Treating Immunodeficiency through HSC Gene Therapy

Trends in Molecular Medicine

Volumn 22, Issue 4, 2016, Pages 317-327

  Booth, Claire ^a,b   Gaspar, H Bobby ^a,b   Thrasher, Adrian J ^a,b  

^a UNIVERSITY COLLEGE LONDON   (United Kingdom)

^b GREAT ORMOND STREET HOSPITAL   (United Kingdom)

Author keywords

Gene therapy; Lentiviral vectors; Primary immunodeficiency

Indexed keywords

RETROVIRUS VECTOR; TRANSCRIPTION ACTIVATOR LIKE EFFECTOR NUCLEASE; ZINC FINGER NUCLEASE;

BIOSAFETY; CLINICAL EFFECTIVENESS; CRISPR CAS SYSTEM; HEMATOPOIETIC STEM CELL; HUMAN; IMMUNE DEFICIENCY; LEUKEMOGENESIS; PHASE 1 CLINICAL TRIAL (TOPIC); PHASE 2 CLINICAL TRIAL (TOPIC); REVIEW; STEM CELL GENE THERAPY; TARGETED GENE REPAIR; ANIMAL; CLINICAL TRIAL (TOPIC); CYTOLOGY; GENE EXPRESSION; GENE THERAPY; GENE VECTOR; GENETIC TRANSDUCTION; GENETICS; HEMATOPOIETIC STEM CELL TRANSPLANTATION; IMMUNOLOGIC DEFICIENCY SYNDROMES; LENTIVIRINAE; METABOLISM; PROCEDURES; TRANSGENE;

ANIMALS; CLINICAL TRIALS AS TOPIC; GENE EXPRESSION; GENETIC THERAPY; GENETIC VECTORS; HEMATOPOIETIC STEM CELL TRANSPLANTATION; HEMATOPOIETIC STEM CELLS; HUMANS; IMMUNOLOGIC DEFICIENCY SYNDROMES; LENTIVIRUS; TARGETED GENE REPAIR; TRANSDUCTION, GENETIC; TRANSGENES;

EID: 84961170446     PISSN: 14714914     EISSN: 1471499X     Source Type: Journal    
DOI: 10.1016/j.molmed.2016.02.002     Document Type: Review

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