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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

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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.

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

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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