Customizing the genome as therapy for the β-hemoglobinopathies

Blood

Volumn 127, Issue 21, 2016, Pages 2536-2545

  Canver, Matthew C ^a   Orkin, Stuart H ^a,b,c,d  

^a BOSTON CHILDREN S HOSPITAL   (United States)

^b DANA FARBER CANCER INSTITUTE   (United States)

^c HARVARD MEDICAL SCHOOL   (United States)

^d HOWARD HUGHES MEDICAL INSTITUTE   (United States)

Author keywords

[No Author keywords available]

Indexed keywords

CD34 ANTIGEN; GRANULOCYTE COLONY STIMULATING FACTOR; HEMOGLOBIN BETA CHAIN; HEMOGLOBIN F; PLERIXAFOR; PROTEIN MYB; TRANSCRIPTION ACTIVATOR LIKE EFFECTOR NUCLEASE; TRANSCRIPTION FACTOR; TRANSCRIPTION FACTOR BCL11A; TRANSCRIPTION FACTOR KLF1; TRANSCRIPTION FACTOR LRF; UNCLASSIFIED DRUG; ZINC FINGER NUCLEASE;

AUTOLOGOUS BONE MARROW TRANSPLANTATION; AUTOLOGOUS STEM CELL TRANSPLANTATION; BETA THALASSEMIA; CLUSTERED REGULARLY INTERSPACED SHORT PALINDROMIC REPEAT; DNA END JOINING REPAIR; GENE DISRUPTION; GENE LOCUS; GENETIC DISORDER; HEMATOPOIETIC STEM CELL; HEMOGLOBINOPATHY; HUMAN; NONHUMAN; PRIORITY JOURNAL; PROTEIN EXPRESSION; REVIEW; SICKLE CELL ANEMIA; STEM CELL; ANIMAL; AUTOGRAFT; GENE EDITING; GENETICS; HEMATOPOIETIC STEM CELL TRANSPLANTATION; HEMOGLOBINOPATHIES; PROCEDURES; TARGETED GENE REPAIR;

ANIMALS; AUTOGRAFTS; GENE EDITING; HEMATOPOIETIC STEM CELL TRANSPLANTATION; HEMOGLOBINOPATHIES; HUMANS; TARGETED GENE REPAIR;

EID: 84974602652     PISSN: 00064971     EISSN: 15280020     Source Type: Journal    
DOI: 10.1182/blood-2016-01-678128     Document Type: Review

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