,Guangdong Prov Key Lab, 90 Kaiyuan Ave,Guangzhou Sci Pk, Guangzhou 510530, Peoples R China Chinese Acad Sci, South China Inst Stem Cell Biol & Regenerat Med, Guangzhou Inst Hlth, CAS Key Lab Regenerat Biol,Guangdong Prov Key Lab, 90 Kaiyuan Ave,Guangzhou Sci Pk, Guangzhou 510530, Peoples R China Univ Sci & Technol China, Sch Life Sci, Hefei, Peoples R ChinaTang, Chengcheng论文数: 0 引用数: 0 h-index: 0机构: Wuyi Univ, Sch Biotechnol & Hlth Sci, 22 Dongcheng Village, Jiangmen 529020, Guangdong, Peoples R China Univ Sci & Technol China, Sch Life Sci, Hefei, Peoples R ChinaLiao, Yuan论文数: 0 引用数: 0 h-index: 0机构: Chinese Acad Sci, South China Inst Stem Cell Biol & Regenerat Med, Guangzhou Inst Biomed, CAS Key Lab Regenerat Biol,Guangdong Prov Key Lab, 90 Kaiyuan Ave,Guangzhou Sci Pk, Guangzhou 510530, Peoples R China Chinese Acad Sci, South China Inst Stem Cell Biol & Regenerat Med, Guangzhou Inst Hlth, CAS Key Lab Regenerat Biol,Guangdong Prov Key Lab, 90 Kaiyuan Ave,Guangzhou Sci Pk, Guangzhou 510530, Peoples R China Anhui Univ, Inst Phys Sci & Informat Technol, Hefei, Peoples R China Univ Sci & Technol China, Sch Life Sci, Hefei, Peoples R ChinaZhang, Quanjun论文数: 0 引用数: 0 h-index: 0机构: Chinese Acad Sci, South China Inst Stem Cell Biol & Regenerat Med, Guangzhou Inst Biomed, CAS Key Lab Regenerat Biol,Guangdong Prov Key Lab, 90 Kaiyuan Ave,Guangzhou Sci Pk, Guangzhou 510530, Peoples R China Chinese Acad Sci, South China Inst Stem Cell Biol & Regenerat Med, Guangzhou Inst Hlth, CAS Key Lab Regenerat Biol,Guangdong Prov Key Lab, 90 Kaiyuan Ave,Guangzhou Sci Pk, Guangzhou 510530, Peoples R China Guangzhou Regenerat Med & Hlth Guangdong Lab, Bioland Lab, Guangzhou, Peoples R China Chinese Acad Sci, Inst Stem Cell & Regenerat, Beijing, Peoples R China Chinese Acad Med Sci 2019RU015, Res Unit Generat Large Anim Dis Models, Guangzhou, Peoples R China Univ Sci & Technol China, Sch Life Sci, Hefei, Peoples R ChinaWang, Xiaomin论文数: 0 引用数: 0 h-index: 0机构: Chinese Acad Sci, South China Inst Stem Cell Biol & Regenerat Med, Guangzhou Inst Biomed, CAS Key Lab Regenerat Biol,Guangdong Prov Key Lab, 90 Kaiyuan Ave,Guangzhou Sci Pk, Guangzhou 510530, Peoples R China Chinese Acad Sci, South China Inst Stem Cell Biol & Regenerat Med, Guangzhou Inst Hlth, CAS Key Lab Regenerat Biol,Guangdong Prov Key Lab, 90 Kaiyuan Ave,Guangzhou Sci Pk, Guangzhou 510530, Peoples R China Univ Sci & Technol China, Sch Life Sci, Hefei, Peoples R ChinaZhou, Xiaoqing论文数: 0 引用数: 0 h-index: 0机构: Wuyi Univ, Sch Biotechnol & Hlth Sci, 22 Dongcheng Village, Jiangmen 529020, Guangdong, Peoples R China Univ Sci & Technol China, Sch Life Sci, Hefei, Peoples R ChinaLai, Liangxue论文数: 0 引用数: 0 h-index: 0机构: Chinese Acad Sci, South China Inst Stem Cell Biol & Regenerat Med, Guangzhou Inst Biomed, CAS Key Lab Regenerat Biol,Guangdong Prov Key Lab, 90 Kaiyuan Ave,Guangzhou Sci Pk, Guangzhou 510530, Peoples R China Chinese Acad Sci, South China Inst Stem Cell Biol & Regenerat Med, Guangzhou Inst Hlth, CAS Key Lab Regenerat Biol,Guangdong Prov Key Lab, 90 Kaiyuan Ave,Guangzhou Sci Pk, Guangzhou 510530, Peoples R China Guangzhou Regenerat Med & Hlth Guangdong Lab, Bioland Lab, Guangzhou, Peoples R China Chinese Acad Sci, Inst Stem Cell & Regenerat, Beijing, Peoples R China Chinese Acad Med Sci 2019RU015, Res Unit Generat Large Anim Dis Models, Guangzhou, Peoples R China Univ Sci & Technol China, Sch Life Sci, Hefei, Peoples R ChinaZou, Qingjian论文数: 0 引用数: 0 h-index: 0机构: Wuyi Univ, Sch Biotechnol & Hlth Sci, 22 Dongcheng Village, Jiangmen 529020, Guangdong, Peoples R China Univ Sci & Technol China, Sch Life Sci, Hefei, Peoples R China
