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- [31] Initial Results from the BEACON Clinical Study: A Phase 1/2 Study Evaluating the Safety and Efficacy of a Single Dose of Autologous CD34+Base Edited Hematopoietic Stem Cells (BEAM-101) in Patients with Sickle Cell Disease with Severe Vaso-Occlusive Crises[J]. BLOOD, 2024, 144 : 514 - 515Gupta, Ashish O.论文数: 0 引用数: 0 h-index: 0机构: Univ Minnesota, Div Pediat Blood & Marrow Transplant & Cellular Th, Minneapolis, MN USA Univ Minnesota, Div Pediat Blood & Marrow Transplant & Cellular Th, Minneapolis, MN USASharma, Akshay论文数: 0 引用数: 0 h-index: 0机构: St Jude Childrens Res Hosp, Dept Bone Marrow Transplantat & Cellular Therapy, Memphis, TN USA Univ Minnesota, Div Pediat Blood & Marrow Transplant & Cellular Th, Minneapolis, MN USAFrangoul, Haydar论文数: 0 引用数: 0 h-index: 0机构: TriStar Centennial, Sarah Cannon Res Inst, Childrens Hosp, Nashville, TN USA Univ Minnesota, Div Pediat Blood & Marrow Transplant & Cellular Th, Minneapolis, MN USADalal, Jignesh论文数: 0 引用数: 0 h-index: 0机构: Rainbow Babies & Childrens Hosp, Div Hematol & Bone Marrow Transplant, Cleveland, OH USA Univ Minnesota, Div Pediat Blood & Marrow Transplant & Cellular Th, Minneapolis, MN USA论文数: 引用数: h-index:机构:Alavi, Asif论文数: 0 引用数: 0 h-index: 0机构: Henry Ford Hlth Syst, Detroit, MI USA Univ Minnesota, Div Pediat Blood & Marrow Transplant & Cellular Th, Minneapolis, MN USADipersio, John论文数: 0 引用数: 0 h-index: 0机构: Washington Univ, Div Oncol, St Louis, MO USA Univ Minnesota, Div Pediat Blood & Marrow Transplant & Cellular Th, Minneapolis, MN USAEapen, Mary论文数: 0 引用数: 0 h-index: 0机构: Med Coll Wisconsin Inc, DEPT MED, MILWAUKEE, WI USA Univ Minnesota, Div Pediat Blood & Marrow Transplant & Cellular Th, Minneapolis, MN USAJaroscak, Jennifer J.论文数: 0 引用数: 0 h-index: 0机构: Med Univ South Carolina, Charleston, SC USA Univ Minnesota, Div Pediat Blood & Marrow Transplant & Cellular Th, Minneapolis, MN USAAyala, Ernesto论文数: 0 引用数: 0 h-index: 0机构: Mayo Clin, Dept Hematol Oncol, Jacksonville, FL USA Univ Minnesota, Div Pediat Blood & Marrow Transplant & Cellular Th, Minneapolis, MN USAZiga, Edward D.论文数: 0 引用数: 0 h-index: 0机构: Miller Sch Med, Miami, FL USA Univ Minnesota, Div Pediat Blood & Marrow Transplant & Cellular Th, Minneapolis, MN USARifkin-Zenenberg, Stacey论文数: 0 引用数: 0 h-index: 0机构: Hackensack Univ, Pediat Hematol Oncol Div, Med Ctr, Hackensack, NJ USA Univ Minnesota, Div Pediat Blood & Marrow Transplant & Cellular Th, Minneapolis, MN USAMinella, Alex C.论文数: 0 引用数: 0 h-index: 0机构: Beam Therapeut Inc, Cambridge, MA USA Univ Minnesota, Div Pediat Blood & Marrow Transplant & Cellular Th, Minneapolis, MN USAChen, Guo论文数: 0 引用数: 0 h-index: 0机构: Beam Therapeut Inc, Cambridge, MA USA Univ Minnesota, Div Pediat Blood & Marrow Transplant & Cellular Th, Minneapolis, MN USAChen, Yinzhong论文数: 0 引用数: 0 h-index: 0机构: Beam Therapeut Inc, Cambridge, MA USA Univ Minnesota, Div Pediat Blood & Marrow Transplant & Cellular Th, Minneapolis, MN USAChockalingam, Priya S.