Gene therapy for ADA-SCID, the first marketing approval of an ex vivo gene therapy in Europe: paving the road for the next generation of advanced therapy medicinal products

EMBO Molecular Medicine

Volumn 9, Issue 6, 2017, Pages 737-740

  Aiuti, Alessandro ^a,b   Roncarolo, Maria Grazia ^a,b,c   Naldini, Luigi ^a,b  

^a SAN RAFFAELE SCIENTIFIC INSTITUTE   (Italy)

^b VITA SALUTE SAN RAFFAELE UNIVERSITY   (Italy)

^c STANFORD UNIVERSITY SCHOOL OF MEDICINE   (United States)

Author keywords

[No Author keywords available]

Indexed keywords

STRIMVELIS; ADA PROTEIN, HUMAN; ADENOSINE DEAMINASE;

ADENOSINE DEAMINASE DEFICIENCY; DISEASE SEVERITY; DRUG APPROVAL; DRUG EFFECT; EUROPE; EX VIVO STUDY; GENE FUNCTION; GENE THERAPY; HEMATOPOIETIC STEM CELL TRANSPLANTATION; HUMAN; IMMUNOGENICITY; MULTIPOTENT STEM CELL; NOTE; PRIORITY JOURNAL; SEVERE COMBINED IMMUNODEFICIENCY; SURVIVAL RATE; AGAMMAGLOBULINEMIA; AUTOTRANSPLANTATION; CHILD; DEFICIENCY; DRUG THERAPY; GENE VECTOR; GENETICS;

ADENOSINE DEAMINASE; AGAMMAGLOBULINEMIA; CHILD; EUROPE; GENETIC THERAPY; GENETIC VECTORS; HEMATOPOIETIC STEM CELL TRANSPLANTATION; HUMANS; SEVERE COMBINED IMMUNODEFICIENCY; TRANSPLANTATION, AUTOLOGOUS;

EID: 85017415923     PISSN: 17574676     EISSN: 17574684     Source Type: Journal    
DOI: 10.15252/emmm.201707573     Document Type: Note

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