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Zinc finger gene therapy in the brain for treating Huntington's disease

Total Cost €


EC-Contrib. €






 Fingers4Cure project word cloud

Explore the words cloud of the Fingers4Cure project. It provides you a very rough idea of what is the project "Fingers4Cure" about.

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Project "Fingers4Cure" data sheet

The following table provides information about the project.


Organization address
city: LONDON
postcode: SW7 2AZ

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 United Kingdom [UK]
 Total cost 149˙995 €
 EC max contribution 149˙995 € (100%)
 Programme 1. H2020-EU.1.1. (EXCELLENT SCIENCE - European Research Council (ERC))
 Code Call ERC-2014-PoC
 Funding Scheme ERC-POC
 Starting year 2015
 Duration (year-month-day) from 2015-02-01   to  2016-07-31


Take a look of project's partnership.

# participants  country  role  EC contrib. [€] 


 Project objective

Huntington’s disease (HD) is a lethal inherited neurodegenerative disorder which currently has no cure. Patients develop a devastating loss of muscle control and brain function, with symptoms typically developing at 35 - 45 years of age, although they can start much earlier. Suffering is high for both patients and carers, with death generally occurring within 10 - 15 years of diagnosis. We are working on a novel, curative gene therapy approach based on reducing the levels of the HTT disease gene products, while maintaining the levels of the normal protein. It is based on the delivery of proprietary zinc finger proteins (ZFPs) to the brain. Efficient delivery is accomplished through the use of viral vectors that carry the sequences coding for our ZFPs. Such ZFPs are artificially engineered proteins that specifically bind the defective expanded HTT DNA sequences (but not the normal HTT sequences or other, unrelated sequences). Binding represses the synthesis of the toxic gene products. We have published data demonstrating efficient mutant HTT repression in mouse (Garriga-Canut, M., et al. & Isalan M. Synthetic zinc finger repressors reduce mutant Huntingtin expression in the brain of R6/2 mice. Proc Natl Acad Sci USA 109: E3136-E3145, 2012). We now need to improve the viral vectors for the long term expression of our ZFPs in the mouse brain, as the basis for a single-intervention, long-term therapy in humans (technical improvements). In parallel, we need to maintain the patent applications that protect the technology through various national and regional phases, and protect new developments that may arise (intellectual property), while engaging the necessary IPR and market studies/activities that help us find an industry partner that licenses our technology and moves it forward into the clinic (exploitation). This proof of concept grant is critical in all three aspects.


year authors and title journal last update
List of publications.
2015 Michal Mielcarek, Mark Isalan
A shared mechanism of muscle wasting in cancer and Huntington’s disease
published pages: , ISSN: 2001-1326, DOI: 10.1186/s40169-015-0076-z
Clinical and Translational Medicine 4/1 2019-07-23
2016 Carmen Agustín-Pavón, Michal Mielcarek, Mireia Garriga-Canut, Mark Isalan
Deimmunization for gene therapy: host matching of synthetic zinc finger constructs enables long-term mutant Huntingtin repression in mice
published pages: , ISSN: 1750-1326, DOI: 10.1186/s13024-016-0128-x
Molecular Neurodegeneration 11/1 2019-07-23
2015 Michal Mielcarek
Huntington\'s disease is a multi-system disorder
published pages: e1058464, ISSN: 2167-5511, DOI: 10.1080/21675511.2015.1058464
Rare Diseases 3/1 2019-07-23

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