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

Total Cost €


EC-Contrib. €






 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.

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

Project "PD UpReg" data sheet

The following table provides information about the project.


Organization address
postcode: 14

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


Take a look of project's partnership.

# participants  country  role  EC contrib. [€] 


 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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