论文数: 0 引用数: 0 h-index: 0机构: Beam Therapeut Inc, Cambridge, MA USA Univ Minnesota, Div Pediat Blood & Marrow Transplant & Cellular Th, Minneapolis, MN USALin, Ling论文数: 0 引用数: 0 h-index: 0机构: Beam Therapeut Inc, Cambridge, MA USA Univ Minnesota, Div Pediat Blood & Marrow Transplant & Cellular Th, Minneapolis, MN USAJoseney-Antoine, Marcelyne论文数: 0 引用数: 0 h-index: 0机构: Beam Therapeut Inc, Cambridge, MA USA Univ Minnesota, Div Pediat Blood & Marrow Transplant & Cellular Th, Minneapolis, MN USAIanniello, Leanne论文数: 0 引用数: 0 h-index: 0机构: Beam Therapeut Inc, Cambridge, MA USA Univ Minnesota, Div Pediat Blood & Marrow Transplant & Cellular Th, Minneapolis, MN USAGardner, Beth论文数: 0 引用数: 0 h-index: 0机构: Beam Therapeut Inc, Cambridge, MA USA Univ Minnesota, Div Pediat Blood & Marrow Transplant & Cellular Th, Minneapolis, MN USAHartigan, Adam J.论文数: 0 引用数: 0 h-index: 0机构: Beam Therapeut Inc, Cambridge, MA USA Univ Minnesota, Div Pediat Blood & Marrow Transplant & Cellular Th, Minneapolis, MN USACiaramella, Giuseppe论文数: 0 引用数: 0 h-index: 0机构: Beam Therapeut Inc, Cambridge, MA USA Univ Minnesota, Div Pediat Blood & Marrow Transplant & Cellular Th, Minneapolis, MN USAGoyal, Sunita论文数: 0 引用数: 0 h-index: 0机构: Beam Therapeut Inc, Cambridge, MA USA Univ Minnesota, Div Pediat Blood & Marrow Transplant & Cellular Th, Minneapolis, MN USASimon, Amy论文数: 0 引用数: 0 h-index: 0机构: Beam Therapeut Inc, Cambridge, MA USA Univ Minnesota, Div Pediat Blood & Marrow Transplant & Cellular Th, Minneapolis, MN USAThompson, Alexis论文数: 0 引用数: 0 h-index: 0机构: Childrens Hosp Philadelphia, Div Hematol, Philadelphia, PA USA Univ Minnesota, Div Pediat Blood & Marrow Transplant & Cellular Th, Minneapolis, MN USAHeeney, Matthew M.论文数: 0 引用数: 0 h-index: 0机构: Dana Farber Boston Childrens Canc, Boston, MA USA Blood Disorders Ctr, Boston, MA USA Univ Minnesota, Div Pediat Blood & Marrow Transplant & Cellular Th, Minneapolis, MN USA
- [32] GMI 1070: Reduction In Time To Resolution Of Vaso-Occlusive Crisis and Decreased Opioid Use In a Prospective, Randomized, Multi-Center Double Blind, Adaptive Phase 2 Study In Sickle Cell Disease[J]. BLOOD, 2013, 122 (21)Telen, Marilyn J.论文数: 0 引用数: 0 h-index: 0Wun, Ted论文数: 0 引用数: 0 h-index: 0McCavit, Timothy L.论文数: 0 引用数: 0 h-index: 0De Castro, Laura M.论文数: 0 引用数: 0 h-index: 0Krishnamurti, Lakshmanan论文数: 0 引用数: 0 h-index: 0Lanzkron, Sophie论文数: 0 引用数: 0 h-index: 0Hsu, Lewis L.论文数: 0 引用数: 0 h-index: 0Smith, Wally R.论文数: 0 引用数: 0 h-index: 0Rhee, Seungshin论文数: 0 引用数: 0 h-index: 0Magnani, John L.论文数: 0 引用数: 0 h-index: 0Thackray, Helen论文数: 0 引用数: 0 h-index: 0
