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inSight SIGNED

Moving a novel gene therapy paradigm to treat blindness to the market

Total Cost €

0

EC-Contrib. €

0

Partnership

0

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 inSight project word cloud

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

final    repressor    2011    safety    virus    translation    synthetic    pigmentosa    vector    discover    28    urgent    mussolino    binding    silencing    incurable    blindness    scaffold    showed    demonstrated       grant    animal    mol    118    retina    enabled    adrp    causing    cassettes    zinc    dna    therapeutic    embo    disorders    ectopic    retinitis    disorder    gain    2016    replacement    generate    botta    2017    14    authorization    al    therapeutically    unmet    mar    modes    wild    treat    pi    hrho    endogenous    zf6    mutations    mode    representing    poc    jci    embodies    social    inherited    protein    elife    balanced    patients    therapies    delivered    tf    gene    dec    commercialization    21    medical    at    clinical    adeno    human    aav    primary    dominant    finger    autosomal    single    med    repression    suffering    independent    tfs    copy    models    market    et    db    24    erc    allelechoker    containing    efficiency    transcription    expression    function    rhodopsin    simultaneous    toxic    transcriptional   

Project "inSight" data sheet

The following table provides information about the project.

Coordinator
UNIVERSITA DEGLI STUDI DI NAPOLI FEDERICO II 

Organization address
address: CORSO UMBERTO I, 40
city: NAPOLI
postcode: 80138
website: www.unina.it

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 150˙000 €
 EC max contribution 150˙000 € (100%)
 Programme 1. H2020-EU.1.1. (EXCELLENT SCIENCE - European Research Council (ERC))
 Code Call ERC-2018-PoC
 Funding Scheme ERC-POC
 Starting year 2019
 Duration (year-month-day) from 2019-06-01   to  2020-11-30

 Partnership

Take a look of project's partnership.

# participants  country  role  EC contrib. [€] 
1    UNIVERSITA DEGLI STUDI DI NAPOLI FEDERICO II IT (NAPOLI) coordinator 107˙625.00
2    FONDAZIONE TELETHON IT (ROMA) participant 42˙375.00

Map

 Project objective

At present therapies for inherited dominant disorders are not available, thus representing an urgent unmet medical and social need. The ERC Grant ALLELECHOKER enabled the PI to discover three modes to generate transcriptional repression by transcription factors (TFs) based on Zinc-finger scaffold: i- synthetic transcription factor (TF; Mussolino et al., EMBO Mol. Med. 2011 Mar;3(3):118-28; Botta S. et al. Elife. 2016 Mar 14;5), ii- synthetic DNA-binding protein (Botta S. et al. Elife. 2016 Mar 14;5) and iii- the ectopic expression of an endogenous TF (Botta S, et al. JCI Insight. 2017 Dec 21;2(24). Thus, the PI demonstrated that transcriptional repression by TFs embodies a novel therapeutically effective mode to treat the toxic effects of gain-of-function mutations causing incurable inherited dominant disorders. In particular, mutations in the RHODOPSIN gene can cause the blindness disorder autosomal dominant retinitis pigmentosa (adRP). The PI demonstrated safety and efficiency of RHODOPSIN gene transcriptional silencing in pre-clinical animal models by a specific synthetic transcriptional repressor (ZF6-DB) delivered to the retina by an adeno-associated virus (AAV) vector (AAV-ZF6-DB). Furthermore, within the ERC Grant ALLELECHOKER the PI showed that a single AAV vector containing two independent expression cassettes (AAV-ZF6-DB-hRHO), enables balanced silencing of RHODOPSIN by ZF6-DB and its simultaneous replacement with a human wild-type copy of the RHODOPSIN gene. The primary objective of the inSight ERC-PoC grant is to support AAV-ZF6-DB-hRHO clinical translation, with the final goal of bringing this therapeutic to patients suffering adRP through market authorization and commercialization.

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The information about "INSIGHT" are provided by the European Opendata Portal: CORDIS opendata.

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