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BrainCRISPR

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In Vivo CRISPR-Based Nanoplatform for Gene Editing: A New Disruptive Avenue for...

In Vivo CRISPR-Based Nanoplatform for Gene Editing: A New Disruptive Avenue for Non-Invasive Treatment of Genetic Brain Diseases Genetic brain diseases are among the most devastating and fatal diseases, typically having only palliative treatments and no cure. The revolutionary CRISPR/Cas gene editing technology provides a new horizon and enormous potential... ver más

31/05/2027

BIU

2M€

Presupuesto del proyecto: 2M€

Líder del proyecto

BAR ILAN UNIVERSITY No se ha especificado una descripción o un objeto social para esta compañía.

TRL 4-5

Fecha límite participación Sin fecha límite de participación.

Financiación concedida El organismo HORIZON EUROPE notifico la concesión del proyecto el día 2022-06-01

ERC-2021-COG: ERC CONSOLIDATOR GRANTS Scope:Objectives

I+D

Cerrada hace 3 años

0% 100%

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No hay información privada compartida para este proyecto. Habla con el coordinador.

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Duración del proyecto: 59 meses Fecha Inicio: 2022-06-01
Fecha Fin: 2027-05-31

Líder del proyecto

BAR ILAN UNIVERSITY No se ha especificado una descripción o un objeto social para esta compañía.

TRL 4-5

Presupuesto del proyecto 2M€

Fecha límite de participación Sin fecha límite de participación.

Descripción del proyecto Genetic brain diseases are among the most devastating and fatal diseases, typically having only palliative treatments and no cure. The revolutionary CRISPR/Cas gene editing technology provides a new horizon and enormous potential for treating such diseases. However, efficient and safe delivery of CRISPR machinery to diseased cells within the brain is one of the greatest challenges in medicine today. Here, I plan to expand far beyond the state-of-the-art, and propose a game-changer approach for this unmet need: A breakthrough nanoplatform, which will transform CRISPR into a clinically-relevant, non-invasive technology, enabling therapeutic genome editing in the brain. Our proof-of-concept results serve as the baseline of this pioneering research project, revealing the exceptional capabilities of insulin as a key to overcoming formidable brain and cell barriers. We will harness these unique abilities within the novel nanoplatform, and shuttle CRISPR machinery across the blood-brain barrier, transport it into deep brain regions, and mediate its successful entry into specific diseased brain cells, leading to highly effective gene editing. The nanoplatform will be designed to meet key criteria for non-invasive, safe and efficient delivery of CRISPR to the brain, while conferring a high degree of modularity and compositional heterogeneity - thus providing both universal and patient-specific components. The nanoplatform will be thoroughly investigated in primary brain cells, 3D organoids, and case-studies of monogenic brain disease models. This comprehensive research will culminate with a universal and modular BrainCRISPR nanoplatform, and delineate design principles for its precise tailoring to specific needs of different brain diseases. Overall, this research will provide in-depth fundamental knowledge and have a transformative effect on applying CRISPR in brain, whilst opening a wide array of possibilities with broader impact on genetic brain therapy and beyond.

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