- [33] Preliminary Safety and Efficacy Results from Precizn-1: An Ongoing Phase 1/2 Study on Zinc Finger Nuclease-Modified Autologous CD34+HSPCs for Sickle Cell Disease (SCD)[J]. BLOOD, 2021, 138Alavi, Asif论文数: 0 引用数: 0 h-index: 0机构: Wayne State Univ, Karmanos Canc Inst, Detroit, MI USA Wayne State Univ, Karmanos Canc Inst, Detroit, MI USAKrishnamurti, Lakshmanan论文数: 0 引用数: 0 h-index: 0机构: Emory Healthcare, Dept Pediat, Aflac Canc & Blood Disorders Ctr, Atlanta, GA USA Wayne State Univ, Karmanos Canc Inst, Detroit, MI USAAbedi, Mehrdad论文数: 0 引用数: 0 h-index: 0机构: Univ Davis, Med Ctr, Sacramento, CA USA Wayne State Univ, Karmanos Canc Inst, Detroit, MI USAGaleon, Isobelle论文数: 0 引用数: 0 h-index: 0机构: Sanofi, Cambridge, MA USA Wayne State Univ, Karmanos Canc Inst, Detroit, MI USAReiner, David论文数: 0 引用数: 0 h-index: 0机构: Sanofi, Bridgewater, NJ USA Wayne State Univ, Karmanos Canc Inst, Detroit, MI USASmith, Sharon E.论文数: 0 引用数: 0 h-index: 0机构: Sanofi, Cambridge, MA USA Wayne State Univ, Karmanos Canc Inst, Detroit, MI USAWang, Lin论文数: 0 引用数: 0 h-index: 0机构: Sanofi, Cambridge, MA USA Wayne State Univ, Karmanos Canc Inst, Detroit, MI USARamezi, Anne论文数: 0 引用数: 0 h-index: 0机构: Sanofi, Cambridge, MA USA Wayne State Univ, Karmanos Canc Inst, Detroit, MI USARendo, Pablo论文数: 0 引用数: 0 h-index: 0机构: Sanofi, Cambridge, MA USA Wayne State Univ, Karmanos Canc Inst, Detroit, MI USAWalters, Mark C.论文数: 0 引用数: 0 h-index: 0机构: UCSF, Div Hematol, Benioff Childrens Hosp Oakland, Oakland, CA USA Wayne State Univ, Karmanos Canc Inst, Detroit, MI USA
- [34] Early Results from a Phase 1/2 Study of ARU-1801 Gene Therapy for Sickle Cell Disease (SCD): Safety and Efficacy of a Modified Gamma Globin Lentivirus Vector and Reduced Intensity Conditioning Transplant[J]. MOLECULAR THERAPY, 2021, 29 (04) : 39 - 40Grimley, Michael论文数: 0 引用数: 0 h-index: 0机构: Cincinnati Childrens Hosp Med Ctr, Cincinnati, OH 45229 USA Cincinnati Childrens Hosp Med Ctr, Cincinnati, OH 45229 USAAsnani, Monika论文数: 0 引用数: 0 h-index: 0机构: Carribean Inst Hlth Res, Kingston, Jamaica Cincinnati Childrens Hosp Med Ctr, Cincinnati, OH 45229 USAShrestha, Archana论文数: 0 引用数: 0 h-index: 0机构: Cincinnati Childrens Hosp Med Ctr, Cincinnati, OH 45229 USA Cincinnati Childrens Hosp Med Ctr, Cincinnati, OH 45229 USAFelker, Sydney论文数: 0 引用数: 0 h-index: 0机构: Cincinnati Childrens Hosp Med Ctr, Cincinnati, OH 45229 USA Cincinnati Childrens Hosp Med Ctr, Cincinnati, OH 45229 USALutzko, Carolyn论文数: 0 引用数: 0 h-index: 0机构: Cincinnati Childrens Hosp Med Ctr, Cincinnati, OH 45229 USA Cincinnati Childrens Hosp Med Ctr, Cincinnati, OH 45229 USAArumugam, Paritha I.