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GENE FOR CURE SIGNED

Expanding and extending gene therapy of monogenic diseases of the haematopoietic system

Total Cost €

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EC-Contrib. €

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Partnership

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 GENE FOR CURE project word cloud

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

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Project "GENE FOR CURE" data sheet

The following table provides information about the project.

Coordinator
INSTITUT NATIONAL DE LA SANTE ET DE LA RECHERCHE MEDICALE 

Organization address
address: RUE DE TOLBIAC 101
city: PARIS
postcode: 75654
website: www.inserm.fr

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 France [FR]
 Total cost 2˙445˙267 €
 EC max contribution 2˙445˙267 € (100%)
 Programme 1. H2020-EU.1.1. (EXCELLENT SCIENCE - European Research Council (ERC))
 Code Call ERC-2015-AdG
 Funding Scheme ERC-ADG
 Starting year 2016
 Duration (year-month-day) from 2016-10-01   to  2021-09-30

 Partnership

Take a look of project's partnership.

# participants  country  role  EC contrib. [€] 
1    INSTITUT NATIONAL DE LA SANTE ET DE LA RECHERCHE MEDICALE FR (PARIS) coordinator 2˙137˙827.00
2    ASSISTANCE PUBLIQUE HOPITAUX DE PARIS FR (PARIS) participant 307˙440.00

Map

 Project objective

Given that (i) not all patients with a monogenic disease affecting the lymphohaematopoietic system have an HLA-genoidentical sibling donor and (ii) severe immunological complications worsen the outcome in HLA-partially-matched hematopoietic stem cell transplantation (HSCT), the genetic modification of autologous hematopoietic stem cells (HSCs) has become a powerful curative treatment option for these individuals. The present project seeks to further consolidate the rationale for replacing HLA-partially-matched HSCT with a gene therapy approach. Wiskott-Aldrich syndrome is a primary immunodeficiency whose severity is due to impairment of both lymphoid and myeloid cell function. We have reported robust evidence showing that the infusion of gene-corrected autologous HSCs enables the restoration of the T cell function. However, we are still cautious with regard to claiming the stable correction of autoimmunity and thrombocytopenia. Accordingly, we plan to thoroughly monitor long-term B cell functional outcomes and the platelet count in our treated patients. Moreover, we wish to extend the gene therapy approach to the SCID caused by mutations in the DLREIC gene, since the long-term post-HSCT outcomes are particularly poor. The preclinical work has been completed; initiation of a clinical protocol is the next step. Immune-dysregulation polyendocrinopathy enteropathy X-linked (IPEX) and sickle cell anaemia (SCA) are the last two target diseases. IPEX is a devastating disease caused by mutation of FOXP3 transcription factor; it may be possible to correct it by infusing gene-modified CD4Tcells. We intend to accumulate the data required to prove our working hypothesis. SCA is a worldwide public health issue. We are seeking to improve the conventional gene therapy procedure and to evaluate the efficacy of CrisP/Cas9-mediated disruption of the CIS-regulatory elements required for HbF silencing. This disruption may provide a cure for SCA.

 Publications

year authors and title journal last update
List of publications.
2018 Leslie Weber, Valentina Poletti, Elisa Magrin, Chiara Antoniani, Samia Martin, Charles Bayard, Hanem Sadek, Tristan Felix, Vasco Meneghini, Michael N. Antoniou, Wassim El-Nemer, Fulvio Mavilio, Marina Cavazzana, Isabelle Andre-Schmutz, Annarita Miccio
An Optimized Lentiviral Vector Efficiently Corrects the Human Sickle Cell Disease Phenotype
published pages: 268-280, ISSN: 2329-0501, DOI: 10.1016/j.omtm.2018.07.012
Molecular Therapy - Methods & Clinical Development 10 2020-02-19
2018 Chiara Antoniani, Vasco Meneghini, Annalisa Lattanzi, Tristan Felix, Oriana Romano, Elisa Magrin, Leslie Weber, Giulia Pavani, Sara El Hoss, Ryo Kurita, Yukio Nakamura, Thomas J. Cradick, Ante S. Lundberg, Matthew Porteus, Mario Amendola, Wassim El Nemer, Marina Cavazzana, Fulvio Mavilio, Annarita Miccio
Induction of fetal hemoglobin synthesis by CRISPR/Cas9-mediated editing of the human β-globin locus
published pages: 1960-1973, ISSN: 0006-4971, DOI: 10.1182/blood-2017-10-811505
Blood 131/17 2020-02-19
2017 Marina Cavazzana, Chiara Antoniani, Annarita Miccio
Gene Therapy for β-Hemoglobinopathies
published pages: 1142-1154, ISSN: 1525-0016, DOI: 10.1016/j.ymthe.2017.03.024
Molecular Therapy 25/5 2020-02-19

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