Development of mammalian artificial chromosomes for the treatment of genetic diseases: Sandhoff and Krabbe diseases

Expert Opinion on Biological Therapy

Volumn 5, Issue 2, 2005, Pages 195-206

  Bunnell, Bruce A ^a   Izadpanah, Reza ^a   Ledebur Jr , Harry C ^b   Perez, Carl F ^b  

^a TULANE UNIVERSITY HEALTH SCIENCES CENTER   (United States)

^b Chromos Molecular Systems Inc   (Canada)

Author keywords

Artificial chromosomes; Gene therapy; Krabbe disease; Lysosomal storage disease; Sandhoff disease; Stem cells

Indexed keywords

GALC GENE; HEXB GENE; SATELLITE DNA; UNCLASSIFIED DRUG; VIRUS VECTOR;

ARTIFICIAL CHROMOSOME; CELL TRANSFER; CENTROMERE; DNA SEQUENCE; EPISOME; EX VIVO STUDY; EXPRESSION VECTOR; GENE TARGETING; GENE THERAPY; GENETIC DISORDER; GLOBOID CELL LEUKODYSTROPHY; HEMATOPOIETIC STEM CELL; LYSOSOME STORAGE DISEASE; MESENCHYMAL STEM CELL; NONHUMAN; PURIFICATION; REVIEW; SAFETY; SANDHOFF DISEASE;

ANIMALS; CHROMOSOMES, ARTIFICIAL, MAMMALIAN; GENE THERAPY; GENETIC VECTORS; HUMANS; LEUKODYSTROPHY, GLOBOID CELL; SANDHOFF DISEASE;

EID: 15244354387     PISSN: 14712598     EISSN: None     Source Type: Journal    
DOI: 10.1517/14712598.5.2.195     Document Type: Review

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