Gene/Protein Disease Symptom Drug Enzyme Compound
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Query: UMLS:C0039730 (thalassemia)
 10,305 document(s) hit in 31,850,051 MEDLINE articles (0.00 seconds)

A series of polymers bearing hydroxamic acid-terminated side chains were prepared for the purpose of developing new iron chelators for treating iron overload in beta-thalassemia (Cooley's anemia) and other iron diseases. The polymers are for the most part amino acid amide derivatives of acrylic and methacrylic acid with the terminal carboxyl group converted to the hydroxamic acid. The polymers are generally water soluble and sequester iron(III) avidly. The polymeric iron chelators were assayed via a mouse screen for activity in removing iron. Iron overloaded mice were administered i.p. the iron chelator over a 7-day period. Urine and feces were collected and the iron content measured by atomic absorption. At the end of the treatment period the mice were sacrificed and the livers and spleens were homogenized and examined for iron content. The results were compared with similar data obtained for the iron chelator drug desferrioxamine as a standard. Four of the polymers prepared exhibited strong activity, as good or better than desferrioxamine in iron removal capability. The four polymers are the polyacroloyl and polymethacryloyl derivatives of beta-alanine with the side chain carboxyls converted to the N'--H or N'--CH3 hydroxamic acids. Of these four the polyacryloyl N'--CH3 derivative exhibited superior behavior, being 3 to 5 times as effective as desferrioxamine at the lower dose level. None of the four polymers produced toxic signs and the administration was accompanied by little or no pain response.
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PMID:Evaluation of polymeric hydroxamic acid iron chelators for treatment of iron overload. 397 22

Pathogenesis of different diseases showed that some of them, especially thalassemia (T) and rheumatoid arthritis (RA) have an implicit association with oxidative stress and altered levels of reactive oxygen species (ROS). Introducing ROS level and the balance between ROS and antioxidants as essential metrics, an attempt was made to classify T and RA from normal individuals (treated as controls)(C) using synchronous fluorescence spectroscopy (SFS) and Raman line intensity of water. This non-invasive and label-free approach was backed up by a categorization algorithm that helped in the prediction of disease types from serum samples. The predictive system constituted principal component analysis (PCA) with four parameters, namely spectral intensity ratios of reduced nicotinamide adenine dinucleotide (NADH) to tryptophan (Trp) (NADH/Trp), kynurenine (Kyn) to tryptophan (Kyn/Trp), kynurenine to NADH (Kyn/NADH), and logarithmic changes in Raman line intensity of water (Rline), with the index headers containing the disease types. Rline has a positive correlation with both Kyn/Trp and Kyn/NADH and a negative correlation with NADH/Trp ratio, implying its direct or indirect association with oxidative stress. In addition to the classification of T, RA, and C a sub-classification of T into beta major and E-beta in their post and pre-splenectomized surgical stages could also be realized. Furthermore, receiver operating characteristic (ROC) analysis was deployed to ascertain that the misclassification error (ME) was negligible for the disease types. Graphical Abstract A schematic representation of the workflow converging into the categorical classification of disease classes.
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PMID:Label-free detection of thalassemia and other ROS impairing diseases. 3268 Dec 15