Detalhe da pesquisa
1.
Gene Therapy in Patients with the Crigler-Najjar Syndrome.
N Engl J Med;
389(7): 620-631, 2023 Aug 17.
Artigo
em Inglês
| MEDLINE
| ID: mdl-37585628
2.
Activation of Alternative Bilirubin Clearance Pathways Partially Reduces Hyperbilirubinemia in a Mouse Model Lacking Functional Ugt1a1 Activity.
Int J Mol Sci;
23(18)2022 Sep 14.
Artigo
em Inglês
| MEDLINE
| ID: mdl-36142606
3.
Experimental models assessing bilirubin neurotoxicity.
Pediatr Res;
87(1): 17-25, 2020 01.
Artigo
em Inglês
| MEDLINE
| ID: mdl-31493769
4.
Modulation of bilirubin neurotoxicity by the Abcb1 transporter in the Ugt1-/- lethal mouse model of neonatal hyperbilirubinemia.
Hum Mol Genet;
26(1): 145-157, 2017 01 01.
Artigo
em Inglês
| MEDLINE
| ID: mdl-28025333
5.
Attenuation of neuro-inflammation improves survival and neurodegeneration in a mouse model of severe neonatal hyperbilirubinemia.
Brain Behav Immun;
70: 166-178, 2018 05.
Artigo
em Inglês
| MEDLINE
| ID: mdl-29458193
6.
Inflammatory signature of cerebellar neurodegeneration during neonatal hyperbilirubinemia in Ugt1 -/- mouse model.
J Neuroinflammation;
14(1): 64, 2017 03 24.
Artigo
em Inglês
| MEDLINE
| ID: mdl-28340583
7.
Rescue of bilirubin-induced neonatal lethality in a mouse model of Crigler-Najjar syndrome type I by AAV9-mediated gene transfer.
FASEB J;
26(3): 1052-63, 2012 Mar.
Artigo
em Inglês
| MEDLINE
| ID: mdl-22094718
8.
CRISPR-Cas9-mediated somatic correction of a one-base deletion in the Ugt1a gene ameliorates hyperbilirubinemia in Crigler-Najjar syndrome mice.
Mol Ther Methods Clin Dev;
31: 101161, 2023 Dec 14.
Artigo
em Inglês
| MEDLINE
| ID: mdl-38094199
9.
Fludarabine increases nuclease-free AAV- and CRISPR/Cas9-mediated homologous recombination in mice.
Nat Biotechnol;
40(8): 1285-1294, 2022 08.
Artigo
em Inglês
| MEDLINE
| ID: mdl-35393561
10.
Low efficacy of recombinant SV40 in Ugt1a1-/- mice with severe inherited hyperbilirubinemia.
PLoS One;
16(4): e0250605, 2021.
Artigo
em Inglês
| MEDLINE
| ID: mdl-33891666
11.
Long-Term Effects of Biliverdin Reductase a Deficiency in Ugt1-/- Mice: Impact on Redox Status and Metabolism.
Antioxidants (Basel);
10(12)2021 Dec 20.
Artigo
em Inglês
| MEDLINE
| ID: mdl-34943131
12.
Long-term correction of ornithine transcarbamylase deficiency in Spf-Ash mice with a translationally optimized AAV vector.
Mol Ther Methods Clin Dev;
20: 169-180, 2021 Mar 12.
Artigo
em Inglês
| MEDLINE
| ID: mdl-33473356
13.
Efficacy of AAV8-hUGT1A1 with Rapamycin in neonatal, suckling, and juvenile rats to model treatment in pediatric CNs patients.
Mol Ther Methods Clin Dev;
20: 287-297, 2021 Mar 12.
Artigo
em Inglês
| MEDLINE
| ID: mdl-33511243
14.
Human liver stem cells express UGT1A1 and improve phenotype of immunocompromised Crigler Najjar syndrome type I mice.
Sci Rep;
10(1): 887, 2020 01 21.
Artigo
em Inglês
| MEDLINE
| ID: mdl-31965023
15.
A Quantitative In Vitro Potency Assay for Adeno-Associated Virus Vectors Encoding for the UGT1A1 Transgene.
Mol Ther Methods Clin Dev;
18: 250-258, 2020 Sep 11.
Artigo
em Inglês
| MEDLINE
| ID: mdl-32637454
16.
Coupling AAV-mediated promoterless gene targeting to SaCas9 nuclease to efficiently correct liver metabolic diseases.
JCI Insight;
52019 06 18.
Artigo
em Inglês
| MEDLINE
| ID: mdl-31211694
17.
Preclinical Development of an AAV8-hUGT1A1 Vector for the Treatment of Crigler-Najjar Syndrome.
Mol Ther Methods Clin Dev;
12: 157-174, 2019 Mar 15.
Artigo
em Inglês
| MEDLINE
| ID: mdl-30705921
18.
Bilirubin-Induced Oxidative Stress Leads to DNA Damage in the Cerebellum of Hyperbilirubinemic Neonatal Mice and Activates DNA Double-Strand Break Repair Pathways in Human Cells.
Oxid Med Cell Longev;
2018: 1801243, 2018.
Artigo
em Inglês
| MEDLINE
| ID: mdl-30598724
19.
Serum Bilirubin Levels and Promoter Variations in HMOX1 and UGT1A1 Genes in Patients with Fabry Disease.
Oxid Med Cell Longev;
2017: 9478946, 2017.
Artigo
em Inglês
| MEDLINE
| ID: mdl-28951772
20.
Promoterless gene targeting without nucleases rescues lethality of a Crigler-Najjar syndrome mouse model.
EMBO Mol Med;
9(10): 1346-1355, 2017 10.
Artigo
em Inglês
| MEDLINE
| ID: mdl-28751579