- [33] Targeted gene correction of human hematopoietic stem cells for the treatment of Wiskott - Aldrich SyndromeNature Communications, 11Rajeev Rai论文数: 0 引用数: 0 h-index: 0机构: University College London,Infection, Immunity and Inflammation Research and Teaching Department, Great Ormond Street Institute of Child HealthMarianna Romito论文数: 0 引用数: 0 h-index: 0机构: University College London,Infection, Immunity and Inflammation Research and Teaching Department, Great Ormond Street Institute of Child HealthElizabeth Rivers论文数: 0 引用数: 0 h-index: 0机构: University College London,Infection, Immunity and Inflammation Research and Teaching Department, Great Ormond Street Institute of Child HealthGiandomenico Turchiano论文数: 0 引用数: 0 h-index: 0机构: University College London,Infection, Immunity and Inflammation Research and Teaching Department, Great Ormond Street Institute of Child HealthGeorges Blattner论文数: 0 引用数: 0 h-index: 0机构: University College London,Infection, Immunity and Inflammation Research and Teaching Department, Great Ormond Street Institute of Child HealthWinston Vetharoy论文数: 0 引用数: 0 h-index: 0机构: University College London,Infection, Immunity and Inflammation Research and Teaching Department, Great Ormond Street Institute of Child HealthDariusz Ladon论文数: 0 引用数: 0 h-index: 0机构: University College London,Infection, Immunity and Inflammation Research and Teaching Department, Great Ormond Street Institute of Child HealthGeoffroy Andrieux论文数: 0 引用数: 0 h-index: 0机构: University College London,Infection, Immunity and Inflammation Research and Teaching Department, Great Ormond Street Institute of Child HealthFang Zhang论文数: 0 引用数: 0 h-index: 0机构: University College London,Infection, Immunity and Inflammation Research and Teaching Department, Great Ormond Street Institute of Child HealthMarta Zinicola论文数: 0 引用数: 0 h-index: 0机构: University College London,Infection, Immunity and Inflammation Research and Teaching Department, Great Ormond Street Institute of Child HealthDiego Leon-Rico论文数: 0 引用数: 0 h-index: 0机构: University College London,Infection, Immunity and Inflammation Research and Teaching Department, Great Ormond Street Institute of Child HealthGiorgia Santilli论文数: 0 引用数: 0 h-index: 0机构: University College London,Infection, Immunity and Inflammation Research and Teaching Department, Great Ormond Street Institute of Child HealthAdrian J. Thrasher论文数: 0 引用数: 0 h-index: 0机构: University College London,Infection, Immunity and Inflammation Research and Teaching Department, Great Ormond Street Institute of Child HealthAlessia Cavazza论文数: 0 引用数: 0 h-index: 0机构: University College London,Infection, Immunity and Inflammation Research and Teaching Department, Great Ormond Street Institute of Child Health
