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http://dx.doi.org/10.1165/rcmb.2021-0269ED | DOI Listing |
PLoS Negl Trop Dis
February 2024
Institute of Biochemistry and Cell Biology, National Research Council (IBBC-CNR), Adriano Buzzati-Traverso Campus, Monterotondo, Rome, Italy.
Schistosomiasis is a major neglected parasitic disease that affects more than 240 million people worldwide caused by Platyhelminthes of the genus Schistosoma. The treatment of schistosomiasis relies on the long-term application of a single safe drug, praziquantel (PZQ). Unfortunately, PZQ is very effective on adult parasites and poorly on larval stage and immature juvenile worms; this can partially explain the re-infection in endemic areas where patients are likely to host parasites at different developmental stages concurrently.
View Article and Find Full Text PDFAm J Respir Cell Mol Biol
December 2021
National Heart and Lung Institute Imperial College London, United Kingdom.
ChemMedChem
December 2017
Organic and Bioorganic Chemistry, Bielefeld University, Universitätsstrasse 25, 33615, Bielefeld, Germany.
As histone deacetylases (HDACs) play an important role in the treatment of cancer, their selective inhibition has been the subject of various studies. These continuous investigations have given rise to a large collection of pan- and selective HDAC inhibitors, containing diverse US Food and Drug Administration (FDA)-approved representatives. In previous studies, a class of alkyne-based HDAC inhibitors was presented.
View Article and Find Full Text PDFOrphanet J Rare Dis
May 2013
Unit of Molecular Neurogenetics, Istituto di Ricovero e Cura a Carattere Scientifico (IRCCS), Milan, Italy.
Background: We studied a family including two half-siblings, sharing the same mother, affected by slowly progressive, adult-onset neurological syndromes. In spite of the diversity of the clinical features, characterized by a mild movement disorder with cognitive impairment in the elder patient, and severe motor-neuron disease (MND) in her half-brother, the brain Magnetic Resonance Imaging (MRI) features were compatible with adult-onset Alexander's disease (AOAD), suggesting different expression of the same, genetically determined, condition.
Methods: Since mutations in the alpha isoform of glial fibrillary acidic protein, GFAP-α, the only cause so far known of AOAD, were excluded, we applied exome Next Generation Sequencing (NGS) to identify gene variants, which were then functionally validated by molecular characterization of recombinant and patient-derived cells.
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