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CRISS SIGNED

CRISPR Gene Correction for Severe Combined Immunodeficiency Caused by Mutations in Recombination-activating gene 1 and 2 (RAG1 and RAG2)

Total Cost €

0

EC-Contrib. €

0

Partnership

0

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 CRISS project word cloud

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

interspaced    complete    rag    disease    hspcs    developmental    safety    corrective    genome    translate    homologous    bone    cellular    hematopoietic    limitations    correct    basic    clinical    immunodeficiencies    transformation    active    rag1    viral    genetic    clinically    lineage    functional    combined    disorders    fundamental    precise    correction    life    therapy    precisely    scids    curative    patient    protein    vectors    trials    science    transgene    recombination    methodical    gt    severe    patients    crispr    shown    efficacy    hr    fashion    cas9    rag2    therapies    clear    clustered    allogeneic    cells    threatening    manner    editing    transgenes    immune    expressed    born    uncontrolled    unable    frequencies    palindromic    scid    diseases    wave    transplantation    caused    stimulate    marrow    stem    risks    single    containing    successfully    gene    contrast    causing    mutation    therapeutically    modifying    mutations    beneficial    progenitor    genes    ada    10    careful   

Project "CRISS" data sheet

The following table provides information about the project.

Coordinator
BAR ILAN UNIVERSITY 

Organization address
address: BAR ILAN UNIVERSITY CAMPUS
city: RAMAT GAN
postcode: 52900
website: www.biu.ac.il

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 Israel [IL]
 Total cost 1˙372˙839 €
 EC max contribution 1˙372˙839 € (100%)
 Programme 1. H2020-EU.1.1. (EXCELLENT SCIENCE - European Research Council (ERC))
 Code Call ERC-2017-STG
 Funding Scheme ERC-STG
 Starting year 2017
 Duration (year-month-day) from 2017-10-01   to  2022-09-30

 Partnership

Take a look of project's partnership.

# participants  country  role  EC contrib. [€] 
1    BAR ILAN UNIVERSITY IL (RAMAT GAN) coordinator 1˙372˙839.00

Map

 Project objective

The severe combined immunodeficiencies (SCIDs) are a set of life threatening genetic diseases in which patients are born with mutations in single genes and are unable to develop functional immune systems. While allogeneic bone marrow transplantation can be curative for these diseases, there remain significant limitations to this approach. Gene therapy using viral vectors containing a corrective transgene is being developed for some of these disorders, most successfully for ADA-SCID. However, for other SCID disorders, such as those caused by genetic mutations in RAG1 and RAG2, the transgene needs to be expressed in a precise, developmental and lineage specific manner to achieve functional gene correction and to avoid the risks of cellular transformation. In contrast to using viral vectors to deliver transgenes in an uncontrolled fashion, we are working towards using genome editing by homologous recombination (HR) to correct the disease causing mutation by precisely modifying the genome. We have shown that by using clustered, regularly interspaced, short palindromic repeats (CRISPR) and the CRISPR-associated protein 9 (Cas9) system we can stimulate genome editing by HR at frequencies that should be therapeutically beneficial (>10%) in hematopoietic stem and progenitor cells (HSPCs). The overall focus of the proposal is to translate our basic science studies to use in RAG-SCID patient-derived HSPCs in methodical, careful and pre-clinically relevant fashion. The fundamental approach is to develop a highly active functional genome editing system using CRISPR-Cas9 for RAG-SCIDs and complete pre-clinical efficacy and safety studies to show the approach has a clear path towards future clinical trials. Our goal with this proposal is to develop the next wave of curative therapies for SCIDs and other hematopoietic disorders using genome editing.

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

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