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Ivana Trapani

Assistant Investigator, TIGEM

Research Associate, Department of Advanced Biomedicine, University of Naples "Federico II", Italy


Gene-dependent and -independent approaches for therapy of retinal diseases which are inaccessible to conventional gene therapy

Inherited retinal diseases (IRDs) are a major cause of blindness worldwide. Gene therapy represents an attractive therapeutic option for treatment of these diseases, and vectors based on adeno-associated viruses (AAVs) are considered the gold-standard for retinal cells transduction. As a confirmation of this, the first gene therapy product to receive a landmark market authorization in both the USA and Europe for treatment of a common form of IRD is based on an AAV vector. However, many tissue- and disease-related challenges in the field of retinal gene therapy are still unsolved, including the possibility to efficiently transfer large genes (>5 kb) via AAVs. Therefore, strategies to overcome such hurdles are being developed at a rapid pace.
Our lab was set up in January 2021, we plan to focus our attention on developing AAV-based approaches which can tackle retinal diseases at multiple levels. We will explore novel gene replacement and genome editing approaches, which overcome the limitations of the existing ones, to directly provide a functional copy of diseased genes. In parallel, we will develop gene-independent gene therapy approaches to target key pathways which are altered in IRDs. We will use cutting-edge in vitro models, three-dimensional retinal organoids, and in vivo models. While our studies will be focused on testing these new approaches in common forms of IRDs due to mutations in large genes, such as Retinitis Pigmentosa and Stargardt disease, the overall goal of our project is to provide insights to treat additional common inherited and acquired retinal diseases.

Ph.D. Student: Eugenio Pugni
Lab Manager:

Tornabene P., Trapani I. (2020). Can Adeno-Associated Viral Vectors Deliver Effectively Large Genes? Hum Gene Ther. Jan;31(1-2):47-56

Tornabene P.*, Trapani I.*, Minopoli R., Centrulo M., Lupo M., de Simone S., Tiberi P., Dell'Aquila F., Marrocco E., Iodice C., Iuliano A., Gesualdo C., Rossi S., Giaquinto L., Albert S., Hoyng C.B., Polishchuk E., Cremers F.P.M., Surace E.M., Simonelli F., De Matteis M.A., Polishchuk R., Auricchio A. (2019). Intein-mediated protein trans-splicing expands adeno-associated virus transfer capacity in the retina. Sci Transl Med. May 15;11(492). pii: eaav4523

Trapani I.*, A.Auricchio* (2018). Seeing the light after 25 years of retinal gene therapy. Trends Mol Med. Aug;24(8):669-681. *co-corresponding author

Trapani I., Toriello E, de Simone S, Colella P, Iodice C, Polishchuk EV, Sommella A, Colecchi L, Rossi S, Simonelli F, Giunti M, Bacci ML, Polishchuk RS, Auricchio A. (2015). Improved dual AAV vectors with reduced expression of truncated proteins are safe and effective in the retina of a mouse model of Stargardt disease. Hum Mol Genet. Dec 1;24(23):6811-25.

Trapani I., Colella P., Sommella A., Iodice C., Cesi G., De Simone S.,  Marrocco E., Rossi S., Giunti M., Palfi A., Jane Farrar G., Polishchuk R. and Auricchio A. (2014). Effective delivery of large genes to the retina by dual AAV vectors. EMBO Mol Med. Feb;6(2):194-211.

Ivana Trapani PhD

Molecular Therapy

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