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GT-GM1 SIGNED

Ex vivo gene therapy for GM1-gangliosidosis

Total Cost €

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EC-Contrib. €

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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.

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

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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lastchecktime (2022-05-28 6:51:08) correctly updated