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The mRNA-Binding Protein IGF2BP1 Restores Fetal Hemoglobin in Cultured Erythroid Cells from Patients with β-Hemoglobin Disorders

Sickle cell disease (SCD) and β-thalassemia are caused by structural abnormality or inadequate production of adult hemoglobin (HbA, α(2)β(2)), respectively. Individuals with either disorder are asymptomatic before birth because fetal hemoglobin (HbF, α(2)γ(2)) is unaffected. Thus, reversal of the sw...

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Detalles Bibliográficos
Autores principales: Chambers, Christopher B., Gross, Jeffrey, Pratt, Katherine, Guo, Xiang, Byrnes, Colleen, Lee, Y. Terry, Lavelle, Donald, Dean, Ann, Miller, Jeffery L., Wilber, Andrew
Formato: Online Artículo Texto
Lenguaje:English
Publicado: American Society of Gene & Cell Therapy 2020
Materias:
Acceso en línea:https://www.ncbi.nlm.nih.gov/pmc/articles/PMC7056608/
https://www.ncbi.nlm.nih.gov/pubmed/32154328
http://dx.doi.org/10.1016/j.omtm.2020.01.011