ERD

ERDERA | EUROPEAN RARE DISEASES RESEARCH ALLIANCE

01-09-2024

-

31-08-2031

Show more information

Organisation ASSOCIATION GENETHON

ARDAT | Accelerating Research & Development for Advanced Therapies

01-11-2020

-

31-10-2025

Show more information

Organisation ASSOCIATION GENETHON

Muscle stress relief | Muscle Stress Relief: An integrated research program linking together basic research on secondary myopathies in stress states to innovative translation in applied myology.

01-03-2016

-

29-02-2020

Show more information

Organisation ASSOCIATION GENETHON

CureCN | Adeno-Associated Virus Vector-Mediated Liver Gene Therapy for Crigler-Najjar Syndrome

01-01-2018

-

30-06-2023

Show more information

Organisation ASSOCIATION GENETHON

MIROCALS | Efficacy and safety of low-dose IL-2 (ld-IL-2) as a Treg enhancer for anti-neuroinflammatory therapy in newly diagnosed Amyotrophic Lateral Sclerosis (ALS) patients

01-09-2015

-

30-09-2021

Show more information

Organisation ASSOCIATION GENETHON

SCIDNET | DevelopIng Genetic medicines for Severe Combined Immunodeficiency (SCID)

01-01-2016

-

31-12-2019

Show more information

Organisation ASSOCIATION GENETHON

UshTher | Clinical trial of gene therapy with dual AAV vectors for retinitis pigmentosa in patients with Usher syndrome type IB

01-01-2018

-

30-06-2023

Show more information

Organisation ASSOCIATION GENETHON

AAVolution | Next-generation AAV vectors for liver-directed gene therapy

01-10-2022

-

30-09-2026

Show more information

Organisation ASSOCIATION GENETHON

MYOCURE | Development of an innovative gene therapy platform to cure rare hereditary muscle disorders

01-01-2016

-

31-12-2019

Show more information

Organisation ASSOCIATION GENETHON

DMD2CURE | Correction of duplications in the DMD gene by a CRISPR/Cas9 approach

01-03-2017

-

28-02-2019

Show more information

Organisation ASSOCIATION GENETHON

GLYCODIS3 | Genetic correction of glycogen debranching enzyme deficiency in Glycogen Storage disease III: a proof of concept study

01-01-2016

-

31-12-2017

Show more information

Organisation ASSOCIATION GENETHON

MITOPOMPE | Targeting mitochondrial defects and oxidative stress in Pompe Disease: from pathogenesis to therapy

01-01-2019

-

31-12-2020

Show more information

Organisation ASSOCIATION GENETHON

Projects overview

Selected filters:

ERDERA | EUROPEAN RARE DISEASES RESEARCH ALLIANCE

ARDAT | Accelerating Research & Development for Advanced Therapies

Muscle stress relief | Muscle Stress Relief: An integrated research program linking together basic research on secondary myopathies in stress states to innovative translation in applied myology.

CureCN | Adeno-Associated Virus Vector-Mediated Liver Gene Therapy for Crigler-Najjar Syndrome

MIROCALS | Efficacy and safety of low-dose IL-2 (ld-IL-2) as a Treg enhancer for anti-neuroinflammatory therapy in newly diagnosed Amyotrophic Lateral Sclerosis (ALS) patients

SCIDNET | DevelopIng Genetic medicines for Severe Combined Immunodeficiency (SCID)

UshTher | Clinical trial of gene therapy with dual AAV vectors for retinitis pigmentosa in patients with Usher syndrome type IB

AAVolution | Next-generation AAV vectors for liver-directed gene therapy

MYOCURE | Development of an innovative gene therapy platform to cure rare hereditary muscle disorders

DMD2CURE | Correction of duplications in the DMD gene by a CRISPR/Cas9 approach

GLYCODIS3 | Genetic correction of glycogen debranching enzyme deficiency in Glycogen Storage disease III: a proof of concept study

MITOPOMPE | Targeting mitochondrial defects and oxidative stress in Pompe Disease: from pathogenesis to therapy