论文数: 0 引用数: 0 h-index: 0机构: Cincinnati Childrens Hosp Med Ctr, Cincinnati, OH 45229 USA Cincinnati Childrens Hosp Med Ctr, Cincinnati, OH 45229 USAWitting, Scott论文数: 0 引用数: 0 h-index: 0机构: Cincinnati Childrens Hosp Med Ctr, Cincinnati, OH 45229 USA Cincinnati Childrens Hosp Med Ctr, Cincinnati, OH 45229 USAKnight-Madden, Jennifer论文数: 0 引用数: 0 h-index: 0机构: Carribean Inst Hlth Res, Kingston, Jamaica Cincinnati Childrens Hosp Med Ctr, Cincinnati, OH 45229 USANiss, Omar论文数: 0 引用数: 0 h-index: 0机构: Cincinnati Childrens Hosp Med Ctr, Cincinnati, OH 45229 USA Cincinnati Childrens Hosp Med Ctr, Cincinnati, OH 45229 USAQuinn, Charles T.论文数: 0 引用数: 0 h-index: 0机构: Cincinnati Childrens Hosp Med Ctr, Cincinnati, OH 45229 USA Cincinnati Childrens Hosp Med Ctr, Cincinnati, OH 45229 USALo, Christopher论文数: 0 引用数: 0 h-index: 0机构: Aruvant Sci, New York, NY USA Cincinnati Childrens Hosp Med Ctr, Cincinnati, OH 45229 USALittle, Courtney R.论文数: 0 引用数: 0 h-index: 0机构: Aruvant Sci, New York, NY USA Cincinnati Childrens Hosp Med Ctr, Cincinnati, OH 45229 USAMcIntosh, Joseph W.论文数: 0 引用数: 0 h-index: 0机构: Aruvant Sci, New York, NY USA Cincinnati Childrens Hosp Med Ctr, Cincinnati, OH 45229 USAMalik, Punam论文数: 0 引用数: 0 h-index: 0机构: Cincinnati Childrens Hosp Med Ctr, Cincinnati, OH 45229 USA Cincinnati Childrens Hosp Med Ctr, Cincinnati, OH 45229 USA
- [35] Trials in Progress: The Randomized, Double-Blind, Placebo-Controlled Phase 1b CROSSWALK-a and Phase 2a CROSSWALK-c Trials - Crovalimab for the Treatment and Prevention of Vaso-Occlusive Episodes in Sickle Cell Disease[J]. BLOOD, 2023, 142Salvino, Marco Aurelio论文数: 0 引用数: 0 h-index: 0机构: Univ Fed Bahia, Hosp Sao Rafael, IDOR, PPGMS, Salvador, BA, Brazil Univ Fed Bahia, Hosp Sao Rafael, IDOR, PPGMS, Salvador, BA, BrazilAntmen, Bulent论文数: 0 引用数: 0 h-index: 0机构: Adana Acibadem Hosp, Dept Pediat Hematol, Adana, Turkiye Univ Fed Bahia, Hosp Sao Rafael, IDOR, PPGMS, Salvador, BA, BrazilAsirwa, Frederick Chite论文数: 0 引用数: 0 h-index: 0机构: Int Canc Inst ICI, Eldoret, Kenya Univ Fed Bahia, Hosp Sao Rafael, IDOR, PPGMS, Salvador, BA, Brazil论文数: 引用数: h-index:机构:De Franceschi, Lucia论文数: 0 引用数: 0 h-index: 0机构: Univ Verona, Dept Med, Verona, Italy AOUI Verona, Policlin GB Rossi, Verona, Italy Univ Fed Bahia, Hosp Sao Rafael, IDOR, PPGMS, Salvador, BA, BrazilEleftheriou, Perla论文数: 0 引用数: 0 h-index: 0机构: Univ Coll London Hosp, London, England Univ Fed Bahia, Hosp Sao Rafael, IDOR, PPGMS, Salvador, BA, BrazilInati, Adlette论文数: 0 引用数: 0 h-index: 0机构: Lebanese Amer Univ, Gilbert & Rose Marie Chagoury Sch Med, Byblos, Lebanon NINI Hosp, Tripoli, Lebanon Univ Fed Bahia, Hosp Sao Rafael, IDOR, PPGMS, Salvador, BA, Brazil论文数: 引用数: h-index:机构:Nur, Erfan论文数: 0 引用数: 0 h-index: 0机构: Amsterdam UMC, Locat AMC, Dept Hematol, Amsterdam, Netherlands Univ Fed Bahia, Hosp Sao Rafael, IDOR, PPGMS, Salvador, BA, BrazilPayan-Pernia, Salvador论文数: 0 引用数: 0 h-index: 0机构: Hosp Univ Virgen del RocIo, Inst Biomed Sevilla IBiS, Serv Hemat, Seville, Spain Univ Fed Bahia, Hosp