Correcting CGD safely, iPSo facto

Blood

Volumn 117, Issue 21, 2011, Pages 5554-5556

  Notarangelo, Luigi D ^a  

^a HARVARD MEDICAL SCHOOL   (United States)

Author keywords

[No Author keywords available]

Indexed keywords

BETA GLOBIN; GLYCOPROTEIN GP 91; NUCLEASE; REDUCED NICOTINAMIDE ADENINE DINUCLEOTIDE PHOSPHATE OXIDASE; RETROVIRUS VECTOR; UNCLASSIFIED DRUG; ZINC FINGER NUCLEASE;

BONE MARROW TRANSPLANTATION; CHRONIC GRANULOMATOUS DISEASE; ENZYME ACTIVITY; GENE INSERTION; GENE TARGETING; GENE THERAPY; GENE TRANSFER; HEMATOPOIETIC CELL; HOMOLOGOUS RECOMBINATION; HUMAN; IN VITRO STUDY; NEUTROPHIL; NONHUMAN; NOTE; PHENOTYPE; PLURIPOTENT STEM CELL; PRIORITY JOURNAL; PROTEIN EXPRESSION; X CHROMOSOME LINKED DISORDER; X LINKED CHRONIC GRANULOMATOUS DISEASE;

EID: 79957596888     PISSN: 00064971     EISSN: 15280020     Source Type: Journal    
DOI: 10.1182/blood-2011-04-344119     Document Type: Note

References (10)

1. Park IH, Arora N, Huo H, et al. Disease-specific induced pluripotent stem cells. Cell. 2008;134(5):877-886.
2. Zou J, Sweeney CL, Chou BK, et al. Oxidase deficient neutrophils from X-linked chronic granulomatous disease iPS cells: functional correction by zinc finger nuclease mediated safe harbor targeting. Blood. 2011;117(21):5561-5572.
3. Kuhns DB, Alvord WG, Heller T, et al. Residual NADPH oxidase and survival in chronic granulomatous disease.N Engl J Med. 2010;363(27):2600-2610.
4. Smith JR, Maguire S, Davis LA, et al. Robust, persistent transgene expression in human embryonic stem cells is achieved with AAVS1-targeted integration. Stem Cells. 2008;26(2):496-504. (Pubitemid 351413610)
5. Mukherjee S, Santilli G, Blundell MP, et al. Generation of functional neutrophils from a mouse model of X-linked chronic granulomatous disorder using induced pluripotent stem cells. PLoS ONE. 2011;6(3):e17565.
6. Pessach IM, Ordovas-Montanes J, Zhang SY, et al. Induced pluripotent stem cells: A novel frontier in the study of human primary immunodeficiencies [published online ahead of print December 23, 2010]. J Allergy Clin Immunol. doi:10.1016/j.jaci.2010.11.008.
7. Stein S, Ott MG, Schultze-Strasser S, et al. Genomic instability and myelodysplasia with monosomy 7 consequent to EVI1 activation after gene therapy for chronic granulomatous disease. Nat Med. 2010;16(2):198-204.
8. Papapetrou EP, Lee G, Malani N, et al. Genomic safe harbors permit high beta-globin transgene expression in thalassemia induced pluripotent stem cells. Nat Biotechnol. 2011;29(1):73-78.
9. Urnov FD, Rebar EJ, Holmes MC, et al. Genome editing with engineered zinc finger nucleases. Nat Rev Genet. 2010;11(9):636-646.
10. Silva G, Poirot L, Galetto R, et al. Meganucleases and other tools for targeted genome engineering: perspectives and challenges for gene therapy. Curr Gene Ther. 2011;11(1):11-27.