Fatal propionic acidemia in mice lacking propionyl-CoA carboxylase and its rescue by postnatal, liver-specific supplementation via a transgene
about
Antisense mediated splicing modulation for inherited metabolic diseases: challenges for deliveryStructure and function of biotin-dependent carboxylasesA knock-out mouse model for methylmalonic aciduria resulting in neonatal lethalityIdentification of novel molecular candidates for fatty liver in the hyperlipidemic mouse model, HcB19.Effects of adeno-associated virus serotype and tissue-specific expression on circulating biomarkers of propionic acidemia.Marginal maternal biotin deficiency in CD-1 mice reduces fetal mass of biotin-dependent carboxylasesA sensitive assay system to test antisense oligonucleotides for splice suppression therapy in the mouse liver.Adeno-associated virus serotype 8 gene transfer rescues a neonatal lethal murine model of propionic acidemia.Long-term sex-biased correction of circulating propionic acidemia disease markers by adeno-associated virus vectors.Generation of a hypomorphic model of propionic acidemia amenable to gene therapy testing.Adenovirus-mediated gene delivery rescues a neonatal lethal murine model of mut(0) methylmalonic acidemia.Short-chain fatty acid-mediated effects on erythropoiesis in primary definitive erythroid cells.Chronic kidney disease in an adult with propionic acidemia.Advances and challenges in the treatment of branched-chain amino/keto acid metabolic defects.Propionate as a health-promoting microbial metabolite in the human gut.Readthrough strategies for therapeutic suppression of nonsense mutations in inherited metabolic disease.Interrelations between 3-hydroxypropionate and propionate metabolism in rat liver: Relevance to disorders of propionyl-CoA metabolism.Short-term rescue of neonatal lethality in a mouse model of propionic acidemia by gene therapy.Secondary mitochondrial dysfunction in propionic aciduria: a pathogenic role for endogenous mitochondrial toxins.Pseudoexon exclusion by antisense therapy in 6-pyruvoyl-tetrahydropterin synthase deficiency.Propionyl-CoA carboxylase - A review.Feasibility of nonsense mutation readthrough as a novel therapeutical approach in propionic acidemia.
P2860
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P2860
Fatal propionic acidemia in mice lacking propionyl-CoA carboxylase and its rescue by postnatal, liver-specific supplementation via a transgene
description
2001 թուականի Սեպտեմբերին հրատարակուած գիտական յօդուած
@hyw
2001 թվականի սեպտեմբերին հրատարակված գիտական հոդված
@hy
article publié dans la revue scientifique Journal of Biological Chemistry
@fr
artículu científicu espublizáu en 2001
@ast
im September 2001 veröffentlichter wissenschaftlicher Artikel
@de
scientific journal article
@en
vedecký článok (publikovaný 2001/09/21)
@sk
vědecký článek publikovaný v roce 2001
@cs
wetenschappelijk artikel (gepubliceerd op 2001/09/21)
@nl
наукова стаття, опублікована у вересні 2001
@uk
name
Fatal propionic acidemia in mi ...... upplementation via a transgene
@ast
Fatal propionic acidemia in mi ...... upplementation via a transgene
@en
Fatal propionic acidemia in mi ...... upplementation via a transgene
@nl
type
label
Fatal propionic acidemia in mi ...... upplementation via a transgene
@ast
Fatal propionic acidemia in mi ...... upplementation via a transgene
@en
Fatal propionic acidemia in mi ...... upplementation via a transgene
@nl
prefLabel
Fatal propionic acidemia in mi ...... upplementation via a transgene
@ast
Fatal propionic acidemia in mi ...... upplementation via a transgene
@en
Fatal propionic acidemia in mi ...... upplementation via a transgene
@nl
P2093
P2860
P356
P1476
Fatal propionic acidemia in mi ...... upplementation via a transgene
@en
P2093
C. Minjares
M. Kobayashi
S. Yamaguchi
T. Miyazaki
P2860
P304
35995–35999
P356
10.1074/JBC.M105467200
P407
P577
2001-09-21T00:00:00Z