Sao Rafael, IDOR, PPGMS, Salvador, BA, BrazilCharania, Asad论文数: 0 引用数: 0 h-index: 0机构: F Hoffmann Roche Ltd, London, England Univ Fed Bahia, Hosp Sao Rafael, IDOR, PPGMS, Salvador, BA, BrazilGradinaru, Diana论文数: 0 引用数: 0 h-index: 0机构: F Hoffmann Roche Ltd, Basel, Switzerland Univ Fed Bahia, Hosp Sao Rafael, IDOR, PPGMS, Salvador, BA, BrazilHsia, Allison论文数: 0 引用数: 0 h-index: 0机构: Genentech Inc, San Francisco, CA USA Univ Fed Bahia, Hosp Sao Rafael, IDOR, PPGMS, Salvador, BA, BrazilLuder, Yves论文数: 0 引用数: 0 h-index: 0机构: F Hoffmann Roche Ltd, Basel, Switzerland Univ Fed Bahia, Hosp Sao Rafael, IDOR, PPGMS, Salvador, BA, BrazilPerretti, Thomas论文数: 0 引用数: 0 h-index: 0机构: F Hoffmann Roche Ltd, Basel, Switzerland Univ Fed Bahia, Hosp Sao Rafael, IDOR, PPGMS, Salvador, BA, BrazilSrekovic, Sasha论文数: 0 引用数: 0 h-index: 0机构: Genentech Inc, San Francisco, CA USA Univ Fed Bahia, Hosp Sao Rafael, IDOR, PPGMS, Salvador, BA, BrazilBartolucci, Pablo论文数: 0 引用数: 0 h-index: 0机构: CHU Henri Mondor, Serv Med Interne, Creteil, France Univ Fed Bahia, Hosp Sao Rafael, IDOR, PPGMS, Salvador, BA, Brazil
- [36] Isaralgagene civaparvovec (ST-920) gene therapy in adults with Fabry disease: Updated results from an ongoing phase 1/2 study (STAAR)[J]. MOLECULAR GENETICS AND METABOLISM, 2024, 141 (02)Hopkin, Robert J.论文数: 0 引用数: 0 h-index: 0机构: Cincinnati Childrens Hosp Med Ctr, Cincinnati, OH 45229 USA Cincinnati Childrens Hosp Med Ctr, Cincinnati, OH 45229 USAGanesh, Jaya论文数: 0 引用数: 0 h-index: 0机构: Icahn Sch Med Mt Sinai, New York, NY 10029 USA Cincinnati Childrens Hosp Med Ctr, Cincinnati, OH 45229 USABernat, John论文数: 0 引用数: 0 h-index: 0机构: Univ Iowa Hosp & Clin, Iowa City, IA 52242 USA Cincinnati Childrens Hosp Med Ctr, Cincinnati, OH 45229 USAGoker-Alpan, Ozlem论文数: 0 引用数: 0 h-index: 0机构: Lysosomal & Rare Disorders Res & Treatment Ctr In, Fairfax, VA USA Cincinnati Childrens Hosp Med Ctr, Cincinnati, OH 45229 USANicholls, Kathleen论文数: 0 引用数: 0 h-index: 0机构: Royal Melbourne Hosp, Parkville, Vic, Australia Cincinnati Childrens Hosp Med Ctr, Cincinnati, OH 45229 USAPahl, Madeleine V.论文数: 0 引用数: 0 h-index: 0机构: Univ Calif Irvine, Orange, CA 92668 USA Cincinnati Childrens Hosp Med Ctr, Cincinnati, OH 45229 USADeegan, Patrick B.论文数: 0 引用数: 0 h-index: 0机构: Addenbrookes Hosp, Cambridge, England Cincinnati Childrens Hosp Med Ctr, Cincinnati, OH 45229 USAWhitley, Chester B.论文数: 0 引用数: 0 h-index: 0机构: Univ Minnesota, Minneapolis, MN USA Cincinnati Childrens Hosp Med Ctr, Cincinnati, OH 45229 USAHughes, Derralynn论文数: 0 引用数: 0 h-index: 0机构: Royal Free London Hosp, London, England Cincinnati Childrens Hosp Med Ctr, Cincinnati, OH 45229 USACao, Liching论文数: 0 引用数: 0 h-index: 0机构: Sangamo Therapeut Inc, Brisbane, CA USA Cincinnati Childrens Hosp Med Ctr, Cincinnati, OH 45229 USAChen, Michael论文数: 0 引用数: 0 h-index: 0机构: Sangamo Therapeut Inc, Brisbane, CA USA Cincinnati Childrens Hosp Med Ctr, Cincinnati, OH 45229 USAShiue, Lisa论文数: 0 引用数: 0 h-index: 0机构: Sangamo Therapeut Inc, Brisbane, CA USA Cincinnati Childrens Hosp Med Ctr, Cincinnati, OH 45229 USARojkjaer, Lisa论文数: 0 引用数: 0 h-index: 0机构: Sangamo Therapeut Inc, Brisbane, CA USA Cincinnati Childrens Hosp Med Ctr, Cincinnati, OH 45229 USAWilcox, William R.