Project description
Novel adeno-associated vectors for liver gene transfer
Recombinant adeno-associated vectors (AAV) achieved remarkable success in recent clinical trials for liver gene transfer. However, several hurdles prevent expansion of AAV-mediated therapy to all patients, including transient AAV-mediated expression in proliferating hepatocytes due to episomal AAV genome dilution, hepatotoxicity and immune response against AAVs, and pre-existing immunity to AAV capsids. The EU-funded AAVolution project proposes to find novel small Cas nucleases for in vivo AAV-mediated genome editing with self-replicating episomal AAVs. The aim is to avoid transgene dilution, create synthetic AAVs with reduced toxicity and develop technologies to overcome pre-existing immunity to AAVs. The novel tool set will enable broad expansion of AAV-mediated gene therapy for indications beyond rare diseases.
Fields of science
Programme(s)
- HORIZON.3.1 - The European Innovation Council (EIC) Main Programme
Topic(s)
Funding Scheme
HORIZON-AG - HORIZON Action Grant Budget-BasedCoordinator
00185 Roma
Italy
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Participants (8)
Legal entity other than a subcontractor which is affiliated or legally linked to a participant. The entity carries out work under the conditions laid down in the Grant Agreement, supplies goods or provides services for the action, but did not sign the Grant Agreement. A third party abides by the rules applicable to its related participant under the Grant Agreement with regard to eligibility of costs and control of expenditure.
80078 Pozzuoli
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The organization defined itself as SME (small and medium-sized enterprise) at the time the Grant Agreement was signed.
Legal entity other than a subcontractor which is affiliated or legally linked to a participant. The entity carries out work under the conditions laid down in the Grant Agreement, supplies goods or provides services for the action, but did not sign the Grant Agreement. A third party abides by the rules applicable to its related participant under the Grant Agreement with regard to eligibility of costs and control of expenditure.
80078 Pozzuoli
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The organization defined itself as SME (small and medium-sized enterprise) at the time the Grant Agreement was signed.
91002 Evry
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38122 Trento
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31008 Pamplona
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30625 Hannover
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10115 Berlin
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The organization defined itself as SME (small and medium-sized enterprise) at the time the Grant Agreement was signed.
80138 Napoli
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