- [34] Genome editing for primary immunodeficiencies: A therapeutic perspective on Wiskott-Aldrich syndromeFRONTIERS IN IMMUNOLOGY, 2022, 13Naseem, Asma论文数: 0 引用数: 0 h-index: 0机构: UCL, Great Ormond St Inst Child Hlth, Infect Immun & Inflammat Res & Teaching Dept, London, England UCL, Great Ormond St Inst Child Hlth, Infect Immun & Inflammat Res & Teaching Dept, London, EnglandSteinberg, Zohar论文数: 0 引用数: 0 h-index: 0机构: UCL, Great Ormond St Inst Child Hlth, Infect Immun & Inflammat Res & Teaching Dept, London, England UCL, Great Ormond St Inst Child Hlth, Infect Immun & Inflammat Res & Teaching Dept, London, EnglandCavazza, Alessia论文数: 0 引用数: 0 h-index: 0机构: UCL, Great Ormond St Inst Child Hlth, Infect Immun & Inflammat Res & Teaching Dept, London, England UCL, Great Ormond St Inst Child Hlth, Infect Immun & Inflammat Res & Teaching Dept, London, England
- [35] Targeted gene correction of human hematopoietic stem cells for the treatment of Wiskott - Aldrich SyndromeNATURE COMMUNICATIONS, 2020, 11 (01)Rai, Rajeev论文数: 0 引用数: 0 h-index: 0机构: UCL, Great Ormond St Inst Child Hlth, Infect Immun & Inflammat Res & Teaching Dept, 30 Guilford St, London WC1N 1EH, England UCL, Great Ormond St Inst Child Hlth, Infect Immun & Inflammat Res & Teaching Dept, 30 Guilford St, London WC1N 1EH, EnglandRomito, Marianna论文数: 0 引用数: 0 h-index: 0机构: UCL, Great Ormond St Inst Child Hlth, Infect Immun & Inflammat Res & Teaching Dept, 30 Guilford St, London WC1N 1EH, England UCL, Great Ormond St Inst Child Hlth, Infect Immun & Inflammat Res & Teaching Dept, 30 Guilford St, London WC1N 1EH, EnglandRivers, Elizabeth论文数: 0 引用数: 0 h-index: 0机构: UCL, Great Ormond St Inst Child Hlth, Infect Immun & Inflammat Res & Teaching Dept, 30 Guilford St, London WC1N 1EH, England UCL, Great Ormond St Inst Child Hlth, Infect Immun & Inflammat Res & Teaching Dept, 30 Guilford St, London WC1N 1EH, EnglandTurchiano, Giandomenico论文数: 0 引用数: 0 h-index: 0机构: UCL, Great Ormond St Inst Child Hlth, Infect Immun & Inflammat Res & Teaching Dept, 30 Guilford St, London WC1N 1EH, England UCL, Great Ormond St Inst Child Hlth, Infect Immun & Inflammat Res & Teaching Dept, 30 Guilford St, London WC1N 1EH, EnglandBlattner, Georges论文数: 0 引用数: 0 h-index: 0机构: UCL, Great Ormond St Inst Child Hlth, Infect Immun & Inflammat Res & Teaching Dept, 30 Guilford St, London WC1N 1EH, England UCL, Great Ormond St Inst Child Hlth, Infect Immun & Inflammat Res & Teaching Dept, 30 Guilford St, London WC1N 1EH, EnglandVetharoy, Winston论文数: 0 引用数: 0 h-index: 0机构: UCL, Great Ormond St Inst Child Hlth, Infect Immun & Inflammat Res & Teaching Dept, 30 Guilford St, London WC1N 1EH, England UCL, Great Ormond St Inst Child Hlth, Infect Immun & Inflammat Res & Teaching Dept, 30 Guilford St, London WC1N 1EH, EnglandLadon, Dariusz论文数: 0 引用数: 0 h-index: 0机构: Great Ormond St Hosp Children NHS Fdn Trust, SIHMDS Acquired Genom, Great Ormond St, London WC1N 3JH, England UCL, Great Ormond St Inst Child Hlth, Infect Immun & Inflammat Res & Teaching Dept, 30 Guilford St, London WC1N 1EH, EnglandAndrieux, Geoffroy论文数: 0 引用数: 0 h-index: 0机构: Univ Freiburg, Inst Med Bioinformat & Syst Med, 26 Stefan Meier Str, D-79104 Freiburg, Germany UCL, Great Ormond St Inst Child Hlth, Infect Immun & Inflammat Res & Teaching Dept, 30 Guilford St, London WC1N 1EH, EnglandZhang, Fang论文数: 0 引用数: 0 h-index: 0机构: UCL, Great Ormond St Inst Child Hlth, Infect Immun & Inflammat Res & Teaching Dept, 30 Guilford St, London WC1N 1EH, England UCL, Great Ormond St Inst Child Hlth, Infect Immun & Inflammat Res & Teaching Dept, 30 Guilford St, London WC1N 1EH, EnglandZinicola, Marta论文数: 0 引用数: 0 h-index: 0机构: UCL, Great Ormond St Inst Child Hlth, Infect Immun & Inflammat Res & Teaching Dept, 30 Guilford St, London WC1N 1EH, England UCL, Great Ormond St Inst Child