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GT-GM1 SIGNED

Ex vivo gene therapy for GM1-gangliosidosis

Total Cost €

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EC-Contrib. €

0

Partnership

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 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.

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

Project "GT-GM1" data sheet

The following table provides information about the project.

Coordinator
UNIVERSITA DEGLI STUDI DI PADOVA 

Organization address
address: VIA 8 FEBBRAIO 2
city: PADOVA
postcode: 35122
website: www.unipd.it

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

 Partnership

Take a look of project's partnership.

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

Map

 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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