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PD UpReg SIGNED

Gene knock-up via 3’UTR targeting to treat Parkinson’s disease

Total Cost €

0

EC-Contrib. €

0

Partnership

0

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 PD UpReg project word cloud

Explore the words cloud of the PD UpReg project. It provides you a very rough idea of what is the project "PD UpReg" about.

experimentally    utr    revolutionary    restricted    met    pattern    transgenics    experiments    dopaminergic    physiologically    parkinson    made    disease    therapeutic    concurrently    endogenous    least    strategy    humans    potent       death    neuronal    mitochondrial    cas9    hypothesize    ectopically    pathogenesis    treat    limited    mouse    provides    restore    elevated    degeneration    editing    toward    deploy    proteostasis    gdnf    first    unlike    overexpressed    create    genes    proof    function    persons    appropriate    mice    thereby    upregulate    incurable    mediated    axons    upregulating    model    adult    shown    ectopic    suggesting    adulthood    progress    expression    pd    validation    elderly    alpha    attempts    treatment    crispr    elevation    safe    ntfs    protect    protects    gene    instead    neurotrophic    cells    causes    synuclein    collectively    levels    models    life    spatiotemporal    defects    treating    therapy    overexpressing   

Project "PD UpReg" data sheet

The following table provides information about the project.

Coordinator
HELSINGIN YLIOPISTO 

Organization address
address: YLIOPISTONKATU 3
city: HELSINGIN YLIOPISTO
postcode: 14
website: www.helsinki.fi

contact info
title: n.a.
name: n.a.
surname: n.a.
function: n.a.
email: n.a.
telephone: n.a.
fax: n.a.

 Coordinator Country Finland [FI]
 Total cost 1˙999˙987 €
 EC max contribution 1˙999˙987 € (100%)
 Programme 1. H2020-EU.1.1. (EXCELLENT SCIENCE - European Research Council (ERC))
 Code Call ERC-2016-COG
 Funding Scheme ERC-COG
 Starting year 2017
 Duration (year-month-day) from 2017-09-01   to  2022-08-31

 Partnership

Take a look of project's partnership.

# participants  country  role  EC contrib. [€] 
1    HELSINGIN YLIOPISTO FI (HELSINGIN YLIOPISTO) coordinator 1˙999˙987.00

Map

 Project objective

Parkinson’s disease (PD) affects 1% of elderly persons and is currently incurable. PD pathogenesis is driven, at least in part, by defects in proteostasis and mitochondrial function, leading to degeneration of dopaminergic axons and neuronal death. Neurotrophic factors (NTFs) such as GDNF can protect and restore dopaminergic axons. However, attempts to deploy NTFs ectopically in therapy models, or to increase proteostasis and mitochondrial function, have met with only limited success. I hypothesize that instead of ectopic application, over-expression of relevant pathways restricted to physiologically appropriate cells provides a potent therapeutic approach to treat PD. I have made significant progress toward this goal by targeting the 3’UTR in the mouse Gdnf gene, thereby increasing expression levels without affecting the gene’s spatiotemporal expression pattern. Using this approach I have shown that elevation of endogenous GDNF levels protects mice from experimentally induced PD. Unlike ectopic GDNF application, it causes no side effects. Importantly, I have established that GDNF levels can be elevated by 3’UTR targeting in adulthood, suggesting that this strategy could be applied in humans late in life. I will use 3’UTR targeting to study the therapeutic potential of overexpressing endogenous genes, using transgenics and CRISPR-Cas9‒mediated 3’UTR editing in adult mice. First, I will increase the expression of NTFs in adult mice with experimentally induced PD. Next, I will upregulate genes important in mitochondrial function and proteostasis and test whether concurrently upregulating endogenous NTFs is a viable approach for treating PD. Third, I will use 3’UTR targeting to create a mouse model of PD in which alpha-synuclein is overexpressed, for better validation of my therapeutic strategy. Collectively, these experiments should establish proof of concept for a revolutionary, safe and effective treatment for PD.

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The information about "PD UPREG" are provided by the European Opendata Portal: CORDIS opendata.

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