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Lentiviral globin gene therapy with reduced-intensity conditioning in adults with ß-thalassemia: a phase 1 trial.
Boulad, Farid; Maggio, Aurelio; Wang, Xiuyan; Moi, Paolo; Acuto, Santina; Kogel, Friederike; Takpradit, Chayamon; Prockop, Susan; Mansilla-Soto, Jorge; Cabriolu, Annalisa; Odak, Ashlesha; Qu, Jinrong; Thummar, Keyur; Du, Fang; Shen, Lingbo; Raso, Simona; Barone, Rita; Di Maggio, Rosario; Pitrolo, Lorella; Giambona, Antonino; Mingoia, Maura; Everett, John K; Hokama, Pascha; Roche, Aoife M; Cantu, Vito Adrian; Adhikari, Hriju; Reddy, Shantan; Bouhassira, Eric; Mohandas, Narla; Bushman, Frederic D; Rivière, Isabelle; Sadelain, Michel.
Afiliación
  • Boulad F; Stem Cell Transplant and Cellular Therapy Service, Department of Pediatrics, Memorial Sloan Kettering Cancer Center, New York, NY, USA.
  • Maggio A; Center for Cell Engineering, Memorial Sloan Kettering Cancer Center, New York, NY, USA.
  • Wang X; Department of Hematology and Rare Diseases, Azienda Ospedaliera Ospedali Riuniti Villa Sofia-Cervello, Palermo, Italy.
  • Moi P; Center for Cell Engineering, Memorial Sloan Kettering Cancer Center, New York, NY, USA.
  • Acuto S; Cell Therapy and Cell Engineering Laboratory, Memorial Sloan Kettering Cancer Center, New York, NY, USA.
  • Kogel F; Ospedale Pediatrico Microcitemie 'A.Cao', A.O. 'G.Brotzu', Cagliari, Italy.
  • Takpradit C; Department of Hematology and Rare Diseases, Azienda Ospedaliera Ospedali Riuniti Villa Sofia-Cervello, Palermo, Italy.
  • Prockop S; Center for Cell Engineering, Memorial Sloan Kettering Cancer Center, New York, NY, USA.
  • Mansilla-Soto J; Stem Cell Transplant and Cellular Therapy Service, Department of Pediatrics, Memorial Sloan Kettering Cancer Center, New York, NY, USA.
  • Cabriolu A; Department of Pediatrics, Siriraj Hospital, Mahidol University, Bangkok, Thailand.
  • Odak A; Stem Cell Transplant and Cellular Therapy Service, Department of Pediatrics, Memorial Sloan Kettering Cancer Center, New York, NY, USA.
  • Qu J; Center for Cell Engineering, Memorial Sloan Kettering Cancer Center, New York, NY, USA.
  • Thummar K; Center for Cell Engineering, Memorial Sloan Kettering Cancer Center, New York, NY, USA.
  • Du F; Center for Cell Engineering, Memorial Sloan Kettering Cancer Center, New York, NY, USA.
  • Shen L; Cell Therapy and Cell Engineering Laboratory, Memorial Sloan Kettering Cancer Center, New York, NY, USA.
  • Raso S; Cell Therapy and Cell Engineering Laboratory, Memorial Sloan Kettering Cancer Center, New York, NY, USA.
  • Barone R; Cell Therapy and Cell Engineering Laboratory, Memorial Sloan Kettering Cancer Center, New York, NY, USA.
  • Di Maggio R; Cell Therapy and Cell Engineering Laboratory, Memorial Sloan Kettering Cancer Center, New York, NY, USA.
  • Pitrolo L; Department of Hematology and Rare Diseases, Azienda Ospedaliera Ospedali Riuniti Villa Sofia-Cervello, Palermo, Italy.
  • Giambona A; Department of Hematology and Rare Diseases, Azienda Ospedaliera Ospedali Riuniti Villa Sofia-Cervello, Palermo, Italy.
  • Mingoia M; Department of Hematology and Rare Diseases, Azienda Ospedaliera Ospedali Riuniti Villa Sofia-Cervello, Palermo, Italy.
  • Everett JK; Department of Hematology and Rare Diseases, Azienda Ospedaliera Ospedali Riuniti Villa Sofia-Cervello, Palermo, Italy.
  • Hokama P; Department of Hematology and Rare Diseases, Azienda Ospedaliera Ospedali Riuniti Villa Sofia-Cervello, Palermo, Italy.
  • Roche AM; Ospedale Pediatrico Microcitemie 'A.Cao', A.O. 'G.Brotzu', Cagliari, Italy.
  • Cantu VA; Department of Microbiology, University of Pennsylvania Perelman School of Medicine, Philadelphia, PA, USA.
  • Adhikari H; Department of Microbiology, University of Pennsylvania Perelman School of Medicine, Philadelphia, PA, USA.
  • Reddy S; Department of Microbiology, University of Pennsylvania Perelman School of Medicine, Philadelphia, PA, USA.
  • Bouhassira E; Department of Microbiology, University of Pennsylvania Perelman School of Medicine, Philadelphia, PA, USA.
  • Mohandas N; Department of Microbiology, University of Pennsylvania Perelman School of Medicine, Philadelphia, PA, USA.
  • Bushman FD; Department of Microbiology, University of Pennsylvania Perelman School of Medicine, Philadelphia, PA, USA.
  • Rivière I; Department of Cell Biology, Albert Einstein College of Medicine, Bronx, New York, NY, USA.
  • Sadelain M; Laboratory of Red Cell Physiology, New York Blood Center, New York, NY, USA.
Nat Med ; 28(1): 63-70, 2022 01.
Article en En | MEDLINE | ID: mdl-34980909
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Texto completo: 1 Banco de datos: MEDLINE Asunto principal: Globinas / Terapia Genética / Talasemia beta / Lentivirus / Acondicionamiento Pretrasplante / Vectores Genéticos Límite: Adolescent / Adult / Female / Humans / Male Idioma: En Revista: Nat Med Asunto de la revista: BIOLOGIA MOLECULAR / MEDICINA Año: 2022 Tipo del documento: Article País de afiliación: Estados Unidos
Texto completo
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XML
PubMed Links
Buscar en Google

Texto completo: 1 Banco de datos: MEDLINE Asunto principal: Globinas / Terapia Genética / Talasemia beta / Lentivirus / Acondicionamiento Pretrasplante / Vectores Genéticos Límite: Adolescent / Adult / Female / Humans / Male Idioma: En Revista: Nat Med Asunto de la revista: BIOLOGIA MOLECULAR / MEDICINA Año: 2022 Tipo del documento: Article País de afiliación: Estados Unidos