论文数: 0 引用数: 0 h-index: 0机构: Emory Univ, Sch Med, Atlanta, GA USA Cincinnati Childrens Hosp Med Ctr, Cincinnati, OH 45229 USA
- [37] Isaralgagene civaparvovec (ST-920) gene therapy in adults with Fabry disease: Updated results from an ongoing phase 1/2 study (STAAR)[J]. MOLECULAR GENETICS AND METABOLISM, 2025, 144 (02)Hughes, Derralynn论文数: 0 引用数: 0 h-index: 0机构: Royal Free London Hosp, London, England Royal Free London Hosp, London, EnglandWilcox, William论文数: 0 引用数: 0 h-index: 0机构: Emory Univ, Sch Med, Atlanta, GA USA Royal Free London Hosp, London, EnglandHopkin, Robert J.论文数: 0 引用数: 0 h-index: 0机构: Cincinnati Childrens Hosp Med Ctr, Cincinnati, OH USA Royal Free London Hosp, London, EnglandGanesh, Jaya论文数: 0 引用数: 0 h-index: 0机构: Icahn Sch Med Mt Sinai, New York, NY USA Royal Free London Hosp, London, EnglandBernat, John论文数: 0 引用数: 0 h-index: 0机构: Univ Iowa, Iowa City, IA USA Royal Free London Hosp, London, EnglandGoker-Alpan, Ozlem论文数: 0 引用数: 0 h-index: 0机构: Lysosomal & Rare Disorders Res & Treatment Ctr, Fairfax, VA USA Royal Free London Hosp, London, EnglandNicholls, Kathy论文数: 0 引用数: 0 h-index: 0机构: Addenbrookes Hosp, Cambridge, England Royal Free London Hosp, London, EnglandDeegan, Patrick论文数: 0 引用数: 0 h-index: 0机构: Univ Calif Irvine, Irvine, CA USA Royal Free London Hosp, London, EnglandPahl, Madeleine论文数: 0 引用数: 0 h-index: 0机构: Univ Minnesota, Minneapolis, MN USA Royal Free London Hosp, London, EnglandWhitley, Chester B.论文数: 0 引用数: 0 h-index: 0机构: Sangamo Therapeut Inc, Richmond, CA USA Royal Free London Hosp, London, EnglandChen, Michael论文数: 0 引用数: 0 h-index: 0机构: Royal Free London Hosp, London, EnglandCao, Liching论文数: 0 引用数: 0 h-index: 0机构: Royal Free London Hosp, London, EnglandSchreeb, Katharina H.论文数: 0 引用数: 0 h-index: 0机构: Royal Free London Hosp, London, England
- [38] Early Results from a Phase 1/2 Study of Aru-1801 Gene Therapy for Sickle Cell Disease (SCD): Manufacturing Process Enhancements Improve Efficacy of a Modified Gamma Globin Lentivirus Vector and Reduced Intensity Conditioning Transplant[J]. BLOOD, 2020, 136Grimley, Michael论文数: 0 引用数: 0 h-index: 0Asnani, Monika论文数: 0 引用数: 0 h-index: 0Shrestha, Archana论文数: 0 引用数: 0 h-index: 0Felker, Sydney论文数: 0 引用数: 0 h-index: 0Lutzko, Carolyn论文数: 0 引用数: 0 h-index: 0Arumugam, Paritha I.论文数: 0 引用数: 0 h-index: 0Witting, Scott论文数: 0 引用数: 0 h-index: 0Knight-Madden, Jennifer论文数: 0 引用数: 0 h-index: 0Niss, Omar论文数: 0 引用数: 0 h-index: 0Quinn, Charles T.论文数: 0 引用数: 0 h-index: 0Lo, Chris论文数: 0 引用数: 0 h-index: 0Little, Courtney R.论文数: 0 引用数: 0 h-index: 0McIntosh, Joseph W.论文数: 0 引用数: 0 h-index: 0Malik, Punam论文数: 0 引用数: 0 h-index: 0
