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DADA2GT SIGNED

Development of gene therapy and genome editing strategies to treat adenosine deaminase 2 deficiency

Total Cost €

0

EC-Contrib. €

0

Partnership

0

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 DADA2GT project word cloud

Explore the words cloud of the DADA2GT project. It provides you a very rough idea of what is the project "DADA2GT" about.

researcher    disorders    hspc    systemic    morbidity    innovative    function    adenosine    stroke    treatment    caused    efficacy    effectiveness    technologies    expertise    vasculopathy    proven    cells    mortality    haematopoietic    proof    hspcs    cell    options    mediated    disability    ada2    mice    diseases    pids    presume    death    reasonable    data    deaminase    isolated    collectively    stem    humanized    therapy    efficiency    manifestations    option    transplantation    cytopenia    safer    engineering    editing    deficiency    host    progenitor    inflammation    expression    hardly    patients    lentiviral    unsatisfactory    primary    strategies    pid    prove    genome    therapies    correction    pharmacological    clinical    class    risk    proper    world    drugs    immunosuppressive    vitro    acceptable    dada2    autologous    intracranial    mutations    ipscs    therapeutic    addeficiency    successful    gene    autoinflammatory    hsct    haemorrhages    immunodeficiency    vectors    observation    severe    patient   

Project "DADA2GT" data sheet

The following table provides information about the project.

Coordinator
OSPEDALE SAN RAFFAELE SRL 

Organization address
address: VIA OLGETTINA 60
city: MILANO
postcode: 20132
website: www.hsr.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 183˙473 €
 EC max contribution 183˙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-SE
 Starting year 2019
 Duration (year-month-day) from 2019-06-01   to  2021-05-31

 Partnership

Take a look of project's partnership.

# participants  country  role  EC contrib. [€] 
1    OSPEDALE SAN RAFFAELE SRL IT (MILANO) coordinator 183˙473.00

Map

Leaflet | Map data © OpenStreetMap contributors, CC-BY-SA, Imagery © Mapbox

 Project objective

Deficiency of adenosine deaminase type 2 (DADA2) caused by loss-of-function mutations in the ADDeficiency of adenosine deaminase type 2 (DADA2) caused by loss-of-function mutations in the ADA2 gene is a Primary immunodeficiency (PID) diseases characterized by vasculopathy, stroke, intracranial haemorrhages, systemic inflammation, immunodeficiency, and cytopenia. Without proper treatment, DADA2 patients are at high risk of severe disability or death. Targeted pharmacological therapies are not available, and generic immunosuppressive drugs are commonly used with unsatisfactory effectiveness. Haematopoietic stem cell transplantation (HSCT) has proven effective in patients with severe manifestations, but morbidity and mortality are high and hardly acceptable in less severe cases. Thus, safer and targeted therapeutic options for these patients need to be rapidly developed. Based on the observation that HSCT can be successful, it is reasonable to presume that strategies based on ADA2 correction in autologous haematopoietic stem/progenitor cells (HSPCs) may provide new targeted therapeutic approaches for DADA2. The main goal of this project is to establish gene therapy as a new therapeutic option for DADA2. We propose: i) to develop a pre-clinical approach of gene addition mediated by lentiviral vectors targeting ADA2 expression in patient HSPCs; ii) to exploit gene-editing technologies to prove their efficiency for correction of ADA2 mutations. Gene correction efficacy will be assessed as ADA2 expression and activity in vitro in HSPCs and iPSCs isolated from patients, and in mice humanized with patients’ HSPCs. Collectively, this proof-of-concept study will provide new and robust pre-clinical data for the application of lentiviral-mediated HSPC gene therapy in DADA2. This innovative research project is built upon the world-class research expertise developed by the researcher and the host institution in the areas of PIDs, autoinflammatory disorders, and genome engineering.

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