Somatic correction of junctional epidermolysis bullosa by a highly recombinogenic AAV variant

Molecular Therapy

Volumn 22, Issue 4, 2014, Pages 725-733

  Melo, Sandra P ^a   Lisowski, Leszek ^a,b   Bashkirova, Elizaveta ^a   Zhen, Hanson H ^a   Chu, Kirk ^a   Keene, Douglas R ^c   Marinkovich, M Peter ^a,d   Kay, Mark A ^a   Oro, Anthony E ^a  

^a STANFORD UNIVERSITY SCHOOL OF MEDICINE   (United States)

^b SALK INSTITUTE FOR BIOLOGICAL STUDIES   (United States)

^c SHRINERS HOSPITAL FOR CHILDREN   (United States)

^d VA PALO ALTO HEALTH CARE SYSTEM   (United States)

Author keywords

[No Author keywords available]

Indexed keywords

GENE THERAPY AGENT; KALININ; LAMININ ALPHA3; PARVOVIRUS VECTOR; UNCLASSIFIED DRUG; COL7A1 PROTEIN, HUMAN; COLLAGEN TYPE 7; LAMININ; LAMININ ALPHA 3;

ANIMAL EXPERIMENT; ANIMAL MODEL; ANIMAL TISSUE; ARTICLE; CELL MUTANT; CONTROLLED STUDY; EPIDERMOLYSIS BULLOSA; GENE LOCUS; GENE MUTATION; GENE TARGETING; HOMOLOGOUS RECOMBINATION; HUMAN; HUMAN CELL; HUMAN TISSUE; IN VITRO STUDY; IN VIVO STUDY; KERATINOCYTE; MOUSE; NONHUMAN; PROTEIN EXPRESSION; SEROTYPE; SKIN GRAFT; SOMATIC GENE THERAPY; VIRAL GENE DELIVERY SYSTEM; VIRAL GENE THERAPY; ANIMAL; DEPENDOPARVOVIRUS; EPIDERMOLYSIS BULLOSA, JUNCTIONAL; GENE THERAPY; GENETICS; METABOLISM; MUTATION; PATHOLOGY; PROCEDURES; XENOGRAFT;

MAMMALIA;

ANIMALS; COLLAGEN TYPE VII; DEPENDOVIRUS; EPIDERMOLYSIS BULLOSA, JUNCTIONAL; GENETIC THERAPY; HETEROGRAFTS; HOMOLOGOUS RECOMBINATION; HUMANS; KERATINOCYTES; LAMININ; MICE; MUTATION;

EID: 84897572739     PISSN: 15250016     EISSN: 15250024     Source Type: Journal    
DOI: 10.1038/mt.2013.290     Document Type: Article

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