Hola,
¿eres nuevo aquí?

Regístrate gratis y conecta tu empresa con financiación pública, partners y proyectos.

Tengo cuenta

Regístrate

Ver video

Cor-Edit-P

Financiado

Cerrado

Cardiac open reading frame edition to study cardiomyopathies in pigs

Heart failure represents a common cause of death in European societies and is frequently based on dilated cardiomyopathy (DCM) which might be caused by mutations in cardiomyocyte genes. While no specific treatment exists, new ther... ver más

31/12/2026

TUM-MED

2M€

Presupuesto del proyecto: 2M€

Líder del proyecto

KLINIKUM RECHTS DER ISAR DER TECHNISCHEN UNIV... No se ha especificado una descripción o un objeto social para esta compañía.

TRL 4-5

Fecha límite participación Sin fecha límite de participación.

Ver 2 Participantes

Financiación concedida El organismo H2020 notifico la concesión del proyecto el día 2021-12-09

ERC-2020-ADG: ERC ADVANCED GRANT

I+D

Cerrada hace 4 años

0% 100% 100%

Información adicional privada

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

2 Participantes

TUM-MED

2.50M€ | Lider

FORMATGERIA GRANJA RINYA

422.46K€ | Participante

Conecta tu I+D

¿Tienes un proyecto y buscas un partner? Gracias a nuestro motor inteligente podemos recomendarte los mejores socios y ponerte en contacto con ellos. Te lo explicamos en este video

Proyectos interesantes

GEREMY Gene Therapy for treatment of rare inherited Arrhythmogenic... 7M€ Cerrado

Edit-hCOs Precise Genome Editing to Correct Cardiomyopathies in Human... 189K€ Cerrado

HeartGenes Next Generation Gene Therapy for the Treatment of Chronic My... 3M€ Cerrado

TRACTION Transcription Factor Gene Therapy for Bradyarrhythmias 2M€ Cerrado

DIAMONDCOR A molecular approach to treat diabetes mellitus onset depend... 1M€ Cerrado

R-FunSel In vivo functional screening via CRISPR Cas9 to systematical... 213K€ Cerrado

Duración del proyecto: 60 meses Fecha Inicio: 2021-12-09
Fecha Fin: 2026-12-31

Líder del proyecto

KLINIKUM RECHTS DER ISAR DER TECHNISCHEN UNIV... 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 Heart failure represents a common cause of death in European societies and is frequently based on dilated cardiomyopathy (DCM) which might be caused by mutations in cardiomyocyte genes. While no specific treatment exists, new therapeutic options are a major unmet clinical need. As attractive novel key approach, Cor-Edit-P will use Crispr-Cas9 based gene editing for distinct gene therapy of genetic cardiomyopathy, using pigs as a unique, clinic-related large animal model system. My lab tailored highly cardiotropic adeno-associated viral (AAV) vectors and their use in pigs in vivo, applying precise, reliable and versatile Cas9 technology. Pioneering this approach, we were able to restore significant dystrophin expression in muscles and hearts of pigs suffering from Duchenne muscle dystrophy. Exploiting unique and cutting-edge technology, Cor-edit-P aims at specifically eliminating the underlying cause of genetic DCM to improve cardiac function, reduce the risk of deadly arrhythmias and increase span and quality of life. Cor-edit-P will - generate currently lacking porcine models of genetic cardiomyopathy, using AAV-Cas9 to induce mutations in sarcomere genes, e.g. titin (TTN) and ß-myosin heavy chain (MYH7); - exercise curative Crispr-Cas9 mediated gene editing of DCM in pigs in vivo, using the PLN-R14del mutation in the phospholamban (PLN) gene as prominent example; - use human patient-derived PLN-R14del ventricular progenitor cells for gene correction ex vivo followed by transplantation of corrected cells into PLN-R14del pigs. Our approach implements a new paradigm for treating genetic cardiomyopathy and develops Crispr-Cas9 based gene therapy in pigs to foster clinical translation. Our work will influence the development of gene therapy by industry and academia and will benefit patients suffering genetic cardiomyopathy, but also further genetic diseases which are manifold prevalent in Europe.

Conecta tu I+D

Entra hoy

¿Olvidé mi contraseña?

Financiación

Empresas

CTIs/Universidades

Proyectos

Investigadores