Therapeutic Genome Editing With CRISPR/Cas9 in a Humanized Mouse Model Ameliorates α1-antitrypsin Deficiency Phenotype

EBioMedicine

Volumn 29, Issue , 2018, Pages 104-111

  Bjursell, Mikael ^a   Porritt, Michelle J ^a   Ericson, Elke ^a   Taheri Ghahfarokhi, Amir ^a   Clausen, Maryam ^a   Magnusson, Lisa ^a   Admyre, Therese ^a   Nitsch, Roberto ^a   Mayr, Lorenz ^a   Aasehaug, Leif ^a   Seeliger, Frank ^a   Maresca, Marcello ^a   Bohlooly Y, Mohammad ^a   Wiseman, John ^a  

^a ASTRAZENECA R AND D   (Sweden)

Author keywords

CRISPR Cas9 gene editing; Liver fibrosis; Protein aggregation; 1 antitrypsin deficiency

Indexed keywords

ALANINE AMINOTRANSFERASE; ASPARTATE AMINOTRANSFERASE; ALPHA 1 ANTITRYPSIN;

ALPHA 1 ANTITRYPSIN DEFICIENCY; ANIMAL EXPERIMENT; ANIMAL MODEL; ANIMAL TISSUE; ARTICLE; CELL PROLIFERATION; CONTROLLED STUDY; CRISPR-CAS9 SYSTEM; DNA MODIFICATION; ENZYME LINKED IMMUNOSORBENT ASSAY; FEMALE; GENE DOSAGE; GENE EDITING; GENE EXPRESSION; GENE FREQUENCY; GENETIC TRANSFECTION; IMMUNOHISTOCHEMISTRY; LIVER CELL; LIVER FIBROSIS; LIVER HISTOLOGY; LIVER INJURY; MOUSE; NEXT GENERATION SEQUENCING; NONHUMAN; PHENOTYPE; PLASMID; POINT MUTATION; PRIORITY JOURNAL; PROTEIN AGGREGATION; PROTEIN EXPRESSION; REAL TIME POLYMERASE CHAIN REACTION; SINGLE NUCLEOTIDE POLYMORPHISM; ADENOVIRIDAE; ANIMAL; BLOOD; CRISPR CAS SYSTEM; DISEASE MODEL; GENE VECTOR; GENETIC TRANSDUCTION; GENETICS; HUMAN; METABOLISM; PATHOLOGY; TRANSGENE; TRANSGENIC MOUSE;

ADENOVIRIDAE; ALPHA 1-ANTITRYPSIN; ALPHA 1-ANTITRYPSIN DEFICIENCY; ANIMALS; CELL PROLIFERATION; CRISPR-CAS SYSTEMS; DISEASE MODELS, ANIMAL; GENE EDITING; GENE EXPRESSION; GENETIC VECTORS; HUMANS; MICE; MICE, TRANSGENIC; PHENOTYPE; TRANSDUCTION, GENETIC; TRANSGENES;

EID: 85042616653     PISSN: None     EISSN: 23523964     Source Type: Journal    
DOI: 10.1016/j.ebiom.2018.02.015     Document Type: Article

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