Strategies for retrovirus-based correction of Severe, Combined Immunodeficiency (SCID)

Methods in Enzymology

Volumn 507, Issue , 2012, Pages 15-27

  Fischer, Alain ^a,b   Hacein Bey Abina, Salima ^a,b   Cavazzana Calvo, Marina ^a,b  

^a UNIVERSITÉ PARIS DESCARTES   (France)

^b HÔPITAL NECKER ENFANTS MALADES   (France)

Author keywords

Adenosine deaminase deficiency; Ex vivo gene therapy; Insertional mutagenesis; Retrovirus; SCID X1; Self inactivated retrovirus; Severe combined immunodeficiency

Indexed keywords

BUSULFAN; LENTIVIRUS VECTOR; MELPHALAN; RETROVIRUS VECTOR;

ADENOSINE DEAMINASE DEFICIENCY; ARTICLE; BIOSAFETY; CELL BASED GENE THERAPY; CLINICAL TRIAL (TOPIC); DISEASE SEVERITY; DRUG DESIGN; DRUG EFFICACY; DRUG SAFETY; ENHANCER REGION; EX VIVO GENE TRANSFER; GENE DELETION; GENE LOCUS; GENOTOXICITY; HEMATOPOIETIC STEM CELL; HUMAN; MYELOABLATIVE CONDITIONING; NONHUMAN; PRIORITY JOURNAL; PROMOTER REGION; PROTO ONCOGENE; TANDEM REPEAT; TRANSACTIVATION; VIRAL GENE DELIVERY SYSTEM; VIRAL GENE THERAPY; VIRUS DNA CELL DNA INTERACTION; X LINKED SEVERE COMBINED IMMUNODEFICIENCY;

EID: 84857584749     PISSN: 00766879     EISSN: 15577988     Source Type: Book Series    
DOI: 10.1016/B978-0-12-386509-0.00002-8     Document Type: Chapter

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