Durable correction of inherited metabolic liver disorders requires preventing transgene off-targeting from gene therapy vectors: The value of MicroRNAs

Gastroenterology

Volumn 139, Issue 3, 2010, Pages 726-729

  Gonzalez Aseguinolaza, Gloria ^a   Prieto, Jesus ^a  

^a UNIVERSITY OF NAVARRA   (Spain)

Author keywords

[No Author keywords available]

Indexed keywords

BILIRUBIN; GLUCURONOSYLTRANSFERASE 1A1; LENTIVIRUS VECTOR; MICRORNA; MICRORNA 142; UNCLASSIFIED DRUG; ANTIBODY; BIOLOGICAL MARKER; GLUCURONOSYLTRANSFERASE; MESSENGER RNA; UGT1A1 ENZYME;

AUTOSOMAL RECESSIVE DISORDER; BILIRUBIN BLOOD LEVEL; CRIGGLER NAJJAR SYNDROME TYPE I; EDITORIAL; GENE THERAPY; HYPERBILIRUBINEMIA; NONHUMAN; PHOTOTHERAPY; PRIORITY JOURNAL; TRANSGENE; ANIMAL; ANTIGEN PRESENTING CELL; BIOSYNTHESIS; BLOOD; CRIGLER-NAJJAR SYNDROME; DISEASE MODEL; ENZYMOLOGY; GENE VECTOR; GENETICS; GUNN RAT; HUMAN; IMMUNOLOGY; LENTIVIRINAE; LIVER; METABOLISM; PROCEDURES; RAT; RNA STABILITY;

ANIMALS; ANTIBODIES; ANTIGEN-PRESENTING CELLS; BILIRUBIN; BIOLOGICAL MARKERS; CRIGLER-NAJJAR SYNDROME; DISEASE MODELS, ANIMAL; GENETIC THERAPY; GENETIC VECTORS; GLUCURONOSYLTRANSFERASE; HUMANS; LENTIVIRUS; LIVER; MICRORNAS; RATS; RATS, GUNN; RNA STABILITY; RNA, MESSENGER;

EID: 77956160758     PISSN: 00165085     EISSN: None     Source Type: Journal    
DOI: 10.1053/j.gastro.2010.07.016     Document Type: Editorial

References (18)

1. A. Kadakol, S.S. Ghosh, and B.S. Sappal Genetic lesions of bilirubin uridine-diphosphoglucuronate glucuronosyltransferase (UGT1A1) causing Crigler-Najjar and Gilbert syndromes: correlation of genotype to phenotype Hum Mutat 16 2000 297 306
2. T. Iyanagi, Y. Emi, and S. Ikushiro Biochemical and molecular aspects of genetic disorders of bilirubin metabolism Biochim Biophys Acta 1407 1998 173 184
3. P.L. Jansen Diagnosis and management of Crigler-Najjar syndrome Eur J Pediatr 158 suppl 2 1999 S89 S94
4. P.S. Miranda, and P.J. Bosma Towards liver-directed gene therapy for Crigler-Najjar syndrome Curr Gene Ther 9 2009 72 82
5. J.R. Chowdhury, R. Kondapalli, and N.R. Chowdhury Gunn rat: a model for inherited deficiency of bilirubin glucuronidation Adv Vet Sci Comp Med 37 1993 149 173
6. M. Bellodi-Privato, D. Aubert, and V. Pichard Successful gene therapy of the Gunn rat by in vivo neonatal hepatic gene transfer using murine oncoretroviral vectors Hepatology 42 2005 431 438
7. J. Seppen, C. Bakker, and B. de Jong Adeno-associated virus vector serotypes mediate sustained correction of bilirubin UDP glucuronosyltransferase deficiency in rats Mol Ther 13 2006 1085 1092
8. P. van der Wegen, R. Louwen, and A.M. Imam Successful treatment of UGT1A1 deficiency in a rat model of Crigler-Najjar disease by intravenous administration of a liver-specific lentiviral vector Mol Ther 13 2006 374 381
9. T.H. Nguyen, D. Aubert, and M. Bellodi-Privato Critical assessment of lifelong phenotype correction in hyperbilirubinemic Gunn rats after retroviral mediated gene transfer Gene Ther 14 2007 1270 1277
10. R.M. Shull, X. Lu, and M.F. McEntee Myoblast gene therapy in canine mucopolysaccharidosis I: abrogation by an immune response to alpha-L-iduronidase Hum Gene Ther 7 1996 1595 1603
11. R. Shull, X. Lu, and I. Dub Humoral immune response limits gene therapy in canine MPS I Blood 88 1996 377 379
12. B.D. Brown, M.A. Venneri, and A. Zingale Endogenous microRNA regulation suppresses transgene expression in hematopoietic lineages and enables stable gene transfer Nat Med 12 2006 585 591
13. C.Z. Chen, L. Li, and H.F. Lodish MicroRNAs modulate hematopoietic lineage differentiation Science 303 2004 83 86
14. F. Schmitt, S. Remy, and A. Dariel Lentiviral vectors that express UGT1A1 in liver and contain miR-142 target sequences normalize hyperbilirubinemia in Gunn rats Gastroenterology 139 2010 999 1007
15. A. Annoni, B.D. Brown, and A. Cantore In vivo delivery of a microRNA-regulated transgene induces antigen-specific regulatory T cells and promotes immunologic tolerance Blood 114 2009 5152 5161
16. A. Fischer, S. Hacein-Bey-Abina, and M. Cavazzana-Calvo 20 years of gene therapy for SCID Nat Immunol 11 2010 457 460
17. F. Mingozzi, M.V. Maus, and D.J. Hui CD8(+) T-cell responses to adeno-associated virus capsid in humans Nat Med 13 2007 419 422
18. J.D. Finn, D. Hui, and H.D. Downey Proteasome inhibitors decrease AAV2 capsid derived peptide epitope presentation on MHC class I following transduction Mol Ther 18 2010 135 142