Detalhe da pesquisa
1.
Exploring current and emerging therapies for porphyrias.
Liver Int
; 2024 May 30.
Artigo
em Inglês
| MEDLINE | ID: mdl-38813953
2.
Nutritional Interventions with Bacillus coagulans Improved Glucose Metabolism and Hyperinsulinemia in Mice with Acute Intermittent Porphyria.
Int J Mol Sci
; 24(15)2023 07 26.
Artigo
em Inglês
| MEDLINE | ID: mdl-37569315
3.
Brain ventricular enlargement in human and murine acute intermittent porphyria.
Hum Mol Genet
; 29(19): 3211-3223, 2020 11 25.
Artigo
em Inglês
| MEDLINE | ID: mdl-32916704
4.
Understanding Carbohydrate Metabolism and Insulin Resistance in Acute Intermittent Porphyria.
Int J Mol Sci
; 24(1)2022 Dec 20.
Artigo
em Inglês
| MEDLINE | ID: mdl-36613492
5.
Bioengineered PBGD variant improves the therapeutic index of gene therapy vectors for acute intermittent porphyria.
Hum Mol Genet
; 27(21): 3688-3696, 2018 11 01.
Artigo
em Inglês
| MEDLINE | ID: mdl-30085095
6.
Messenger RNA therapy for rare genetic metabolic diseases.
Gut
; 68(7): 1323-1330, 2019 07.
Artigo
em Inglês
| MEDLINE | ID: mdl-30796097
7.
Non-parenchymal TREM-2 protects the liver from immune-mediated hepatocellular damage.
Gut
; 68(3): 533-546, 2019 03.
Artigo
em Inglês
| MEDLINE | ID: mdl-29374630
8.
Current and innovative emerging therapies for porphyrias with hepatic involvement.
J Hepatol
; 71(2): 422-433, 2019 08.
Artigo
em Inglês
| MEDLINE | ID: mdl-31102718
9.
Computational disease model of phenobarbital-induced acute attacks in an acute intermittent porphyria mouse model.
Mol Genet Metab
; 128(3): 367-375, 2019 11.
Artigo
em Inglês
| MEDLINE | ID: mdl-30639045
10.
Glucose metabolism during fasting is altered in experimental porphobilinogen deaminase deficiency.
Hum Mol Genet
; 25(7): 1318-27, 2016 Apr 01.
Artigo
em Inglês
| MEDLINE | ID: mdl-26908609
11.
Hydroxymethylbilane synthase (aka porphobilinogen deaminase): A novel metabolic tumor suppressor gene in hepatocellular carcinoma.
J Hepatol
; 77(4): 912-914, 2022 10.
Artigo
em Inglês
| MEDLINE | ID: mdl-35798130
12.
Phase I open label liver-directed gene therapy clinical trial for acute intermittent porphyria.
J Hepatol
; 65(4): 776-783, 2016 10.
Artigo
em Inglês
| MEDLINE | ID: mdl-27212246
13.
Emerging therapies for acute intermittent porphyria.
Expert Rev Mol Med
; 18: e17, 2016 11 02.
Artigo
em Inglês
| MEDLINE | ID: mdl-27804912
14.
Acute intermittent porphyria, givosiran, and homocysteine.
J Inherit Metab Dis
; 44(4): 790-791, 2021 07.
Artigo
em Inglês
| MEDLINE | ID: mdl-34145602
15.
Helper-dependent adenoviral liver gene therapy protects against induced attacks and corrects protein folding stress in acute intermittent porphyria mice.
Hum Mol Genet
; 22(14): 2929-40, 2013 Jul 15.
Artigo
em Inglês
| MEDLINE | ID: mdl-23562909
16.
Transient and intensive pharmacological immunosuppression fails to improve AAV-based liver gene transfer in non-human primates.
J Transl Med
; 10: 122, 2012 Jun 15.
Artigo
em Inglês
| MEDLINE | ID: mdl-22704060
17.
Sustained enzymatic correction by rAAV-mediated liver gene therapy protects against induced motor neuropathy in acute porphyria mice.
Mol Ther
; 19(2): 243-50, 2011 Feb.
Artigo
em Inglês
| MEDLINE | ID: mdl-20877347
18.
Mechanistic modelling of enzyme-restoration effects of new recombinant liver-targeted proteins in acute intermittent porphyria.
Br J Pharmacol
; 179(14): 3815-3830, 2022 07.
Artigo
em Inglês
| MEDLINE | ID: mdl-35170015
19.
Messenger RNA as a personalized therapy: The moment of truth for rare metabolic diseases.
Int Rev Cell Mol Biol
; 372: 55-96, 2022.
Artigo
em Inglês
| MEDLINE | ID: mdl-36064267
20.
Recombinant porphobilinogen deaminase targeted to the liver corrects enzymopenia in a mouse model of acute intermittent porphyria.
Sci Transl Med
; 14(627): eabc0700, 2022 01 12.
Artigo
em Inglês
| MEDLINE | ID: mdl-35020410