Hola,
¿eres nuevo aquí?

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

Tengo cuenta

Regístrate

Ver video

Edit-hCOs

Financiado

Cerrado

Precise Genome Editing to Correct Cardiomyopathies in Human Cardiac Organoids

Inherited cardiomyopathies are a major cause of sudden cardiac death and are caused by mutations in genes encoding sarcomeric proteins. With an incidence of 3 per 1,000 adults within the European population, inherited cardiomyopat... ver más

30/09/2024

UNIPD

189K€

Presupuesto del proyecto: 189K€

Líder del proyecto

UNIVERSITA DEGLI STUDI DI PADOVA 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 3 Participantes

Financiación concedida El organismo HORIZON EUROPE notifico la concesión del proyecto el día 2024-09-30

HORIZON-MSCA-2021-PF-01-01: MSCA Postdoctoral Fellowships 2021 ExpectedOutcome:

I+D

Cerrada hace 3 años

0% 100% 100%

Información adicional privada

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

3 Participantes

UNIPD

188.59K€ | Lider

THE UNIVERSITY OF TEXAS SYST...

N.D. | 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

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

Cor-Edit-P Cardiac open reading frame edition to study cardiomyopathies... 2M€ Cerrado

DMD2CURE Correction of duplications in the DMD gene by a CRISPR Cas9... 185K€ Cerrado

CARDIOREPAIR Comprehensive Analysis of RBM20-induced Dilated Cardiomyopat... 4M€ Cerrado

PTA2019-017676-I Soporte para la edición genética de células mediante la tecn... 42K€ Cerrado

Duración del proyecto: 27 meses Fecha Inicio: 2022-06-14
Fecha Fin: 2024-09-30

Líder del proyecto

UNIVERSITA DEGLI STUDI DI PADOVA No se ha especificado una descripción o un objeto social para esta compañía.

TRL 4-5

Presupuesto del proyecto 189K€

Fecha límite de participación Sin fecha límite de participación.

Descripción del proyecto Inherited cardiomyopathies are a major cause of sudden cardiac death and are caused by mutations in genes encoding sarcomeric proteins. With an incidence of 3 per 1,000 adults within the European population, inherited cardiomyopathies present a substantial burden to the healthcare services of all European nations. Although a few medications can slow the progression of the disease, today there is no cure for cardiomyopathies. The CRISPR-Cas9 precision genome editing technologies, base and prime editing, can permanently edit point mutations of genes, making them ideal tools to treat cardiomyopathies. To demonstrate the efficacies and therapeutic benefits of these editors in correcting cardiomyopathy-causing mutations, it is necessary to develop robust in vitro models of mature human cardiomyocytes that recapitulate the cellular complexity of the human heart. In the Edit-hCOs project, I will generate and characterize human cardiac organoids (hCOs) harboring clinically relevant cardiomyopathy-causing mutations in the FLNC gene, which encodes for the sarcomeric protein filamin C. hCOs will be generated as three-dimensional scaffold-free cardiac microtissues using tri-cellular combinations of human induced pluripotent stem cell -derived cardiomyocytes, cardiac fibroblasts, and cardiac endothelial cells. Then, I will deploy base and prime editors to fix common FLNC gene mutations in the hCOs, and I will characterize their molecular and functional improvements following correction. In addition, I will generate humanized mouse models harboring the same mutations as patients for future studies on delivery, therapeutic safety, and efficacy of the genome editing components in vivo. Completion of the Edit-hCOs project will allow me to establish a completely innovative and ambitious research line focused on the permanent treatment of cardiomyopathies. This will establish a crucial and important pre-clinical step towards therapeutic genome editing of cardiovascular diseases.

Conecta tu I+D

Entra hoy

¿Olvidé mi contraseña?

Financiación

Empresas

CTIs/Universidades

Proyectos

Investigadores