- [39] Safety and Efficacy of Aru-1801 in Patients with Sickle Cell Disease: Early Results from the Phase 1/2 Momentum Study of a Modified Gamma Globin Gene Therapy and Reduced Intensity Conditioning[J]. BLOOD, 2021, 138Grimley, Michael论文数: 0 引用数: 0 h-index: 0机构: Cincinnati Childrens Hosp, Div Bone Marrow Transplantat & Immune Deficiency, Med Ctr, Cincinnati, OH USA Univ Cincinnati, Coll Med, Dept Pediat, Cincinnati, OH USA Cincinnati Childrens Hosp, Div Bone Marrow Transplantat & Immune Deficiency, Med Ctr, Cincinnati, OH USA论文数: 引用数: h-index:机构:Shrestha, Archana论文数: 0 引用数: 0 h-index: 0机构: Cincinnati Childrens Hosp & Med Ctr, Div Expt Hematol & Canc Biol, Cincinnati, OH USA Cincinnati Childrens Hosp, Div Bone Marrow Transplantat & Immune Deficiency, Med Ctr, Cincinnati, OH USAFelker, Sydney论文数: 0 引用数: 0 h-index: 0机构: Cincinnati Childrens Hosp Med Ctr, Div Expt Hematol & Canc Biol, Cincinnati, OH USA Univ Cincinnati, Coll Med, Dept Pediat, Cincinnati, OH USA Cincinnati Childrens Hosp, Div Bone Marrow Transplantat & Immune Deficiency, Med Ctr, Cincinnati, OH USALutzko, Carolyn论文数: 0 引用数: 0 h-index: 0机构: Cincinnati Childrens Hosp Med Ctr, Div Expt Hematol & Canc Biol, Cincinnati, OH USA Univ Cincinnati, Coll Med, Dept Pediat, Cincinnati, OH USA Cincinnati Childrens Hosp, Div Bone Marrow Transplantat & Immune Deficiency, Med Ctr, Cincinnati, OH USAArumugam, Paritha I.论文数: 0 引用数: 0 h-index: 0机构: Cincinnati Childrens Hosp Med Ctr, Div Expt Hematol & Canc Biol, Cincinnati, OH USA Univ Cincinnati, Coll Med, Dept Pediat, Cincinnati, OH USA Cincinnati Childrens Hosp, Div Bone Marrow Transplantat & Immune Deficiency, Med Ctr, Cincinnati, OH USAWitting, Scott论文数: 0 引用数: 0 h-index: 0机构: Cincinnati Childrens Hosp Med Ctr, Div Expt Hematol & Canc Biol, Cincinnati, OH USA Univ Cincinnati, Coll Med, Dept Pediat, Cincinnati, OH USA Cincinnati Childrens Hosp, Div Bone Marrow Transplantat & Immune Deficiency, Med Ctr, Cincinnati, OH USA论文数: 引用数: h-index:机构:Niss, Omar论文数: 0 引用数: 0 h-index: 0机构: Univ Cincinnati, Coll Med, Dept Pediat, Cincinnati, OH USA Cincinnati Childrens Hosp, Med Ctr, Div Hematol, Cincinnati, OH USA Cincinnati Childrens Hosp, Div Bone Marrow Transplantat & Immune Deficiency, Med Ctr, Cincinnati, OH USAQuinn, Charles T.论文数: 0 引用数: 0 h-index: 0机构: Univ Cincinnati, Coll Med, Dept Pediat, Cincinnati, OH USA Cincinnati Childrens Hosp, Med Ctr, Div Hematol, Cincinnati, OH USA Cincinnati Childrens Hosp, Div Bone Marrow Transplantat & Immune Deficiency, Med Ctr, Cincinnati, OH USALo, Chris论文数: 0 引用数: 0 h-index: 0机构: Aruvant Sci, New York, NY USA Cincinnati Childrens Hosp, Div Bone Marrow Transplantat & Immune Deficiency, Med Ctr, Cincinnati, OH USALittle, Courtney R.