CRISPR-Cas9 interrogation of a putative fetal globin repressor in human erythroid cells

• Published in: PloS one Vol. 14; no. 1; p. e0208237
• Main Authors: Chung, Jennifer E, Magis, Wendy, Vu, Jonathan, Heo, Seok-Jin, Wartiovaara, Kirmo, Walters, Mark C, Kurita, Ryo, Nakamura, Yukio, Boffelli, Dario, Martin, David I K, Corn, Jacob E, DeWitt, Mark A
• Format: Journal Article
• Language: English
• Published: United States Public Library of Science 15.01.2019; Public Library of Science (PLoS)
• Subjects: Analysis; Binding sites; Biology and Life Sciences; CD34 antigen; Cell Line; Cells (biology); Chung, Wendy; Clonal deletion; CRISPR; CRISPR-Associated Protein 9 - metabolism; CRISPR-Cas Systems - genetics; CRISPR-Cas technology; Disease; DNA, Intergenic - genetics; Erythroid cells; Erythroid Cells - metabolism; Fetal Hemoglobin - metabolism; Fetuses; gamma-Globins - genetics; Gene deletion; Gene Editing; Gene mutation; Gene Silencing; Genes; Genetic engineering; Genetic modification; Genome editing; Genomes; Genomics; Genotype; Globin genes; Hematological diseases; Hematopoietic stem cells; Hematopoietic Stem Cells - metabolism; Hemoglobin; Hospitals; Humans; Interrogation; Methods; Mutation; Phenotype; Progenitor cells; Red blood cells; Regulatory sequences; Repressor Proteins - metabolism; Research and Analysis Methods; Sequence Deletion - genetics; Sickle cell disease; Stem cells; Thalassemia; Therapeutic applications; Transcription factors; Transplants & implants; Up-Regulation - genetics; California; United States > US; Japan; Finland

Sickle Cell Disease and ß-thalassemia, which are caused by defective or deficient adult ß-globin (HBB) respectively, are the most common serious genetic blood diseases in the world. Persistent expression of the fetal ß-like globin, also known as 𝛾-globin, can ameliorate both disorders by serving in...