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Ex vivo gene therapy for GM1-gangliosidosis

Total Cost €


EC-Contrib. €






 GT-GM1 project word cloud

Explore the words cloud of the GT-GM1 project. It provides you a very rough idea of what is the project "GT-GM1" about.

omim    neurodegenerative    basis    rare    effect    modified    genome    progenitor    inspire    reconstitution    therapeutic    ventricles    disease    gm    rapid    mechanisms    undegraded    strategy    nature    preventing    expression    administration    life    metabolites    glb1    generate    association    nervous    efficacious    lsds    hspcs    clinical    sustained    severe    gene    mutations    delivered    gm1    molecular    direct    recessive    cell    dysphagia    genetically    conventional    anticipate    animal    hypothesis    cns    beta    alone    myeloid    hypotonia    possibly    delay    inflammation    local    model    secondary    proof    disorder    caused    combining    brain    route    ex    hydrolase    mice    hematopoietic    genomics    gt    therapy    infantile    vivo    elucidate    central    lateral    intravenous    stem    multiple    storage    ameliorating    deep    mediating    progression    lysosomal    230500    manifestations    successfully    individual    galactosidase    enzyme    cells    encoding    therapies    seizures    neuroprotective    autosomal    gangliosidosis    optimized    performed    lentiviral    murine    administered    correction    symptoms    damage    neurodevelopmental    death    transplanted    transfer    copies   

Project "GT-GM1" data sheet

The following table provides information about the project.


Organization address
address: VIA 8 FEBBRAIO 2
city: PADOVA
postcode: 35122

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 Italy [IT]
 Total cost 171˙473 €
 EC max contribution 171˙473 € (100%)
 Programme 1. H2020-EU.1.3.2. (Nurturing excellence by means of cross-border and cross-sector mobility)
 Code Call H2020-MSCA-IF-2018
 Funding Scheme MSCA-IF-EF-RI
 Starting year 2020
 Duration (year-month-day) from 2020-04-01   to  2022-03-31


Take a look of project's partnership.

# participants  country  role  EC contrib. [€] 
1    UNIVERSITA DEGLI STUDI DI PADOVA IT (PADOVA) coordinator 171˙473.00


 Project objective

'GM-gangliosidosis (OMIM #230500) is a rare, autosomal recessive, neurodegenerative Lysosomal Storage Disorder. It is caused by mutations in the GLB1 gene, encoding the lysosomal hydrolase β-galactosidase. Infantile GM1-gangliosidosis is characterized by neurodevelopmental delay, hypotonia, dysphagia, seizures and death by 3 years of life. Due to the rapid progression and severe nature of this disease, which involves storage of undegraded metabolites and secondary mechanisms of cell damage, correction requires a rapid and robust enzyme delivery to the whole central nervous system (CNS), possibly associated to reduction of local inflammation. Here we propose an ex vivo gene therapy (GT) strategy aimed at preventing or ameliorating the symptoms of the disease in the murine model. Multiple copies of GLB1, alone or in association with a neuroprotective factor, will be delivered ex vivo to hematopoietic stem/progenitor cells by lentiviral gene transfer to determine a sustained and robust expression of the therapeutic enzyme in the CNS of transplanted mice. Genetically modified HSPCs will be administered by a novel approach combining the conventional intravenous route with direct administration into the brain lateral ventricles, to anticipate the myeloid reconstitution in the brain and possibly the therapeutic effect. Our working hypothesis is that this optimized GT strategy could successfully control disease manifestations in the animal model. Moreover, a deep genome-wide genomics analysis will be performed on individual brain cells to elucidate the molecular mechanisms at the basis of the disease and mediating the therapeutic effect. The study will generate a proof of concept for a future clinical development of an efficacious ex vivo GT for infantile GM1-gangliosidosis and will inspire the development of therapies for other LSDs. '

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