Good Laboratory Practice Preclinical Safety Studies for GSK2696273 (MLV Vector-Based Ex Vivo Gene Therapy for Adenosine Deaminase Deficiency Severe Combined Immunodeficiency) in NSG Mice

Human Gene Therapy Clinical Development

Volumn 28, Issue 1, 2017, Pages 17-27

  Carriglio, Nicola ^a,b   Klapwijk, Jan ^c   Hernandez, Raisa Jofra ^a,b   Vezzoli, Michela ^b   Chanut, Franck ^c   Lowe, Rhiannon ^c   Elena, Draghici ^a,b   Nord, Melanie ^d   Albertini, Paola ^b   Cristofori, Patrizia ^b,c   Richards, Jane ^c   Staton, Hazel ^e   Appleby, Jonathan ^c   Aiuti, Alessandro ^a,f   Sauer, Aisha V ^a,b  

^a SAN RAFFAELE SCIENTIFIC INSTITUTE   (Italy)

^b SAN RAFFAELE HOSPITAL   (Italy)

^c GLAXOSMITHKLINE   (United Kingdom)

^d GLAXOSMITHKLINE   (United States)

^e Department of Genetic and Molecular Toxicology   (United Kingdom)

^f VITA SALUTE SAN RAFFAELE UNIVERSITY   (Italy)

Author keywords

ADA SCID; adenosine deaminase; biodistribution; gene therapy; GLP; retroviral vector

Indexed keywords

ADENOSINE DEAMINASE; CD34 ANTIGEN; GSK 2696273; RETROVIRUS VECTOR; UNCLASSIFIED DRUG; ADA PROTEIN, HUMAN;

ADENOSINE DEAMINASE DEFICIENCY; ANIMAL EXPERIMENT; ANIMAL MODEL; ANIMAL TISSUE; ARTICLE; CELL DIFFERENTIATION; CELL LINEAGE; CHIMERA; CONTROLLED STUDY; DRUG SAFETY; ENGRAFTMENT; EUROPEAN MEDICINES AGENCY; EX VIVO GENE TRANSFER; FEMALE; GOOD LABORATORY PRACTICE; HUMAN; HUMAN CELL; MALE; MOUSE; NOD SCID GAMMA MOUSE; NONHUMAN; PILOT STUDY; PRECLINICAL STUDY; PRIORITY JOURNAL; UMBILICAL CORD BLOOD CELL; VIRAL GENE THERAPY; AGAMMAGLOBULINEMIA; ANIMAL; DEFICIENCY; GENE THERAPY; GENE TRANSFER; GENE VECTOR; GENETICS; LABORATORY; NONOBESE DIABETIC MOUSE; SCID MOUSE; SEVERE COMBINED IMMUNODEFICIENCY; STANDARDS; THERAPEUTIC USE; TISSUE DISTRIBUTION;

ADENOSINE DEAMINASE; AGAMMAGLOBULINEMIA; ANIMALS; DRUG EVALUATION, PRECLINICAL; FEMALE; GENE TRANSFER TECHNIQUES; GENETIC THERAPY; GENETIC VECTORS; HUMANS; LABORATORIES; MALE; MICE; MICE, INBRED NOD; MICE, SCID; SEVERE COMBINED IMMUNODEFICIENCY; TISSUE DISTRIBUTION;

EID: 85016102547     PISSN: 23248637     EISSN: 23248645     Source Type: Journal    
DOI: 10.1089/humc.2016.191     Document Type: Article

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