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- Title
Drug discovery for Diamond-Blackfan anemia using reprogrammed hematopoietic progenitors.
- Authors
Doulatov, Sergei; Vo, Linda T.; Macari, Elizabeth R.; Wahlster, Lara; Kinney, Melissa A.; Taylor, Alison M.; Barragan, Jessica; Gupta, Manav; McGrath, Katherine; Hsiang-Ying Lee; Humphries, Jessica M.; DeVine, Alex; Narla, Anupama; Alter, Blanche P.; Beggs, Alan H.; Agarwal, Suneet; Ebert, Benjamin L.; Gazda, Hanna T.; Lodish, Harvey F.; Sieff, Colin A.
- Abstract
The article presents a study that adopted a reprogramming strategy to generate expandable hematopoietic progenitor cells from induced pluripotent stem cells (iPSCs) from Diamond-Blackfan anemia (DBA) patients. Topics discussed include results showing that DBA iPSCs recapitulate early erythroid defect in vitro, DBA iPSCs recapitulate erythroid defect after transplant and gene complementation rescues erythroid defect.
- Subjects
HEMATOPOIETIC stem cells; PROGENITOR cells; PLURIPOTENT stem cells; ANEMIA; ERYTHROCYTE membranes; DISEASES
- Publication
Science Translational Medicine, 2017, Vol 9, Issue 376, p1
- ISSN
1946-6234
- Publication type
Article
- DOI
10.1126/scitranslmed.aah5645