Opendata, web and dolomites

DADA2GT SIGNED

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

Total Cost €

0

EC-Contrib. €

0

Partnership

0

Views

0

 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.

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

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

 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.

Are you the coordinator (or a participant) of this project? Plaese send me more information about the "DADA2GT" project.

For instance: the website url (it has not provided by EU-opendata yet), the logo, a more detailed description of the project (in plain text as a rtf file or a word file), some pictures (as picture files, not embedded into any word file), twitter account, linkedin page, etc.

Send me an  email (fabio@fabiodisconzi.com) and I put them in your project's page as son as possible.

Thanks. And then put a link of this page into your project's website.

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

More projects from the same programme (H2020-EU.1.3.2.)

InBPSOC (2020)

Increases biomass production and soil organic carbon stocks with innovative cropping systems under climate change

Read More  

TheaTheor (2018)

Theorizing the Production of 'Comedia Nueva': The Process of Play Configuration in Spanish Golden Age Theater

Read More  

PmNC (2019)

Policy-making of early nature conservation. The Netherlands and the United Kingdom compared, 1930-1960

Read More