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 TERMINÉ 

Smart multifunctional GLA-nanoformulation for Fabry disease (Smart-4-Fabry)

Date du début: 1 janv. 2017, Date de fin: 31 déc. 2020,

Lysosomal storage disorders (LSD) diseases are a group of rare diseases that currently lack a definitive cure. LSD incidence is about 1:5,000 - 1:10,000, representing a serious global health problem. In the case of Fabry LSD Disease (FD), the deficiency in α-Galactosidase A (GLA) enzyme activity results in the cellular accumulation of neutral glycosphingolipids, leading to widespread vasculopathy ...
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 TERMINÉ 

Stem Cells for Relevant Efficient Extended and Normalized Toxicology (SCR&TOX)

Date du début: 1 janv. 2011, Date de fin: 31 déc. 2015,

In the development of products for use by humans it is vital to identify compounds with toxic properties at an early stage of their development, to avoid spending time and resource on unsuitable and potentially unsafe candidate products. Human pluripotent stem cell lines offer a unique opportunity to develop a wide variety of human cell-based test systems because they may be expanded indefinitely ...
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 TERMINÉ 
The consortium aims to develop and produce an Anticalin, a member of a novel high-affinity scaffold derived from the lipocalin protein family. The Anticalin is specific for hepcidin which is a central regulator of iron homeostasis, and will be used to antagonize hepcidin for the treatment of “anemia of chronic disease” (ACD). Anticalins are genetically modified lipocalins that can target almost an ...
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