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circRNA4DMD

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Circular RNA Therapeutics for Duchenne Muscular Dystrophy

RNA therapeutics is an emerging field explored in various types of diseases such as genetic disorders, cancer, inflammation and viral infections. Currently, most of the research focuses on the delivery of mRNA molecules that will... ver más

31/12/2027

TAU

3M€

Presupuesto del proyecto: 3M€

Líder del proyecto

TEL AVIV 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 2023-01-01

ERC-2021-ADG: ERC ADVANCED GRANTS Scope:Objectives

I+D

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Este proyecto no cuenta con búsquedas de partenariado abiertas en este momento.

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TAU

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Duración del proyecto: 59 meses Fecha Inicio: 2023-01-01
Fecha Fin: 2027-12-31

Líder del proyecto

TEL AVIV UNIVERSITY No se ha especificado una descripción o un objeto social para esta compañía.

TRL 4-5

Presupuesto del proyecto 3M€

Fecha límite de participación Sin fecha límite de participación.

Descripción del proyecto RNA therapeutics is an emerging field explored in various types of diseases such as genetic disorders, cancer, inflammation and viral infections. Currently, most of the research focuses on the delivery of mRNA molecules that will transiently express a desired protein that can replace a defective protein or manipulate gene expression in the cells. My lab was the first to show systemic, cell-specific delivery of mRNA molecules in animals. Our approach and our novel amino lipids were translated to several clinical trials in the field of infectious and monogenic diseases. In protein replacement therapy, the main hurdle of using mRNA is the relative short half life of the mRNA. To address this, I suggest an approach for long-term expression: Circular RNA (circRNA), a covalently closed loop single stranded RNA that has a significant prolonged stability compared to linear mRNA. Thus, presents an immense advantage in protein replacement therapy. Duchenne muscular dystrophy (DMD) is caused by X-linked recessive mutation in dystrophin gene, leading to lack of functional dystrophin protein. This disease affects 1:5,000 males, causes a progressive loss of muscle tissues, ultimately leading to disability and premature death. Because DMD pathology is caused by the lack of functional dystrophin, restoring the function of dystrophin is a potential therapeutic strategy. As Lipid nanoparticles (LNPs) are the most clinically advanced candidate for RNA delivery, able to entrap large RNA payloads, herein I propose an innovative multidisciplinary approach for the specific delivery of circRNA-LNPs to muscle cells that will express the dystrophin protein and replace the defective one in a DMD mouse model. The long-term expression of the circRNA will offer new hope for the treatment of monogenetic diseases such as DMD. This approach may ultimately become a novel therapeutic modality for DMD and open new avenues for implementing circRNAs for other types of genetic disorders and vaccines

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