论文数: 0 引用数: 0 h-index: 0机构: Cincinnati Childrens Hosp, Div Bone Marrow Transplantat & Immune Deficiency, Med Ctr, Cincinnati, OH USADong, Alisa论文数: 0 引用数: 0 h-index: 0机构: Aruvant Sci, New York, NY USA Cincinnati Childrens Hosp, Div Bone Marrow Transplantat & Immune Deficiency, Med Ctr, Cincinnati, OH USAMalik, Punam论文数: 0 引用数: 0 h-index: 0机构: Cincinnati Childrens Hosp Med Ctr, Div Expt Hematol & Canc Biol, Cincinnati, OH USA Univ Cincinnati, Coll Med, Dept Pediat, Cincinnati, OH USA Cincinnati Childrens Hosp, Div Bone Marrow Transplantat & Immune Deficiency, Med Ctr, Cincinnati, OH USA
- [40] Initial Results from the Northstar Study (HGB-204): A Phase 1/2 Study of Gene Therapy for β-Thalassemia Major Via Transplantation of Autologous Hematopoietic Stem Cells Transduced Ex Vivo with a Lentiviral βA-T87Q Globin Vector (LentiGlobin BB305 Drug Product)[J]. BLOOD, 2014, 124 (21)Thompson, Alexis A.论文数: 0 引用数: 0 h-index: 0机构: Northwestern Univ, Ann & Robert H Lurie Childrens Hosp Chicago, Chicago, IL 60611 USA Northwestern Univ, Ann & Robert H Lurie Childrens Hosp Chicago, Chicago, IL 60611 USARasko, John E.论文数: 0 引用数: 0 h-index: 0机构: Royal Prince Alfred Hosp, Sydney, NSW, Australia Northwestern Univ, Ann & Robert H Lurie Childrens Hosp Chicago, Chicago, IL 60611 USA论文数: 引用数: h-index:机构:Kwiatkowski, Janet L.论文数: 0 引用数: 0 h-index: 0机构: Childrens Hosp Philadelphia, Philadelphia, PA 19104 USA Univ Penn, Perelman Sch Med, Philadelphia, PA 19104 USA Northwestern Univ, Ann & Robert H Lurie Childrens Hosp Chicago, Chicago, IL 60611 USASchiller, Gary论文数: 0 引用数: 0 h-index: 0机构: Univ Calif Los Angeles, David Geffen Sch Med, Los Angeles, CA 90095 USA Northwestern Univ, Ann & Robert H Lurie Childrens Hosp Chicago, Chicago, IL 60611 USAvon Kalle, Christof论文数: 0 引用数: 0 h-index: 0机构: Natl Ctr Tumor Dis NCT, Heidelberg, Germany German Canc Res Ctr, Heidelberg, Germany Northwestern Univ, Ann & Robert H Lurie Childrens Hosp Chicago, Chicago, IL 60611 USACavazzana, Marina论文数: 0 引用数: 0 h-index: 0机构: Hop Univ Necker Enfants Malades, Paris, France Northwestern Univ, Ann & Robert H Lurie Childrens Hosp Chicago, Chicago, IL 60611 USA论文数: 引用数: h-index:机构:Petrusich, Alexandria论文数: 0 引用数: 0 h-index: 0机构: Bluebird Bio Inc, Cambridge, MA USA Northwestern Univ, Ann & Robert H Lurie Childrens Hosp Chicago, Chicago, IL 60611 USASoni, Sandeep论文数: 0 引用数: 0 h-index: 0机构: Bluebird Bio Inc, Cambridge, MA USA Northwestern Univ, Ann & Robert H Lurie Childrens Hosp Chicago, Chicago, IL 60611 USAWalters, Mark C.论文数: 0 引用数: 0 h-index: 0机构: UCSF Benioff Childrens Hosp, Oakland, CA USA Northwestern Univ, Ann & Robert H Lurie Childrens Hosp Chicago, Chicago, IL 60611 USA