Development of novel efficient sin vectors with improved safety features for Wiskott-Aldrich syndrome stem cell based gene therapy

Molecular Pharmaceutics

Volumn 8, Issue 5, 2011, Pages 1525-1537

  Avedillo Díez, Inés ^a,b,c   Zychlinski, Daniela ^b   Coci, Emanuele G ^a,b   Galla, Melanie ^b   Modlich, Ute ^b   Dewey, Ricardo A ^a,c   Schwarzer, Adrian ^b   Maetzig, Tobias ^b   Mpofu, Nonsikelelo ^b   Jaeckel, Elmar ^b   Boztug, Kaan ^a,b   Baum, Christopher ^a,b   Klein, Christoph ^a,b   Schambach, Axel ^b  

^a LUDWIG MAXIMILIANS UNIVERSITY   (Germany)

^b HANNOVER MEDICAL SCHOOL   (Germany)

^c INSTITUTO DE INVESTIGACIONES BIOTECNOLÓGICAS   (Argentina)

Author keywords

codon optimization; gene therapy; self inactivating

Indexed keywords

CD14 ANTIGEN; CD34 ANTIGEN; COMPLEMENTARY DNA; ELONGATION FACTOR 1ALPHA; LENTIVIRUS VECTOR; SELF INACTIVATING VECTOR; UNCLASSIFIED DRUG; WISKOTT ALDRICH SYNDROME PROTEIN;

ARTICLE; CODON; GENE VECTOR; HUMAN; HUMAN CELL; IN VITRO STUDY; PHENOTYPE; PRIORITY JOURNAL; PROTEIN EXPRESSION; STEM CELL GENE THERAPY; WISKOTT ALDRICH SYNDROME;

ANIMALS; ANTIGENS, CD34; CELL DIFFERENTIATION; CELL LINE; COMBINED MODALITY THERAPY; FEASIBILITY STUDIES; GENE EXPRESSION REGULATION, VIRAL; GENE THERAPY; GENETIC VECTORS; HEMATOPOIETIC STEM CELLS; HEPATITIS B VIRUS, WOODCHUCK; HUMANS; LENTIVIRUS; MICE; MICE, KNOCKOUT; MYELOID CELLS; PEPTIDE ELONGATION FACTOR 1; PROMOTER REGIONS, GENETIC; RNA, MESSENGER; STEM CELL TRANSPLANTATION; WISKOTT-ALDRICH SYNDROME; WISKOTT-ALDRICH SYNDROME PROTEIN;

EID: 80053512736     PISSN: 15438384     EISSN: 15438392     Source Type: Journal    
DOI: 10.1021/mp200132u     Document Type: Article

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