AAV-mediated Gene Therapy for Haemophilia A

Projektinformationen

Genophilia

ID Finanzhilfevereinbarung: 326635

Projekt abgeschlossen

Startdatum 1 März 2013

Enddatum 31 August 2014

Finanziert unter

• Specific programme "People" implementing the Seventh Framework Programme of the European Community for research, technological development and demonstration activities (2007 to 2013)

Gesamtkosten € 173 462,40

EU-Beitrag € 173 462,40

Koordiniert durch

UNIVERSITY COLLEGE LONDON

Ziel

Haemophilia is a group of hereditary genetic disorders that impair the body's ability to maintain normal blood coagulation. Haemophilia A (clotting factor VIII deficiency) is the most common form of the disorder, occurring in ~1 in 5,000 males in Europe. Although different gene transfer strategies for factor replacement have been evaluated, several properties make adeno-associated virus (AAV) vectors the most promising. Most importantly, AAV directs long-term transgene expression from non-dividing cells in animal models after a single administration of vector. In addition, AAV has an excellent safety profile. Unlike other vectors of viral origin, AAV has never been associated with any human disease and is naturally replication deficient.
The overriding goal of the proposal is to improve the probability that AAV-mediated FVIII gene transfer can be used to successfully treat patients with haemophilia A. To achieve this goal, the Fellow will 1) establish a simple and efficient GMP compliant manufacturing platform for rAAV-HLP-codop-hFVIII pseudotyped with serotype 8 capsid2) assess the safety and efficacy of this AAV vector encoding a novel, potent FVIII-variant in mice.

Wissenschaftliches Gebiet

• medical and health sciences › medical biotechnology › genetic engineering › gene therapy
• natural sciences › biological sciences › microbiology › virology

Programm/Programme

• FP7-PEOPLE - Specific programme "People" implementing the Seventh Framework Programme of the European Community for research, technological development and demonstration activities (2007 to 2013)

Thema/Themen

• FP7-PEOPLE-2012-IEF - Marie-Curie Action: "Intra-European fellowships for career development"

Aufforderung zur Vorschlagseinreichung

FP7-PEOPLE-2012-IEF
Andere Projekte für diesen Aufruf anzeigen

Finanzierungsplan

MC-IEF - Intra-European Fellowships (IEF)

Koordinator

UNIVERSITY COLLEGE LONDON

Kontakt Verwaltung

Greta Borg-Carbott (Ms.)

• EU-Beitrag: € 173 462,40
• Adresse: GOWER STREET ; WC1E 6BT LONDON ;
• Aktivitätstyp: Higher or Secondary Education Establishments
• Gesamtkosten: Keine Daten