| scholarly article | Q13442814 |
| P50 | author | Andrés F. Muro | Q42033197 |
| P2093 | author name string | Adi Barzel | |
| Mark A Kay | |||
| Lorena Zentilin | |||
| Giulia Bortolussi | |||
| Alessandra Iaconcig | |||
| Fabiola Porro | |||
| Simone Vodret | |||
| Alessia De Caneva | |||
| P2860 | cites work | Genetic lesions of bilirubin uridine-diphosphoglucuronate glucuronosyltransferase (UGT1A1) causing Crigler-Najjar and Gilbert syndromes: correlation of genotype to phenotype | Q28140057 |
| Inherited disorders of bilirubin metabolism | Q28217356 | ||
| Treatment of the Crigler-Najjar syndrome type I with hepatocyte transplantation | Q28270302 | ||
| Genome editing with engineered zinc finger nucleases | Q28290795 | ||
| A universal system to select gene-modified hepatocytes in vivo | Q28468539 | ||
| High-frequency off-target mutagenesis induced by CRISPR-Cas nucleases in human cells | Q29616045 | ||
| Rescue of bilirubin-induced neonatal lethality in a mouse model of Crigler-Najjar syndrome type I by AAV9-mediated gene transfer | Q30515514 | ||
| rAAV-mediated tumorigenesis: still unresolved after an AAV assault | Q34310293 | ||
| Increasing the efficiency of precise genome editing with CRISPR-Cas9 by inhibition of nonhomologous end joining | Q34468164 | ||
| Long-term safety and efficacy of factor IX gene therapy in hemophilia B. | Q34783922 | ||
| Promoterless gene targeting without nucleases ameliorates haemophilia B in mice. | Q34979802 | ||
| Vector design influences hepatic genotoxicity after adeno-associated virus gene therapy. | Q35056714 | ||
| Adenovirus-associated virus vector-mediated gene transfer in hemophilia B | Q35691659 | ||
| Inverse zonation of hepatocyte transduction with AAV vectors between mice and non-human primates. | Q35720673 | ||
| Hepatic gene transfer in neonatal mice by adeno-associated virus serotype 8 vector. | Q35988663 | ||
| AAV-mediated gene targeting is significantly enhanced by transient inhibition of nonhomologous end joining or the proteasome in vivo | Q36084471 | ||
| In vivo genome editing of the albumin locus as a platform for protein replacement therapy | Q36143241 | ||
| Cellular analysis by in situ hybridization and immunoperoxidase of alpha-fetoprotein and albumin gene expression in rat liver during the perinatal period | Q36215385 | ||
| Transcriptional control of the murine albumin/alpha-fetoprotein locus during development | Q36310049 | ||
| TALENs: a widely applicable technology for targeted genome editing | Q36540717 | ||
| A dual AAV system enables the Cas9-mediated correction of a metabolic liver disease in newborn mice | Q36673956 | ||
| Enhancing transduction of the liver by adeno-associated viral vectors | Q37042394 | ||
| Robust ZFN-mediated genome editing in adult hemophilic mice. | Q37291607 | ||
| A translationally optimized AAV-UGT1A1 vector drives safe and long-lasting correction of Crigler-Najjar syndrome | Q37313599 | ||
| Inflammatory signature of cerebellar neurodegeneration during neonatal hyperbilirubinemia in Ugt1 -/- mouse model. | Q37720901 | ||
| State-of-the-art gene-based therapies: the road ahead | Q37862074 | ||
| Therapeutic in vivo gene transfer for genetic disease using AAV: progress and challenges | Q37866086 | ||
| Monogenic diseases that can be cured by liver transplantation | Q38098339 | ||
| Genome engineering with targetable nucleases | Q38194197 | ||
| CRISPR/Cas9 in Genome Editing and Beyond | Q38825624 | ||
| An inhibitor of nonhomologous end-joining abrogates double-strand break repair and impedes cancer progression | Q39222368 | ||
| Gene delivery to the juvenile mouse liver using AAV2/8 vectors | Q39991575 | ||
| Human gene targeting by viral vectors | Q41480639 | ||
| Predictability and survival in liver replantransplantation: monocentric experience | Q42186546 | ||
| Life-long correction of hyperbilirubinemia with a neonatal liver-specific AAV-mediated gene transfer in a lethal mouse model of Crigler-Najjar Syndrome | Q42203199 | ||
| Age-dependent pattern of cerebellar susceptibility to bilirubin neurotoxicity in vivo in mice | Q42238476 | ||
| Diverse IgG subclass responses to adeno-associated virus infection and vector administration | Q42667110 | ||
| Crigler-Najjar syndrome in The Netherlands: identification of four novel UGT1A1 alleles, genotype-phenotype correlation, and functional analysis of 10 missense mutants | Q43261006 | ||
| Crigler-Najjar syndrome in four of five siblings with postmortem findings in one | Q43504897 | ||
| Comparison of gene transfer to the murine liver following intraperitoneal and intraportal delivery of hepatotropic AAV pseudo-serotypes. | Q43545330 | ||
| Gilbert and Crigler Najjar syndromes: an update of the UDP-glucuronosyltransferase 1A1 (UGT1A1) gene mutation database. | Q43739103 | ||
| Evolution of indications and results of liver transplantation in Europe. A report from the European Liver Transplant Registry (ELTR). | Q43993163 | ||
| Accelerated degradation of mislocalized UDP-glucuronosyltransferase family 1 (UGT1) proteins in Gunn rat hepatocytes | Q44130829 | ||
| Long-term expression and repeated administration of AAV type 1, 2 and 5 vectors in skeletal muscle of immunocompetent adult mice | Q45858191 | ||
| AAV vector integration sites in mouse hepatocellular carcinoma. | Q45869780 | ||
| Modulation of bilirubin neurotoxicity by the Abcb1 transporter in the Ugt1-/- lethal mouse model of neonatal hyperbilirubinemia. | Q48381016 | ||
| An albumin enhancer located 10 kb upstream functions along with its promoter to direct efficient, liver-specific expression in transgenic mice. | Q52256521 | ||
| Quantitation of transplanted hepatic mass necessary to cure the Gunn rat model of hyperbilirubinemia | Q68013057 | ||
| Congenital familial nonhemolytic jaundice with kernicterus | Q73968795 | ||
| P433 | issue | 10 | |
| P921 | main subject | gene targeting | Q211083 |
| Crigler-Najjar syndrome | Q1140000 | ||
| P304 | page(s) | 1346-1355 | |
| P577 | publication date | 2017-07-27 | |
| P1433 | published in | EMBO Molecular Medicine | Q15817279 |
| P1476 | title | Promoterless gene targeting without nucleases rescues lethality of a Crigler-Najjar syndrome mouse model | |
| P478 | volume | 9 |
| Q90732125 | AAV Gene Transfer with Tandem Promoter Design Prevents Anti-transgene Immunity and Provides Persistent Efficacy in Neonate Pompe Mice |
| Q92845907 | Coupling AAV-mediated promoterless gene targeting to SaCas9 nuclease to efficiently correct liver metabolic diseases |
| Q47554736 | Emerging Issues in AAV-Mediated In Vivo Gene Therapy |
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