Reprogramming metabolic pathways in vivo with CRISPR/Cas9 genome editing to treat hereditary tyrosinaemia

Nature Communications

Volumn 7, Issue , 2016, Pages

  Pankowicz, Francis P ^a,b   Barzi, Mercedes ^a,b   Legras, Xavier ^a,b   Hubert, Leroy ^b   Mi, Tian ^c   Tomolonis, Julie A ^b   Ravishankar, Milan ^a,b   Sun, Qin ^b   Yang, Diane ^a,b,d   Borowiak, Malgorzata ^a,b,d   Sumazin, Pavel ^b,c   Elsea, Sarah H ^b   Bissig Choisat, Beatrice ^a,b   Bissig, Karl Dimiter ^a,b  

^a CENTER FOR CELL AND GENE THERAPY   (United States)

^b BAYLOR COLLEGE OF MEDICINE   (United States)

^c TEXAS CHILDREN S HOSPITAL   (United States)

^d McNair Medical Institute   (United States)

Author keywords

[No Author keywords available]

Indexed keywords

4 HYDROXYPHENYLPYRUVATE DIOXYGENASE; AMINO ACID; CYCLOHEXANONE DERIVATIVE; ENZYME INHIBITOR; FUMARYLACETOACETASE; HPD PROTEIN, MOUSE; HYDROLASE; NITISINONE; NITROBENZOIC ACID DERIVATIVE; OXIDOREDUCTASE;

CATABOLISM; DRUG; GENETIC ANALYSIS; GENOME; METABOLISM; METABOLITE; PHENOTYPE; PHYSIOLOGY; RODENT;

ANIMAL CELL; ANIMAL EXPERIMENT; ANIMAL MODEL; ANIMAL TISSUE; ARTICLE; CATABOLISM; CELL PROLIFERATION; CONTROLLED STUDY; CRISPR CAS SYSTEM; GENE DELETION; GENOME; IN VIVO STUDY; LIVER CELL; METABOLISM; MOUSE; NONHUMAN; NUCLEAR REPROGRAMMING; TYROSINEMIA; ANIMAL; ANTAGONISTS AND INHIBITORS; CELL LINE; CYTOLOGY; DISEASE MODEL; EXON; GENE EDITING; GENE THERAPY; GENETICS; HUMAN; INBRED MOUSE STRAIN; KNOCKOUT MOUSE; LIVER; PATHOLOGY; PHENOTYPE; PROCEDURES;

MUS;

ANIMALS; CELL LINE; CELL PROLIFERATION; CRISPR-CAS SYSTEMS; CYCLOHEXANONES; DISEASE MODELS, ANIMAL; ENZYME INHIBITORS; EXONS; GENE EDITING; GENETIC THERAPY; HEPATOCYTES; HUMANS; HYDROLASES; LIVER; METABOLIC NETWORKS AND PATHWAYS; MICE; MICE, INBRED STRAINS; MICE, KNOCKOUT; NITROBENZOATES; OXIDOREDUCTASES; PHENOTYPE; TYROSINEMIAS;

EID: 84984865278     PISSN: None     EISSN: 20411723     Source Type: Journal    
DOI: 10.1038/ncomms12642     Document Type: Article

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