Construction and validation of an RNA trans-splicing molecule suitable to repair a large number of COL7A1 mutations

Gene Therapy

Volumn 23, Issue 11, 2016, Pages 775-784

  Tockner, B ^a   Kocher, T ^a   Hainzl, S ^a   Reichelt, J ^a   Bauer, J W ^a   Koller, U ^a   Murauer, E M ^a  

^a PARACELSUS MEDICAL UNIVERSITY   (Austria)

Author keywords

[No Author keywords available]

Indexed keywords

COLLAGEN TYPE 7; COLLAGEN TYPE 7 ALPHA 1; COMPLEMENTARY DNA; MESSENGER RNA PRECURSOR; UNCLASSIFIED DRUG; COL7A1 PROTEIN, HUMAN; MESSENGER RNA;

ADHERENT CELL; ARTICLE; DISEASE ASSOCIATION; DNA SYNTHESIS; EMBRYO; EPIDERMOLYSIS BULLOSA DYSTROPHICA; FLOW CYTOMETRY; GEL ELECTROPHORESIS; GENE MUTATION; GENE THERAPY; GENETIC SCREENING; GENETIC TRANSFECTION; HEK293 CELL LINE; HUMAN; HUMAN CELL; IMMUNOFLUORESCENCE; KERATINOCYTE; PRIORITY JOURNAL; RESTRICTION SITE; REVERSE TRANSCRIPTION POLYMERASE CHAIN REACTION; RNA ISOLATION; RNA REPAIR; RNA TRANSCRIPTION; SILENT MUTATION; TRANS SPLICING; WESTERN BLOTTING; CELL CULTURE; EPIDERMOLYSIS BULLOSA; GENE VECTOR; GENETICS; METABOLISM; MUTATION; PROCEDURES;

CELLS, CULTURED; COLLAGEN TYPE VII; EPIDERMOLYSIS BULLOSA; GENETIC THERAPY; GENETIC VECTORS; HEK293 CELLS; HUMANS; KERATINOCYTES; MUTATION; RNA, MESSENGER; TRANS-SPLICING;

EID: 84981531502     PISSN: 09697128     EISSN: 14765462     Source Type: Journal    
DOI: 10.1038/gt.2016.57     Document Type: Article

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