Seamless correction of the sickle cell disease mutation of the HBB gene in human induced pluripotent stem cells using TALENs

Biotechnology and Bioengineering

Volumn 111, Issue 5, 2014, Pages 1048-1053

  Sun, Ning ^a   Zhao, Huimin ^a  

^a UNIVERSITY OF ILLINOIS AT URBANA CHAMPAIGN   (United States)

Author keywords

Gene therapy; Genome editing; Induced pluripotent stem cells; piggyBac transposon; Sickle cell disease; TAL effector nucleases

Indexed keywords

CYTOLOGY; GENE THERAPY; PATIENT TREATMENT; STEM CELLS; TRANSCRIPTION;

AUTOLOGOUS CELL SOURCE; GENETIC DISORDERS; HUMAN GENETIC DISEASE; HUMAN-INDUCED PLURIPOTENT STEM CELLS; INDUCED PLURIPOTENT STEM CELLS; PIGGYBAC TRANSPOSONS; SICKLE CELL DISEASE; TAL EFFECTOR NUCLEASES;

DISEASES;

DEOXYRIBONUCLEASE; GENOMIC DNA; HEMOGLOBIN BETA CHAIN; TRANSCRIPTION ACTIVATOR LIKE EFFECTOR NUCLEASE; UNCLASSIFIED DRUG;

ARTICLE; CELL SUSPENSION; CHROMOSOME TRANSLOCATION; CODON; DNA END JOINING REPAIR; DNA REPAIR; GENE LOCUS; GENE MUTATION; GENE TARGETING; GENE THERAPY; GENOMIC FRAGMENT; GENOTYPE; HUMAN; HUMAN CELL; INDEL MUTATION; INVERTED REPEAT; PLASMID; PLURIPOTENT STEM CELL; POLYMERASE CHAIN REACTION; PROMOTER REGION; SEQUENCE ANALYSIS; SEQUENCE HOMOLOGY; SICKLE CELL ANEMIA; START CODON; TRANSPOSON;

GENE THERAPY; GENOME EDITING; INDUCED PLURIPOTENT STEM CELLS; PIGGYBAC TRANSPOSON; SICKLE CELL DISEASE; TAL EFFECTOR NUCLEASES;

ANEMIA, SICKLE CELL; BETA-GLOBINS; CELLS, CULTURED; CLONING, MOLECULAR; DEOXYRIBONUCLEASES; GENETIC THERAPY; HUMANS; INDUCED PLURIPOTENT STEM CELLS;

EID: 84897042820     PISSN: 00063592     EISSN: 10970290     Source Type: Journal    
DOI: 10.1002/bit.25018     Document Type: Article

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