Results 141 to 150 of about 532,411 (388)

Efficacy of early haematopoietic stem cell transplantation versus enzyme replacement therapy on neurological progression in severe Hunter syndrome: Case report of siblings and literature review

, 2022
Srividya Sreekantam, Laura Smith, Catherine Stewart, Shauna Kearney, Sarah Lawson, Julian Raiman, Suresh Vijay, Saikat Santra   +7 more
openalex   +1 more source

Engineering and Characterization of an Enzyme Replacement Therapy for Classical Homocystinuria.

Biomacromolecules, 2017
Homocystinuria due to loss of cystathionine beta-synthase (CBS) causes accumulation of homocysteine and depletion of cysteine. Current treatments are suboptimal, and thus the development of an enzyme replacement therapy based on PEGylated human truncated
T. Majtan, Insun Park, Richard S Carrillo, Erez M. Bublil, J. Kraus   +4 more
semanticscholar   +1 more source

Dietary nitrate and nitrite protect against doxorubicin‐induced cardiac fibrosis and oxidative protein damage in tumor‐bearing mice

FEBS Open Bio, EarlyView.
Chemotherapies such as doxorubicin can have toxic effects on healthy cardiovascular/heart tissue. Following up on a doxorubicin toxicity study in mice without tumors where nitrate water was cardioprotective (lessened toxicity), this study with tumor‐bearing mice undergoing doxorubicin treatment showed no negative effect of nitrate and nitrite on drug ...
Rama D. Yammani, Xiaofei Chen, Nildris Cruz‐Diaz, Xuewei Zhu, Swati Basu, Daniel B. Kim‐Shapiro, David R. Soto‐Pantoja, Leslie B. Poole   +7 more
wiley   +1 more source

EXPERIENCE OF IMPLEMENTING NEW TECHNOLOGY TREATMENT OF CHILDREN WITH MPS IN THE RUSSIAN PEDIATRIC PRACTICE

Педиатрическая фармакология, 2011
The paper is devoted to one of the rare genetically determined diseases — mucopolysaccharidosis. Despite the great achievement of science — the development of the pathogenetic enzyme replacement therapy, many challenges remain.
A.A. Baranov, L.S. Namazova-Baranova, A.K. Gevorkyan, V.M. Krasnov, L.M. Kuzenkova, G.A. Kashkaradze, N.D. Vashakmadze, T.V. Podkletnova   +7 more
doaj   +2 more sources

Diabetes‐induced vascular calcification is associated with low pyrophosphate and its oral supplementation prevents calcification in diabetic mice

FEBS Open Bio, EarlyView.
Induction of diabetes in three different mouse strains uniformly resulted in an increase in TNAP activity and a reduction in pyrophosphate (PPi) in the circulation. Inhibition of TNAP restored plasma PPi. Diabetes‐induced calcification in the media layer of the aorta was detected only in the Abcc6−/− strain, which is predisposed to ectopic ...
Krisztina Fülöp, Eszter Kozák, Natália Tőkési, Lilian Kocsis, Anikó Kelemen, Zsuzsanna Geszti, Adriána Kutás, Mariann Harangi, Ágnes Diószegi, Zsolt Rapi, József Balla, Olivier Le Saux, András Váradi, Viola Pomozi   +13 more
wiley   +1 more source

Transcranial pulsed ultrasound facilitates brain uptake of laronidase in enzyme replacement therapy for Mucopolysaccharidosis type I disease

Orphanet Journal of Rare Diseases, 2017
Background Mucopolysaccharidosis type I (MPS I) is a debilitating hereditary disease characterized by alpha-L-iduronidase (IDUA) deficiency and consequent inability to degrade glycosaminoglycans.
Yu-Hone Hsu, Ren-Shyan Liu, Win-Li Lin, Yeong-Seng Yuh, Shuan-Pei Lin, Tai-Tong Wong   +5 more
doaj   +1 more source

Evaluation of in vitro toxicity of common phytochemicals included in weight loss supplements using 1H NMR spectroscopy

FEBS Open Bio, EarlyView.
We investigated the toxicity of 12 active compounds commonly found in herbal weight loss supplements (WLS) using human liver and colon cell models. Epigallocatechin‐3‐gallate was the only compound showing significant toxicity. Metabolic profiling revealed protein degradation, disrupted energy and lipid metabolism suggesting that the inclusion of EGCG ...
Emily C. Davies, Garth L. Maker, Ian F. Musgrave, Samantha Lodge   +3 more
wiley   +1 more source

dUTPase is essential in zebrafish development and possesses several single‐nucleotide variants with pronounced structural and functional consequences

FEBS Open Bio, EarlyView.
dUTPases are involved in balancing the appropriate nucleotide pools. We showed that dUTPase is essential for normal development in zebrafish. The different zebrafish genomes contain several single‐nucleotide variations (SNPs) of the dut gene. One of the dUTPase variants displayed drastically lower protein stability and catalytic efficiency as compared ...
Viktória Perey‐Simon, Angéla Békesi, Latifa Kazzazy, Jázmin Mihály, Máté Varga, Beáta G. Vértessy, Kinga Nyíri   +6 more
wiley   +1 more source

Tumor‐stromal crosstalk and macrophage enrichment are associated with chemotherapy response in bladder cancer

FEBS Open Bio, EarlyView.
Chemoresistance in bladder cancer: Macrophage recruitment associated with CXCL1, CXCL5 and CXCL8 expression is characteristic of Gemcitabine/Cisplatin (Gem/Cis) Non‐Responder tumors (right side) while Responder tumors did not show substantial tumor‐stromal crosstalk (left side). All biological icons are attributed to Bioicons: carcinoma, cancerous‐cell‐
Sophie Leypold, Janik Riese, Lancelot Seillier, Mark Kühnel, Julia Pannhausen, Charlotte O. J. Fröhlich, Christian Martin, Peter Boor, Matthias Saar, Danny D. Jonigk, Nadine T. Gaisa, Michael Rose   +11 more
wiley   +1 more source

Dihydroorotate dehydrogenase (DHODH) regulates trophoblast syncytialization through organelle stress–induced cellular senescence

FEBS Open Bio, EarlyView.
The inhibition of mitochondrial dihydroorotate dehydrogenase (DHODH) impairs syncytialization and induces cellular senescence via mitochondrial and endoplasmic reticulum stress in human trophoblast stem cells, elevating sFlt1/PlGF levels, a hallmark of placental dysfunction in hypertensive disorders of pregnancy.
Kanoko Yoshida, Kazuya Kusama, Ayaka Horiuchi, Yu Kawaguchi, Atsuya Tsuru, Mikihiro Yoshie, Kazuhiro Tamura   +6 more
wiley   +1 more source