Generation of a High Number of Healthy Erythroid Cells from Gene-Edited Pyruvate Kinase Deficiency Patient-Specific Induced Pluripotent Stem Cells

Stem Cell Reports

Volumn 5, Issue 6, 2015, Pages 1053-1066

  Garate, Zita ^a,b,c   Quintana Bustamante, Oscar ^a,b   Crane, Ana M ^c   Olivier, Emmanuel ^d   Poirot, Laurent ^e   Galetto, Roman ^e   Kosinski, Penelope ^f   Hill, Collin ^f   Kung, Charles ^f   Agirre, Xabi ^g   Orman, Israel ^a,b   Cerrato, Laura ^a,b   Alberquilla, Omaira ^a,b   Rodriguez Fornes, Fatima ^a,b   Fusaki, Noemi ^h   Garcia Sanchez, Felix ⁱ   Maia, Tabita M ^j   Ribeiro, Maria L ^j   Sevilla, Julian ^k   Prosper, Felipe ^g   Jin, Shengfang ^f   Mountford, Joanne ^d   Guenechea, Guillermo ^a,b   Gouble, Agnes ^e   Bueren, Juan A ^a,b   Davis, Brian R ^c   Segovia, Jose C ^a,b   more..

^a CIEMAT   (Spain)

^b INSTITUTO DE INVESTIGACIÓN SANITARIA DEL HOSPITAL CLÍNICO SAN CARLOS IDISSC   (Spain)

^c University Health Science Center   (United States)

^d UNIVERSITY OF GLASGOW   (United Kingdom)

^e Arvid Wallgrens Backe 20   (France)

^f AGIOS PHARMACEUTICALS   (United States)

^g UNIVERSITY CLINIC OF NAVARRA   (Spain)

^h KEIO UNIVERSITY   (Japan)

ⁱ Centro de Transfusion de Madrid   (Spain)

^j CENTRO HOSPITALAR E UNIVERSITÁRIO DE COIMBRA   (Portugal)

^k HOSPITAL INFANTIL UNIVERSITARIO NIÑO JESÚS   (Spain)

more..

Author keywords

[No Author keywords available]

Indexed keywords

COMPLEMENTARY DNA; PYRUVATE KINASE; VIRUS VECTOR;

ALLELE; ARTICLE; CELL COUNT; CODON; ENERGY; ENZYME DEFICIENCY; ERYTHROID CELL; GENE; GENETIC STABILITY; HEMOLYTIC ANEMIA; HOMOLOGOUS RECOMBINATION; HUMAN; HUMAN CELL; INTRON; METABOLIC DISORDER; METABOLISM; PERIPHERAL BLOOD MONONUCLEAR CELL; PLURIPOTENT STEM CELL; PRIORITY JOURNAL; SINGLE NUCLEOTIDE POLYMORPHISM; ANEMIA, HEMOLYTIC, CONGENITAL NONSPHEROCYTIC; CYTOLOGY; DEFICIENCY; GENE TARGETING; GENE THERAPY; GENETIC RECOMBINATION; GENETICS; INDUCED PLURIPOTENT STEM CELL; MONONUCLEAR CELL; NUCLEOTIDE SEQUENCE; PYRUVATE METABOLISM, INBORN ERRORS;

ALLELES; ANEMIA, HEMOLYTIC, CONGENITAL NONSPHEROCYTIC; BASE SEQUENCE; CELL COUNT; DNA, COMPLEMENTARY; ERYTHROID CELLS; GENE TARGETING; GENETIC THERAPY; HUMANS; INDUCED PLURIPOTENT STEM CELLS; LEUKOCYTES, MONONUCLEAR; PYRUVATE KINASE; PYRUVATE METABOLISM, INBORN ERRORS; RECOMBINATION, GENETIC;

EID: 84949537443     PISSN: 22136711     EISSN: None     Source Type: Journal    
DOI: 10.1016/j.stemcr.2015.10.002     Document Type: Article

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