Genome editing using CRISPR-Cas9 to create the HPFH genotype in HSPCs: An approach for treating sickle cell disease and β-thalassemia

Proceedings of the National Academy of Sciences of the United States of America

Volumn 113, Issue 38, 2016, Pages 10661-10665

  Ye, Lin ^a,b   Wang, Jiaming ^a,b   Tan, Yuting ^a,c,d   Beyer, Ashley I ^e   Xie, Fei ^a,b   Muench, Marcus O ^a,e   Kan, Yuet Wai ^a,b  

^a UNIVERSITY OF CALIFORNIA   (United States)

^b UNIVERSITY OF CALIFORNIA   (United States)

^c SHANGHAI JIAO TONG UNIVERSITY   (China)

^d SHANGHAI JIAO TONG UNIVERSITY SCHOOL OF MEDICINE   (China)

^e BLOOD SYSTEMS RESEARCH INSTITUTE   (United States)

Author keywords

Colony assay; Deletion; Engineered nucleases; Erythroid differentiation; Fetal hemoglobin

Indexed keywords

CD34 ANTIGEN; DEOXYRIBONUCLEASE I; GLOBIN MESSENGER RNA; GUIDE RNA; HEMOGLOBIN BETA CHAIN; HEMOGLOBIN F; HEMOGLOBIN GAMMA CHAIN;

ARTICLE; BETA THALASSEMIA; BINDING SITE; CELL DIFFERENTIATION; CONTROLLED STUDY; CRISPR CAS SYSTEM; DNA END JOINING REPAIR; ENHANCER REGION; GENE DELETION; GENE EXPRESSION; GENE LOCUS; GENE MUTATION; GENE TARGETING; GENETIC DISORDER; GENETIC PROCEDURES; GENOME EDITING; GENOTYPE; HEMATOPOIETIC STEM CELL; HEREDITARY PERSISTENCE OF FETAL HEMOGLOBIN; HOMOZYGOSITY; HUMAN; HUMAN CELL; HYPERHEMOGLOBINEMIA; INDUCED PLURIPOTENT STEM CELL; NONHUMAN; PRIORITY JOURNAL; SICKLE CELL ANEMIA; STAPHYLOCOCCUS AUREUS; GENE EDITING; GENE THERAPY; GENETICS; HETEROZYGOTE; HUMAN GENOME; METABOLISM; PATHOLOGY; PROCEDURES;

ANEMIA, SICKLE CELL; BETA-THALASSEMIA; CRISPR-CAS SYSTEMS; DNA END-JOINING REPAIR; FETAL HEMOGLOBIN; GENE EDITING; GENETIC THERAPY; GENOME, HUMAN; GENOTYPE; HEMATOPOIETIC STEM CELLS; HETEROZYGOTE; HUMANS; SEQUENCE DELETION;

EID: 84988643091     PISSN: 00278424     EISSN: 10916490     Source Type: Journal    
DOI: 10.1073/pnas.1612075113     Document Type: Article

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