Is genetic rescue of cystinosis an achievable treatment goal?

Nephrology Dialysis Transplantation

Volumn 29, Issue 3, 2014, Pages 522-528

  Cherqui, Stephanie ^a  

^a UNIVERSITY OF CALIFORNIA SAN DIEGO   (United States)

Author keywords

Cystinosis; Hematopoietic stem cells; Kidney preservation; Lentivirus vector; Tissue cystine decrease

Indexed keywords

COMPLEMENTARY DNA; CYSTINE; CYSTINOSIN; LENTIVIRUS VECTOR; MEMBRANE PROTEIN; MERCAPTAMINE; UNCLASSIFIED DRUG; AMINO ACID TRANSPORTER; CTNS PROTEIN, HUMAN;

ADULT STEM CELL; ALLOGENEIC HEMATOPOIETIC STEM CELL TRANSPLANTATION; AUTOLOGOUS HEMATOPOIETIC STEM CELL TRANSPLANTATION; CTNS GENE; CYSTINOSIS; DENDRITIC CELL; DIGESTIVE SYSTEM FUNCTION DISORDER; END STAGE RENAL DISEASE; ENGRAFTMENT; FANCONI RENOTUBULAR SYNDROME; GENE; GENE THERAPY; GRAFT VERSUS HOST REACTION; HUMAN; KIDNEY DYSFUNCTION; KUPFFER CELL; NONHUMAN; ODOR; PATIENT COMPLIANCE; PRIORITY JOURNAL; REVIEW; STEM CELL GENE THERAPY; UNSPECIFIED SIDE EFFECT; ANIMAL; BIOSYNTHESIS; GENE TRANSFER; GENETICS; HEMATOPOIETIC STEM CELL TRANSPLANTATION; KIDNEY; KNOCKOUT MOUSE; METABOLISM; MOTIVATION; PATHOLOGY;

AMINO ACID TRANSPORT SYSTEMS, NEUTRAL; ANIMALS; CYSTINOSIS; GENE TRANSFER TECHNIQUES; GENETIC THERAPY; GOALS; HEMATOPOIETIC STEM CELL TRANSPLANTATION; HUMANS; KIDNEY; MICE, KNOCKOUT;

EID: 84895756679     PISSN: 09310509     EISSN: 14602385     Source Type: Journal    
DOI: 10.1093/ndt/gft270     Document Type: Review

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