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Gene therapy of inherited retinal diseases

Total Cost €


EC-Contrib. €






 EYEGET project word cloud

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

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Project "EYEGET" data sheet

The following table provides information about the project.


Organization address
address: VIA VARESE 16/B
city: ROMA
postcode: 185

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 Italy [IT]
 Total cost 2˙499˙564 €
 EC max contribution 2˙499˙564 € (100%)
 Programme 1. H2020-EU.1.1. (EXCELLENT SCIENCE - European Research Council (ERC))
 Code Call ERC-2015-AdG
 Funding Scheme ERC-ADG
 Starting year 2017
 Duration (year-month-day) from 2017-01-01   to  2021-12-31


Take a look of project's partnership.

# participants  country  role  EC contrib. [€] 
1    FONDAZIONE TELETHON IT (ROMA) coordinator 2˙499˙564.00


 Project objective

Inherited retinal degenerations (IRDs) are a major cause of blindness worldwide. IRD patients witness inexorable progressive vision loss as no therapy is currently available. In the last decade my group has significantly contributed to a change of this scenario by developing efficient adeno-associated viral (AAV) vectors for retinal gene therapy that are safe and effective in humans. The objective of EYEGET (EYE GEne Therapy) is to overcome some of the current major limitations in the field of retinal gene therapy to expand initial therapeutic successes to a larger number of IRDs. To achieve this, we propose to use four parallel, highly innovative and complementary approaches: i. expansion of the limited AAV cargo capacity by a novel methodology based on co-administration of multiple AAVs that reassemble in target retinal cells and reconstitute large genes; ii. targeting of frequent dominant gain-of-function mutations that cause RP using state-of-the-art AAV-mediated genome editing technologies; iii. induction of retinal cells clearance of toxic IRD products by AAV-mediated activation of autophagy and lysosomal function; iv. development of methodologies to directly convert fibroblasts to photoreceptors that can be transplanted in retinas from IRD patients with advanced PR loss and for whom in vivo gene therapy is no longer an option. We will use a combination of in vitro and in vivo state-of-the-art technologies including novel AAV vector design, high content screening of drugs that enhance AAV transduction, genome editing, and advanced in vivo retinal phenotyping to obtain proof-of-concept for each of these therapeutic strategies. The results from this study may impact the quality of life of millions of people worldwide by providing a cure based on gene and/or cell therapy for a large group of IRDs.


year authors and title journal last update
List of publications.
2018 Ivana Trapani, Alberto Auricchio
Seeing the Light after 25 Years of Retinal Gene Therapy
published pages: 669-681, ISSN: 1471-4914, DOI: 10.1016/j.molmed.2018.06.006
Trends in Molecular Medicine 24/8 2019-04-02
2018 Andrea Maddalena, Fabio Dell\'Aquila, Pia Giovannelli, Paola Tiberi, Luca Giorgio Wanderlingh, Sandro Montefusco, Patrizia Tornabene, Carolina Iodice, Feliciano Visconte, Annamaria Carissimo, Diego Luis Medina, Gabriella Castoria, Alberto Auricchio
High-Throughput Screening Identifies Kinase Inhibitors That Increase Dual Adeno-Associated Viral Vector Transduction In Vitro and in Mouse Retina
published pages: 886-901, ISSN: 1043-0342, DOI: 10.1089/hum.2017.220
Human Gene Therapy 29/8 2019-09-05
2019 Ivana Trapani, Alberto Auricchio
Has retinal gene therapy come of age? From bench to bedside and back to bench
published pages: , ISSN: 0964-6906, DOI: 10.1093/hmg/ddz130
Human Molecular Genetics 2019-09-05
2018 Andrea Maddalena, Patrizia Tornabene, Paola Tiberi, Renato Minopoli, Anna Manfredi, Margherita Mutarelli, Settimio Rossi, Francesca Simonelli, Jurgen K. Naggert, Davide Cacchiarelli, Alberto Auricchio
Triple Vectors Expand AAV Transfer Capacity in the Retina
published pages: 524-541, ISSN: 1525-0016, DOI: 10.1016/j.ymthe.2017.11.019
Molecular Therapy 26/2 2019-09-05

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