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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.

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

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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