Hlth, Infect Immun & Inflammat Res & Teaching Dept, 30 Guilford St, London WC1N 1EH, EnglandLeon-Rico, Diego论文数: 0 引用数: 0 h-index: 0机构: UCL, Great Ormond St Inst Child Hlth, Infect Immun & Inflammat Res & Teaching Dept, 30 Guilford St, London WC1N 1EH, England UCL, Great Ormond St Inst Child Hlth, Infect Immun & Inflammat Res & Teaching Dept, 30 Guilford St, London WC1N 1EH, EnglandSantilli, Giorgia论文数: 0 引用数: 0 h-index: 0机构: UCL, Great Ormond St Inst Child Hlth, Infect Immun & Inflammat Res & Teaching Dept, 30 Guilford St, London WC1N 1EH, England UCL, Great Ormond St Inst Child Hlth, Infect Immun & Inflammat Res & Teaching Dept, 30 Guilford St, London WC1N 1EH, EnglandThrasher, Adrian J.论文数: 0 引用数: 0 h-index: 0机构: UCL, Great Ormond St Inst Child Hlth, Infect Immun & Inflammat Res & Teaching Dept, 30 Guilford St, London WC1N 1EH, England UCL, Great Ormond St Inst Child Hlth, Infect Immun & Inflammat Res & Teaching Dept, 30 Guilford St, London WC1N 1EH, EnglandCavazza, Alessia论文数: 0 引用数: 0 h-index: 0机构: UCL, Great Ormond St Inst Child Hlth, Infect Immun & Inflammat Res & Teaching Dept, 30 Guilford St, London WC1N 1EH, England UCL, Great Ormond St Inst Child Hlth, Infect Immun & Inflammat Res & Teaching Dept, 30 Guilford St, London WC1N 1EH, England
- [36] Gene therapy in Wiskott-Aldrich syndrome: The next step?Current Allergy and Asthma Reports, 2005, 5 (5) : 347 - 348Julie Y. Patel论文数: 0 引用数: 0 h-index: 0David P. Huston论文数: 0 引用数: 0 h-index: 0
- [37] Efficacy and safety of gene therapy for Wiskott-Aldrich syndromeM S-MEDECINE SCIENCES, 2015, 31 (12): : 1066 - 1069Galy, Anne论文数: 0 引用数: 0 h-index: 0机构: Genethon, Unite Mixte Rech Integrare UMR S951, F-91000 Evry, France Inserm UMR S951, F-91000 Evry, France Univ Evry Val dEssonne, F-91000 Evry, France EPHE, F-91000 Evry, France Genethon, F-91000 Evry, France Genethon, Unite Mixte Rech Integrare UMR S951, F-91000 Evry, FranceCorre, Guillaume论文数: 0 引用数: 0 h-index: 0机构: Genethon, Unite Mixte Rech Integrare UMR S951, F-91000 Evry, France Inserm UMR S951, F-91000 Evry, France Univ Evry Val dEssonne, F-91000 Evry, France EPHE, F-91000 Evry, France Genethon, F-91000 Evry, France Genethon, Unite Mixte Rech Integrare UMR S951, F-91000 Evry, FranceCavazzana-, Marina论文数: 0 引用数: 0 h-index: 0机构: Hop Necker Enfants Malad, AP HP, Dept Biotherapies, Paris, France Grp Hosp Univ Ouest, AP HP, INSERM, CIC Biotherapies, Paris, France Paris Descartes Sorbonne Paris Cite Univ, Inst Imagine, Paris, France Genethon, Unite Mixte Rech Integrare UMR S951, F-91000 Evry, FranceHacein-Bey-Abina, Salima论文数: 0 引用数: 0 h-index: 0机构: Hop Necker Enfants Malad, AP HP, Dept Biotherapies, Paris, France Grp Hosp Univ Ouest, AP HP, INSERM, CIC Biotherapies, Paris, France Univ Paris 05, Fac Sci Pharmaceut & Biol, Inserm U1022, UTCBS CNRS 8258, Paris, France Grp Hosp Univ Paris Sud, Serv Immunol Biol, Le Kremlin Bicetre, France Genethon, Unite Mixte Rech Integrare UMR S951, F-91000 Evry, France
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- [40] ISOLATION OF A NOVEL GENE MUTATED IN WISKOTT-ALDRICH SYNDROMECELL, 1994, 78 (04) : 635 - 644DERRY, JMJ论文数: 0 引用数: 0 h-index: 0机构: STANFORD UNIV, MED CTR, DEPT GENET, STANFORD, CA 94305 USAOCHS, HD论文数: 0 引用数: 0 h-index: 0机构: STANFORD UNIV, MED CTR, DEPT GENET, STANFORD, CA 94305 USAFRANCKE, U论文数: 0 引用数: 0 h-index: 0机构: STANFORD UNIV, MED CTR, DEPT GENET, STANFORD, CA 94305 USA