A genetic strategy to treat sickle cell anemia by coregulating globin transgene expression and RNA interference

Nature Biotechnology

Volumn 24, Issue 1, 2006, Pages 89-94

  Samakoglu, Selda ^a   Lisowski, Leszek ^a   Budak Alpdogan, Tulin ^a,b   Usachenko, Yelena ^a   Acuto, Santina ^c   Di Marzo, Rosalba ^c   Maggio, Aurelio ^c   Zhu, Ping ^a   Tisdale, John F ^d   Riviere, Isabelle ^a   Sadelain, Michel ^a  

^a MEMORIAL SLOAN KETTERING CANCER CENTER   (United States)

^b UNIVERSITY OF MEDICINE AND DENTISTRY OF NEW JERSEY   (United States)

^c VILLA SOFIA CERVELLO HOSPITAL   (Italy)

^d NATIONAL INSTITUTES OF HEALTH   (United States)

Author keywords

[No Author keywords available]

Indexed keywords

CELL CULTURE; CELLS; GENE TRANSFER; PATIENT TREATMENT; RNA; TISSUE;

CELL PROGENY; GENETIC STRATEGY; SICKLE CELL ANEMIA; STEM CELL-BASED GENE THERAPIES;

GENETIC ENGINEERING;

CD34 ANTIGEN; GAMMA GLOBIN; INTERFERON; LENTIVIRUS VECTOR; RECOMBINANT PROTEIN; SHORT HAIRPIN RNA; SMALL INTERFERING RNA;

ARTICLE; CELL BASED GENE THERAPY; CELL DIFFERENTIATION; CONTROLLED STUDY; DOWN REGULATION; ERYTHROID CELL; FEASIBILITY STUDY; GENE EXPRESSION; GENE EXPRESSION REGULATION; GENE SILENCING; GENE TARGETING; GENETIC TRANSDUCTION; HEMATOPOIETIC STEM CELL; HUMAN; HUMAN CELL; INTRON; PRIORITY JOURNAL; PROMOTER REGION; PROTEIN INDUCTION; RECOMBINANT GENE; RNA INTERFERENCE; SICKLE CELL ANEMIA; TRANSGENE; VIRAL GENE DELIVERY SYSTEM;

ANEMIA, SICKLE CELL; CELLS, CULTURED; GENE EXPRESSION REGULATION; GENE SILENCING; GENE TARGETING; GENE THERAPY; GLOBINS; HEMATOPOIETIC STEM CELLS; HUMANS; RNA INTERFERENCE; TREATMENT OUTCOME;

LENTIVIRUS;

EID: 30544436003     PISSN: 10870156     EISSN: 15461696     Source Type: Journal    
DOI: 10.1038/nbt1176     Document Type: Article

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