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PLoS Genetics, ISSN 1553-7390, 05/2017, Volume 13, Issue 5, pp. e1006794 - e1006794
Journal Article
The New England Journal of Medicine, ISSN 0028-4793, 03/2017, Volume 376, Issue 9, pp. 848 - 855
Journal Article
Nature Medicine, ISSN 1078-8956, 2014, Volume 20, Issue 4, pp. 398 - 407
Journal Article
Human Gene Therapy, ISSN 1043-0342, 02/2016, Volume 27, Issue 2, pp. 148 - 165
Journal Article
The New England Journal of Medicine, ISSN 0028-4793, 04/2018, Volume 378, Issue 16, pp. 1479 - 1493
Gene therapy with CD34+ cells transduced with a lentivirus vector carrying a β-globin gene was performed in 22 patients. At a median of 26 months, all the... 
MEDICINE, GENERAL & INTERNAL | HEMOGLOBINOPATHIES | GLOBIN GENE | EFFICACY | BONE-MARROW-TRANSPLANTATION | ERYTHROPOIESIS | DISEASE | STEM-CELL TRANSPLANTATION | BURDEN | CONDITIONING REGIMEN | VECTOR | Index Medicus | Abridged Index Medicus
Journal Article
Molecular Therapy - Methods & Clinical Development, ISSN 2329-0501, 03/2019, Volume 12, pp. 175 - 183
Elements within the γ-hemoglobin promoters ( and ) function to bind transcription complexes that mediate repression of fetal hemoglobin expression. Sickle cell... 
thalassemia | SCD | TALEN | HSC | HPFH | HBG | hemoglobinopathy | gene editing | hemoglobin | sickle cell disease | MEDICINE, RESEARCH & EXPERIMENTAL | BETA-HEMOGLOBINOPATHIES | GLOBIN | DISEASE | EXPRESSION | MULTILINEAGE ENGRAFTMENT | GENOME
Journal Article
Nature Medicine, ISSN 1078-8956, 04/2014, Volume 20, Issue 4, p. 398
  The pathophysiology of ineffective erythropoiesis in [beta]-thalassemia is poorly understood. We report that RAP-011, an activin receptor IIA (ActRIIA)... 
Erythropoiesis | Care and treatment | Cytokines | Physiological aspects | Thalassemia | Development and progression | Genetic aspects | Research | Hemoglobin | Ligands | Cellular biology | Molecular biology
Journal Article
Journal Article
Nature, ISSN 0028-0836, 2014, Volume 514, Issue 7521, pp. 242 - 246
Journal Article
Molecular Therapy, ISSN 1525-0016, 02/2018, Volume 26, Issue 2, pp. 480 - 495
Journal Article