CRISPR/Cas9-mediated somatic correction of a novel coagulator factor IX gene mutation ameliorates hemophilia in mouse

EMBO Molecular Medicine

Volumn 8, Issue 5, 2016, Pages 477-488

  Guan, Yuting ^a   Ma, Yanlin ^b,c   Li, Qi ^b   Sun, Zhenliang ^c   Ma, Lie ^a   Wu, Lijuan ^a   Wang, Liren ^a   Zeng, Li ^a   Shao, Yanjiao ^a   Chen, Yuting ^a   Ma, Ning ^b   Lu, Wenqing ^a   Hu, Kewen ^a   Han, Honghui ^d   Yu, Yanhong ^c   Huang, Yuanhua ^b   Liu, Mingyao ^a,e   Li, Dali ^a  

^a EAST CHINA NORMAL UNIVERSITY   (China)

^b HAINAN MEDICAL UNIVERSITY   (China)

^c SOUTHERN MEDICAL UNIVERSITY   (China)

^d Bioray Laboratories Inc   (China)

^e TEXAS A AND M HEALTH SCIENCE CENTER   (United States)

Author keywords

Gene therapy; Genome editing; Hemophilia B; Hemostasis; Monogenetic disease

Indexed keywords

ADENOVIRUS VECTOR; BLOOD CLOTTING FACTOR 9; CAS9 PROTEIN; CELLULAR APOPTOSIS SUSCEPTIBILITY PROTEIN; DNA; OLIGODEOXYNUCLEOTIDE; UNCLASSIFIED DRUG;

ADULT; ANIMAL CELL; ANIMAL EXPERIMENT; ANIMAL MODEL; ANIMAL TISSUE; ARTICLE; CHILD; CLINICAL ARTICLE; CONTROLLED STUDY; CRISPR CAS SYSTEM; DISEASE SEVERITY; DNA MODIFICATION; F9 GENE; GENE CONSTRUCT; GENE MUTATION; GENE TARGETING; HEMOPHILIA B; HEMOSTASIS; HUMAN; LIVER TOXICITY; MALE; MOUSE; NONHUMAN; PRIORITY JOURNAL; SCHOOL CHILD; SOMATIC GENE THERAPY; VIRAL GENE DELIVERY SYSTEM; ADENOVIRIDAE; ANIMAL; DISEASE MODEL; GENE EDITING; GENE THERAPY; GENE VECTOR; GENETIC RECOMBINATION; GENETICS; MISSENSE MUTATION; PATHOLOGY; PROCEDURES; TREATMENT OUTCOME;

ADENOVIRIDAE; ANIMALS; CHILD; CRISPR-CAS SYSTEMS; DISEASE MODELS, ANIMAL; FACTOR IX; GENE EDITING; GENETIC THERAPY; GENETIC VECTORS; HEMOPHILIA B; HUMANS; MALE; MICE; MUTATION, MISSENSE; RECOMBINATION, GENETIC; TREATMENT OUTCOME;

EID: 84960422519     PISSN: 17574676     EISSN: 17574684     Source Type: Journal    
DOI: 10.15252/emmm.201506039     Document Type: Article

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