TALEN-Mediated Gene Editing of HBG in Human Hematopoietic Stem Cells Leads to Therapeutic Fetal Hemoglobin Induction - 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; et al. (Molecular therapy. Methods & clinical development, 2019)
Keywords: Binding sites; CD34 antigen; Cell differentiation; Clonal deletion; Deoxyribonucleic acid; Design; Disease; DNA; Fetuses; Flow cytometry; Gene deletion; Gene silencing; Genome editing; Genotype & phenotype; Hematopoietic stem cells; Hemoglobin; High-performance liquid chromatography; Medical research; Mutation; Phenotypes; Promoters; Proteins; Sickle cell disease; Stem cell transplantation; Stem cells; Thalassemia; Transcription activator-like effector nucleases; Transcription factors; Transfection; Transplants & implants; thalassemia; SCD; TALEN; HSC; HPFH; HBG; gene editing; hemoglobinopathy; hemoglobin; sickle cell disease