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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.

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

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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