Gene correction in patient-specific iPSCs for therapy development and disease modeling

Human Genetics

Volumn 135, Issue 9, 2016, Pages 1041-1058

  Jang, Yoon Young ^a   Ye, Zhaohui ^a  

^a JOHNS HOPKINS UNIVERSITY SCHOOL OF MEDICINE   (United States)

Author keywords

[No Author keywords available]

Indexed keywords

AGE RELATED MACULAR DEGENERATION; ALPHA 1 ANTITRYPSIN DEFICIENCY; BETA THALASSEMIA; CELL DIFFERENTIATION; CELL SURVIVAL; CELL THERAPY; CYSTIC FIBROSIS; DEGENERATIVE DISEASE; DISEASE MODEL; DUCHENNE MUSCULAR DYSTROPHY; ECTOPIC EXPRESSION; GENE THERAPY; GENETIC ENGINEERING AND GENE TECHNOLOGY; GENOME EDITING; HUMAN; INDUCED PLURIPOTENT STEM CELL; PLURIPOTENT STEM CELL; PRIORITY JOURNAL; REGENERATIVE MEDICINE; REVIEW; SEVERE COMBINED IMMUNODEFICIENCY; SICKLE CELL ANEMIA; STARGARDT DISEASE; TRINUCLEOTIDE REPEAT; ALPHA 1-ANTITRYPSIN DEFICIENCY; ANEMIA, SICKLE CELL; BETA-THALASSEMIA; BIOLOGICAL MODEL; CYTOLOGY; GENE EDITING; MUSCULAR DYSTROPHY, DUCHENNE;

ALPHA 1-ANTITRYPSIN DEFICIENCY; ANEMIA, SICKLE CELL; BETA-THALASSEMIA; CYSTIC FIBROSIS; GENE EDITING; GENETIC THERAPY; HUMANS; INDUCED PLURIPOTENT STEM CELLS; MODELS, BIOLOGICAL; MUSCULAR DYSTROPHY, DUCHENNE; REGENERATIVE MEDICINE; SEVERE COMBINED IMMUNODEFICIENCY;

EID: 84973094139     PISSN: 03406717     EISSN: 14321203     Source Type: Journal    
DOI: 10.1007/s00439-016-1691-5     Document Type: Review

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