Profile of idursulfase for the treatment of Hunter syndrome

Simona Sestito, Ferdinando Ceravolo, Michele Grisolia, Elisa Pascale, Licia Pensabene, Daniela Concolino Department of Pediatrics, University Magna Graecia of Catanzaro, Catanzaro, Italy Abstract: Mucopolysaccharidosis type II (MPS II; Hunter syndrome) is a rare X-linked lysosomal storage disorder caused by deficiency of the enzyme iduronate-2-sulfatase (IDS). Enzyme replacement therapy (ERT) with recombinant human IDS, available since 2005, is currently the most appropriate treatment for this progressive, multisystemic, chronic, and life-threatening disease. Efficacy and safety of therapy with idursulfase have been assessed in several clinical trials, and confirmed in many clinical reports. Long-term follow-up of patients receiving ERT has demonstrated the importance of an early onset of treatment with idursulfase, before irreversible pathological changes occur. Intravenously administered idursulfase is not able to cross the blood–brain barrier, so neurological signs and symptoms cannot benefit from ERT, still remaining a major challenge in the treatment of MPS II. Keywords: MPS II, glycosaminoglycans, enzyme replacement therapy, ER

The Potential of Visible and Far-Red to Near-Infrared Light in Glaucoma Neuroprotection

Alternative treatment strategies are necessary to reduce the severity of glaucoma, a group of eye conditions that progressively damage the optic nerve and impair vision. The aim of this review is to gain insight into potentially exploitable molecular mechanisms to slow down the death of retinal ganglion cells (RGCs), a fundamental element in the pathophysiology of all forms of glaucoma, and to stimulate adult optic nerve repair. For this purpose, we focus our analysis on both visible and far-red to near-infrared light photobiomodulation (PBM) as phototherapeutic agents, which were recently proposed in RGCs, and on the nerve lamina region neural progenitor cell (ONLR-NPC) niche. Both are suggested as potential strategies in glaucoma neuroprotection. We discuss the impact of beneficial molecular effects of PBM on both mitochondrial derangement and the alteration of ion fluxes that are considered important causes of RGC damage, as well as on the stimulation of progenitor cells. We suggest these are the most promising approaches to prevent excessive neuronal cell loss. We describe the experimental evidence supporting the validity of PBM therapy which, despite being a safe, non-invasive, inexpensive, and easy to administer procedure, has not yet been fully explored in the clinical practice of glaucoma treatment

Acute toxicity and cytotoxicity of Bacillus thuringiensis and Bacillus sphaericus strains on fish and mouse bone marrow.

Made available in DSpace on 2018-06-11T17:31:26Z (GMT). No. of bitstreams: 1 SP19703ID31175.pdf: 199147 bytes, checksum: 639b74490a905c882b2db6f70e918560 (MD5) Previous issue date: 2009-03-12bitstream/item/178426/1/SP-19703-ID-31175.pd

Toxicidade reprodutiva do agrotoxico endosulfan para caramujos aquaticos: estudo em duas geracoes de Biomphalaria tenagophila.

bitstream/CPAC-2009/24322/1/p2003_10.pd

Tritium retention in W plasma-facing materials : Impact of the material structure and helium irradiation

This article has an erratum: DOI 10.1016/j.nme.2020.100729Plasma-facing materials for next generation fusion devices, like ITER and DEMO, will be submitted to intense fluxes of light elements, notably He and H isotopes (HI). Our study focuses on tritium (T) retention on a wide range of W samples: first, different types of W materials were investigated to distinguish the impact of the pristine original structure on the retention, from W-coated samples to ITER-grade pure W samples submitted to various annealing and manufacturing procedures, along with monocrystalline W for reference. Then, He and He-D irradiated W samples were studied to investigate the impact on He-damages such as nano-bubbles (exposures in LHD or PSI-2) on T retention. We exposed all the samples to tritium gas-loading using a gentle technique preventing any introduction of new damage in the material. Tritium desorption is measured by Liquid Scintillation counting (LSC) at ambient and high temperatures (800 degrees C). The remaining T inventory is then measured by sample full dissolution and LSC. Results on T inventory on He exposed samples highlighted that in all cases, tritium desorption as a gas (HT) increases significantly due to the formation of He damages. Up to 1.8 times more T can be trapped in the material through a competition of various mechanisms, but the major part of the inventory desorbs at room temperature, and so will most likely not take part to the long-term trapped inventory for safety and operational perspectives. Unfortunately, investigation of "as received" industrial W (used for the making of plasma-facing materials) highlighted a strong impact of the pre existing defects on T retention: up to 2.5 times more T is trapped in "as received W" compared to annealed and polish W, and desorbs only at 800 degrees C, meaning ideal W material studies may underestimate T inventory for tokamak relevant conditions.Peer reviewe

Control of magnetic anisotropy by orbital hybridization in (La0.67Sr0.33MnO3)n/(SrTiO3)n superlattice

The asymmetry of chemical nature at the hetero-structural interface offers an unique opportunity to design desirable electronic structure by controlling charge transfer and orbital hybridization across the interface. However, the control of hetero-interface remains a daunting task. Here, we report the modulation of interfacial coupling of (La0.67Sr0.33MnO3)n/(SrTiO3)n superlattices by manipulating the periodic thickness with n unit cells of SrTiO3 and n unit cells La0.67Sr0.33MnO3. The easy axis of magnetic anisotropy rotates from in-plane (n = 10) to out-of-plane (n = 2) orientation at 150 K. Transmission electron microscopy reveals enlarged tetragonal ratio > 1 with breaking of volume conservation around the (La0.67Sr0.33MnO3)n/(SrTiO3)n interface, and electronic charge transfer from Mn to Ti 3d orbitals across the interface. Orbital hybridization accompanying the charge transfer results in preferred occupancy of 3d3z2-r2 orbital at the interface, which induces a stronger electronic hopping integral along the out-of-plane direction and corresponding out-of-plane magnetic easy axis for n = 2. We demonstrate that interfacial orbital hybridization in superlattices of strongly correlated oxides may be a promising approach to tailor electronic and magnetic properties in device applications