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

Total Cost €


EC-Contrib. €






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

Project "DADA2GT" data sheet

The following table provides information about the project.


Organization address
address: VIA OLGETTINA 60
city: MILANO
postcode: 20132

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


Take a look of project's partnership.

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


 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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The information about "DADA2GT" are provided by the European Opendata Portal: CORDIS opendata.

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