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Assessing efficacy and safety of genome EDITing approaches for Sickle Cell Disease

Project description

Evaluation of genome-editing therapies for sickle cell disease

Sickle cell disease (SCD) is a result of a single amino acid substitution in the beta-globin chain of adult haemoglobin. The severity of SCD is alleviated by the co-inheritance of mutations driving the expression of foetal gamma-globin in adults. Transplantation of autologous, genetically modified haematopoietic stem/progenitor cells (HSPC) represents a therapeutic option for SCD. The EU-funded EDITSCD project aims to understand the molecular and cellular mechanisms underlying SCD HSPC dysfunctions and evaluate the impact of genome editing approaches on SCD HSPC. The study objective is to improve the SCD gene therapy strategy and evaluate the best tools and protocols for genome editing HSPC therapies.

Coordinator

IMAGINE INSTITUT DES MALADIES GENETIQUES NECKER ENFANTS MALADES FONDATION
Net EU contribution
€ 1 486 250,00
Address
24 Bd Du Montparnasse
75015 Paris 15
France

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Region
Ile-de-France Ile-de-France Paris
Activity type
Research Organisations
Other funding
€ 0,00

Participants (6)

OSPEDALE SAN RAFFAELE SRL
Italy
Net EU contribution
€ 1 000 000,00
Address
Via Olgettina 60
20132 Milano

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Region
Nord-Ovest Lombardia Milano
Activity type
Private for-profit entities (excluding Higher or Secondary Education Establishments)
Other funding
€ 0,00
UNIVERSITAETSKLINIKUM FREIBURG
Germany
Net EU contribution
€ 1 330 000,00
Address
Hugstetter Strasse 49
79106 Freiburg

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Region
Baden-Württemberg Freiburg Freiburg im Breisgau, Stadtkreis
Activity type
Higher or Secondary Education Establishments
Other funding
€ 0,00
BAR ILAN UNIVERSITY
Israel
Net EU contribution
€ 610 000,00
Address
Bar Ilan University Campus
52900 Ramat Gan

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Activity type
Higher or Secondary Education Establishments
Other funding
€ 0,00
INSTITUT NATIONAL DE LA SANTE ET DE LA RECHERCHE MEDICALE
France
Net EU contribution
€ 793 750,00
Address
Rue De Tolbiac 101
75654 Paris

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Region
Ile-de-France Ile-de-France Paris
Activity type
Research Organisations
Other funding
€ 0,00
ASTRAZENECA AB
Sweden
Net EU contribution
€ 520 000,00
Address
Vastra Maelarhamnen
151 85 Sodertaelje

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Region
Östra Sverige Stockholm Stockholms län
Activity type
Private for-profit entities (excluding Higher or Secondary Education Establishments)
Other funding
€ 0,00
EATRIS ERIC
Netherlands
Net EU contribution
€ 261 250,00
Address
De Boelelaan 1118
1081 HZ Amsterdam

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Region
West-Nederland Noord-Holland Groot-Amsterdam
Activity type
Research Organisations
Other funding
€ 0,00

Partners (1)

EIDGENOESSISCHE TECHNISCHE HOCHSCHULE ZUERICH
Switzerland
Net EU contribution
€ 0,00
Address
Raemistrasse 101
8092 Zuerich

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Region
Schweiz/Suisse/Svizzera Zürich Zürich
Activity type
Higher or Secondary Education Establishments
Other funding
€ 0,00