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Sustained fetal hemoglobin induction in vivo is achieved by BCL11A interference and coexpressed truncated erythropoietin receptor.
Uchida, Naoya; Ferrara, Francesca; Drysdale, Claire M; Yapundich, Morgan; Gamer, Jackson; Nassehi, Tina; DiNicola, Julia; Shibata, Yoshitaka; Wielgosz, Matthew; Kim, Yoon-Sang; Bauler, Matthew; Throm, Robert E; Haro-Mora, Juan J; Demirci, Selami; Bonifacino, Aylin C; Krouse, Allen E; Linde, N Seth; Donahue, Robert E; Ryu, Byoung; Tisdale, John F.
Afiliación
  • Uchida N; Cellular and Molecular Therapeutics Branch, National Heart Lung and Blood Institutes (NHLBI)/National Institute of Diabetes and Digestive and Kidney Diseases (NIDDK), National Institutes of Health (NIH), Bethesda, MD 20892, USA. uchidan@nhlbi.nih.gov.
  • Ferrara F; Division of Molecular and Medical Genetics, Center for Gene and Cell Therapy, The Institute of Medical Science, The University of Tokyo, Minato-ku, Tokyo 108-8639, Japan.
  • Drysdale CM; Department of Hematology, St. Jude Children's Research Hospital (SJCRH), Memphis, TN 38105, USA.
  • Yapundich M; Cellular and Molecular Therapeutics Branch, National Heart Lung and Blood Institutes (NHLBI)/National Institute of Diabetes and Digestive and Kidney Diseases (NIDDK), National Institutes of Health (NIH), Bethesda, MD 20892, USA.
  • Gamer J; Cellular and Molecular Therapeutics Branch, National Heart Lung and Blood Institutes (NHLBI)/National Institute of Diabetes and Digestive and Kidney Diseases (NIDDK), National Institutes of Health (NIH), Bethesda, MD 20892, USA.
  • Nassehi T; Cellular and Molecular Therapeutics Branch, National Heart Lung and Blood Institutes (NHLBI)/National Institute of Diabetes and Digestive and Kidney Diseases (NIDDK), National Institutes of Health (NIH), Bethesda, MD 20892, USA.
  • DiNicola J; Cellular and Molecular Therapeutics Branch, National Heart Lung and Blood Institutes (NHLBI)/National Institute of Diabetes and Digestive and Kidney Diseases (NIDDK), National Institutes of Health (NIH), Bethesda, MD 20892, USA.
  • Shibata Y; Cellular and Molecular Therapeutics Branch, National Heart Lung and Blood Institutes (NHLBI)/National Institute of Diabetes and Digestive and Kidney Diseases (NIDDK), National Institutes of Health (NIH), Bethesda, MD 20892, USA.
  • Wielgosz M; Cellular and Molecular Therapeutics Branch, National Heart Lung and Blood Institutes (NHLBI)/National Institute of Diabetes and Digestive and Kidney Diseases (NIDDK), National Institutes of Health (NIH), Bethesda, MD 20892, USA.
  • Kim YS; Department of Hematology, St. Jude Children's Research Hospital (SJCRH), Memphis, TN 38105, USA.
  • Bauler M; Department of Hematology, St. Jude Children's Research Hospital (SJCRH), Memphis, TN 38105, USA.
  • Throm RE; Vector Development and Production Laboratory, SJCRH, Memphis, TN 38105, USA.
  • Haro-Mora JJ; Vector Development and Production Laboratory, SJCRH, Memphis, TN 38105, USA.
  • Demirci S; Cellular and Molecular Therapeutics Branch, National Heart Lung and Blood Institutes (NHLBI)/National Institute of Diabetes and Digestive and Kidney Diseases (NIDDK), National Institutes of Health (NIH), Bethesda, MD 20892, USA.
  • Bonifacino AC; Cellular and Molecular Therapeutics Branch, National Heart Lung and Blood Institutes (NHLBI)/National Institute of Diabetes and Digestive and Kidney Diseases (NIDDK), National Institutes of Health (NIH), Bethesda, MD 20892, USA.
  • Krouse AE; Translational Stem Cell Biology Branch, NHLBI, NIH, Bethesda, MD 20892, USA.
  • Linde NS; Translational Stem Cell Biology Branch, NHLBI, NIH, Bethesda, MD 20892, USA.
  • Donahue RE; Translational Stem Cell Biology Branch, NHLBI, NIH, Bethesda, MD 20892, USA.
  • Ryu B; Cellular and Molecular Therapeutics Branch, National Heart Lung and Blood Institutes (NHLBI)/National Institute of Diabetes and Digestive and Kidney Diseases (NIDDK), National Institutes of Health (NIH), Bethesda, MD 20892, USA.
  • Tisdale JF; Department of Hematology, St. Jude Children's Research Hospital (SJCRH), Memphis, TN 38105, USA.
Sci Transl Med ; 13(591)2021 04 28.
Article en En | MEDLINE | ID: mdl-33910976
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Texto completo: 1 Colección: 01-internacional Base de datos: MEDLINE Asunto principal: Hemoglobina Fetal / Receptores de Eritropoyetina Tipo de estudio: Prognostic_studies Límite: Animals Idioma: En Revista: Sci Transl Med Asunto de la revista: CIENCIA / MEDICINA Año: 2021 Tipo del documento: Article País de afiliación: Estados Unidos Pais de publicación: Estados Unidos
Texto completo
Añadir a Mi BVS
Imprimir
XML
PubMed Links
Buscar en Google

Texto completo: 1 Colección: 01-internacional Base de datos: MEDLINE Asunto principal: Hemoglobina Fetal / Receptores de Eritropoyetina Tipo de estudio: Prognostic_studies Límite: Animals Idioma: En Revista: Sci Transl Med Asunto de la revista: CIENCIA / MEDICINA Año: 2021 Tipo del documento: Article País de afiliación: Estados Unidos Pais de publicación: Estados Unidos