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A human H1-HBB11-GFP reporter embryonic stem cell line (WAe001-A-2) generated using TALEN-based genome editing.
Alexeeva, Vera; Aydin, Iraz T; Schaniel, Christoph; Stranahan, Alec W; D'Souza, Sunita L; Bieker, James J.
  • Alexeeva V; Department of Immunology, Memorial Sloan Kettering Cancer Center, New York, NY, USA.
  • Aydin IT; Atreca, Inc., South San Francisco, CA, USA.
  • Schaniel C; Black Family Stem Cell Institute, Icahn School of Medicine at Mount Sinai, New York, NY, USA; Mount Sinai Institute for Systems Biomedicine, Department of Pharmacological Sciences, Icahn School of Medicine at Mount Sinai, New York, NY, USA.
  • Stranahan AW; Department of Cellular, Developmental, and Regenerative Biology, Icahn School of Medicine at Mount Sinai, New York, NY, USA.
  • D'Souza SL; Center for Modeling Pediatric Diseases, St Jude's Children's Research Hospital, Memphis, TN, USA.
  • Bieker JJ; Black Family Stem Cell Institute, Icahn School of Medicine at Mount Sinai, New York, NY, USA; Department of Cellular, Developmental, and Regenerative Biology, Icahn School of Medicine at Mount Sinai, New York, NY, USA. Electronic address: james.bieker@mssm.edu.
Stem Cell Res ; 45: 101837, 2020 05.
Article en En | MEDLINE | ID: mdl-32413789
ABSTRACT
Hemoglobin production during mammalian development is characterized by temporal switches of the genes coding for the α- and ß-globin chains. Defects in this controlled process can lead to hemoglobinapathies such as sickle cell disease and ß-thalassemia. The ability of human embryonic stem cells (hESC) to proceed through hematopoiesis could provide a clinically useful source of red blood cells. However, hESC-derived red cells exhibit an embryonic/fetal, but not adult, mode of hemoglobin expression. The resource described here is a hESC line engineered to express a reporter from its adult globin promoter, providing a screening platform for small molecules that lead to efficient induction of adult globin.
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Texto completo: 1 Banco de datos: MEDLINE Asunto principal: Nucleasas de los Efectores Tipo Activadores de la Transcripción / Edición Génica Límite: Adult / Animals / Humans Idioma: En Año: 2020 Tipo del documento: Article

Texto completo: 1 Banco de datos: MEDLINE Asunto principal: Nucleasas de los Efectores Tipo Activadores de la Transcripción / Edición Génica Límite: Adult / Animals / Humans Idioma: En Año: 2020 Tipo del documento: Article