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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 €

EC-Contrib. €

Partnership

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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.

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

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 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.	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.