In vivo genome editing of the albumin locus as a platform for protein replacement therapy

Blood

Volumn 126, Issue 15, 2015, Pages 1777-1784

  Sharma, Rajiv ^a   Anguela, Xavier M ^a,b   Doyon, Yannick ^c   Wechsler, Thomas ^c   DeKelver, Russell C ^c   Sproul, Scott ^c   Paschon, David E ^c   Miller, Jeffrey C ^c   Davidson, Robert J ^a   Shivak, David ^c   Zhou, Shangzhen ^a   Rieders, Julianne ^a   Gregory, Philip D ^c   Holmes, Michael C ^c   Rebar, Edward J ^c   High, Katherine A ^a,b  

^a CHILDREN S HOSPITAL OF PHILADELPHIA   (United States)

^b HOWARD HUGHES MEDICAL INSTITUTE   (United States)

^c SANGAMO BIOSCIENCES INC   (United States)

Author keywords

[No Author keywords available]

Indexed keywords

ALBUMIN; LYSOSOME ENZYME; NUCLEASE; PARVOVIRUS VECTOR; ZINC FINGER NUCLEASE; ALBUMINOID; BLOOD CLOTTING FACTOR 8; BLOOD CLOTTING FACTOR 9; ENDONUCLEASE; F8 PROTEIN, HUMAN; MESSENGER RNA; ZINC FINGER PROTEIN;

ANIMAL EXPERIMENT; ANIMAL MODEL; ANIMAL TISSUE; ARTICLE; CONTROLLED STUDY; ENZYME ACTIVITY; FABRY DISEASE; GAUCHER DISEASE; GENE LOCUS; GENE TARGETING; GENOME; HEMOPHILIA A; HEMOPHILIA B; HUMAN; HUMAN CELL; HUNTER SYNDROME; HURLER SYNDROME; MOUSE; NONHUMAN; PRIORITY JOURNAL; PROTEIN REPLACEMENT THERAPY; SUBSTITUTION THERAPY; TRANSGENE; ADMINISTRATION AND DOSAGE; ANIMAL; C57BL MOUSE; DEPENDOPARVOVIRUS; ENZYME REPLACEMENT; ENZYMOLOGY; GENE THERAPY; GENE VECTOR; GENETICS; HIGH THROUGHPUT SEQUENCING; LIVER; LYSOSOME; METABOLISM; MUCOPOLYSACCHARIDOSIS I; MUCOPOLYSACCHARIDOSIS II; PHYSIOLOGY; PROMOTER REGION; REAL TIME POLYMERASE CHAIN REACTION; REVERSE TRANSCRIPTION POLYMERASE CHAIN REACTION; RNA EDITING;

ALBUMINS; ANIMALS; DEPENDOVIRUS; ENDONUCLEASES; ENZYME REPLACEMENT THERAPY; FABRY DISEASE; FACTOR IX; FACTOR VIII; GAUCHER DISEASE; GENETIC THERAPY; GENETIC VECTORS; GENOME; HEMOPHILIA A; HEMOPHILIA B; HIGH-THROUGHPUT NUCLEOTIDE SEQUENCING; HUMANS; LIVER; LYSOSOMES; MICE; MICE, INBRED C57BL; MUCOPOLYSACCHARIDOSIS I; MUCOPOLYSACCHARIDOSIS II; PROMOTER REGIONS, GENETIC; REAL-TIME POLYMERASE CHAIN REACTION; REVERSE TRANSCRIPTASE POLYMERASE CHAIN REACTION; RNA EDITING; RNA, MESSENGER; TRANSGENES; ZINC FINGERS;

EID: 84943601842     PISSN: 00064971     EISSN: 15280020     Source Type: Journal    
DOI: 10.1182/blood-2014-12-615492     Document Type: Article

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