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Opendata, web and dolomites

inSight SIGNED

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

Total Cost €

EC-Contrib. €

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.

botta 118 gene retinitis transcriptional medical balanced synthetic unmet finger mar transcription enabled at human demonstrated virus al mol zf6 mussolino copy 2011 jci 24 urgent delivered retina causing efficiency models scaffold simultaneous dominant suffering endogenous med aav binding embo repressor modes vector elife commercialization dna adeno 2017 cassettes expression et 3 mutations hrho db incurable therapeutic market independent therapies pigmentosa showed treat gain animal function primary translation protein authorization social single disorders ectopic safety silencing representing rhodopsin erc inherited adrp tf replacement tfs therapeutically pi generate wild repression final clinical 2016 patients 21 discover zinc autosomal containing allelechoker mode 28 dec disorder 14 grant poc embodies toxic blindness

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 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.	IT (NAPOLI)	coordinator	107˙625.00
2	FONDAZIONE TELETHON FONDAZIONE TELETHON Organization address address: VIA VARESE 16/B city: ROMA postcode: 185 website: www.telethon.it contact info title: n.a. name: n.a. surname: n.a. function: n.a. email: n.a. telephone: n.a. fax: n.a.	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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