TALEN-Mediated Gene Editing of HBG in Human Hematopoietic Stem Cells Leads to Therapeutic Fetal Hemoglobin Induction

Elements within the γ-hemoglobin promoters (HBG1 and HBG2) function to bind transcription complexes that mediate repression of fetal hemoglobin expression. Sickle cell disease (SCD) subjects with a 13-bp deletion in the HBG1 promoter exhibit a clinically favorable hereditary persistence of fetal hem...

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Detalles Bibliográficos
Autores principales: Lux, Christopher T., Pattabhi, Sowmya, Berger, Mason, Nourigat, Cynthia, Flowers, David A., Negre, Olivier, Humbert, Olivier, Yang, Julia G., Lee, Calvin, Jacoby, Kyle, Bernstein, Irwin, Kiem, Hans-Peter, Scharenberg, Andrew, Rawlings, David J.
Formato: Online Artículo Texto
Lenguaje:English
Publicado: American Society of Gene & Cell Therapy 2018
Materias:
Article
Acceso en línea:https://www.ncbi.nlm.nih.gov/pmc/articles/PMC6348980/
https://www.ncbi.nlm.nih.gov/pubmed/30705922
http://dx.doi.org/10.1016/j.omtm.2018.12.008

Internet

https://www.ncbi.nlm.nih.gov/pmc/articles/PMC6348980/
https://www.ncbi.nlm.nih.gov/pubmed/30705922
http://dx.doi.org/10.1016/j.omtm.2018.12.008

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