William Querbes
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Explore the profile of William Querbes including associated specialties, affiliations and a list of published articles.
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31
Citations
3744
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Recent Articles
1.
Cunningham S, Zakas P, Sasaki N, van Dijk E, Zhu E, Fu Y, et al.
J Gene Med
. 2024 Aug;
26(8):e3726.
PMID: 39160647
Background: Conventional adeno-associated viral (AAV) vectors, while highly effective in quiescent cells such as hepatocytes in the adult liver, confer less durable transgene expression in proliferating cells owing to episome...
2.
Zakas P, Cunningham S, Doherty A, van Dijk E, Ibraheim R, Yu S, et al.
Mol Ther
. 2024 Jul;
32(10):3356-3371.
PMID: 38981468
Recombinant adeno-associated virus (rAAV) vector gene delivery systems have demonstrated great promise in clinical trials but continue to face durability and dose-related challenges. Unlike rAAV gene therapy, integrating gene addition...
3.
Ochoa-Sanchez R, Oliveira M, Tremblay M, Petrazzo G, Pant A, Bosoi C, et al.
Liver Int
. 2021 Feb;
41(5):1020-1032.
PMID: 33548108
Methods: One-week post-surgery, BDLs received non-modified EcN (EcN), S-ARG, S-ARG + BUT (3x10 CFU/day) or vehicle until sacrifice at 3 or 5 weeks. Plasma (ammonia/pro-inflammatory/liver function), liver fibrosis (hydroxyproline), liver...
4.
Gouya L, Ventura P, Balwani M, Bissell D, Rees D, Stolzel U, et al.
Hepatology
. 2019 Sep;
71(5):1546-1558.
PMID: 31512765
Background And Aims: Acute hepatic porphyria comprises a group of rare genetic diseases caused by mutations in genes involved in heme biosynthesis. Patients can experience acute neurovisceral attacks, debilitating chronic...
5.
Sardh E, Harper P, Balwani M, Stein P, Rees D, Bissell D, et al.
N Engl J Med
. 2019 Feb;
380(6):549-558.
PMID: 30726693
Background: Induction of delta aminolevulinic acid synthase 1 ( ALAS1) gene expression and accumulation of neurotoxic intermediates result in neurovisceral attacks and disease manifestations in patients with acute intermittent porphyria,...
6.
Simon A, Pompilus F, Querbes W, Wei A, Strzok S, Penz C, et al.
Patient
. 2018 Jun;
11(5):527-537.
PMID: 29915990
Objective: Acute intermittent porphyria is a rare metabolic disorder that affects heme synthesis. Patients with acute intermittent porphyria may experience acute debilitating neurovisceral attacks that require frequent hospitalizations and negatively...
7.
Xu Y, Redon V, Yu H, Querbes W, Pirruccello J, Liebow A, et al.
Atherosclerosis
. 2017 Nov;
268:196-206.
PMID: 29183623
Background And Aims: Angiopoietin-like 3 (ANGPTL3) has emerged as a key regulator of lipoprotein metabolism in humans. Homozygous loss of ANGPTL3 function causes familial combined hypolipidemia characterized by low plasma...
8.
Liebow A, Li X, Racie T, Hettinger J, Bettencourt B, Najafian N, et al.
J Am Soc Nephrol
. 2016 Jul;
28(2):494-503.
PMID: 27432743
Primary hyperoxaluria type 1 (PH1), an inherited rare disease of glyoxylate metabolism, arises from mutations in the enzyme alanine-glyoxylate aminotransferase. The resulting deficiency in this enzyme leads to abnormally high...
9.
Li X, Knight J, Fargue S, Buchalski B, Guan Z, Inscho E, et al.
Biochim Biophys Acta
. 2015 Dec;
1862(2):233-9.
PMID: 26655602
Excessive endogenous oxalate synthesis can result in calcium oxalate kidney stone formation and renal failure. Hydroxyproline catabolism in the liver and kidney contributes to endogenous oxalate production in mammals. To...
10.
Chan A, Liebow A, Yasuda M, Gan L, Racie T, Maier M, et al.
Mol Ther Nucleic Acids
. 2015 Nov;
4:e263.
PMID: 26528940
The acute hepatic porphyrias are caused by inherited enzymatic deficiencies in the heme biosynthesis pathway. Induction of the first enzyme 5-aminolevulinic acid synthase 1 (ALAS1) by triggers such as fasting...