News

Anne Galy receives the WAS Foundation Callahan Award for her work on Wiskott-Aldrich syndrome

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Sickle-cell anemia: a project involving the Gene editing team has just secured European funding

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Gene Therapies Continue to Emerge as Effective Treatments for Rare Diseases, But Complex Challenges Remain

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Five post-doctoral positions open at Genethon, a leader in gene therapy for rare diseases

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From 2021, a seminal year for Genethon, to 2022, a year of challenges

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Myotubular myopathy: “Today my son Jules is able to stand”

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Pierre, suffering from limb girdle muscular dystrophy: “For me, a cure would mean no more losing”

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Spinal muscular atrophy: for Victoire’s mom, treatment was a magical moment

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Preliminary results of clinical trial for rare liver disease Crigler-Najjar syndrome presented at ESGCT congress

After a first presentation at the International Liver Congress last June, new preliminary results have just been presented at the European Society of Geneand Cell Therapy (ESGCT) congress by Dr d’Antiga, one of the investigators of the trial (Bergamo, Italy). Based on initial observations, the drug candidate is well tolerated and the first… (read more)

Cholesterol Metabolism: A Potential Therapeutic Target in Duchenne Muscular Dystrophy

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Genethon’s teams present their progress at the ASGCT meeting

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Genethon announces First Patient dosed in Clinical Trial of Investigational Gene therapy GNT 0004 for Duchenne Muscular Dystrophy

Genethon announces First Patient dosed in Clinical Trial of Investigational Gene therapy GNT 0004 for Duchenne Muscular Dystrophy

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Genethon, 30 years of pioneering research and innovation in treating rare diseases

On February 28, International Rare Disease Day will put the spotlight on the rare diseases community throughout the world. In France, Genethon, a unique not-for-profit laboratory, innovates to develop gene therapy treatments for rare diseases.

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Genethon and WhiteLab Genomics join forces to enhance gene therapy through artificial intelligence

WhiteLab Genomics, a specialist in artificial intelligence applied to gene and cell therapies, has signed a partnership agreement with Genethon, a pioneering research center in the field of gene therapy. The alliance will harness the power of artificial intelligence to accelerate development of innovative gene therapies.

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Genethon is delighted about the launch of a gene therapy clinical trial for late-onset Pompe disease

Genethon is delighted about the launch of a gene therapy clinical trial for late-onset Pompe disease, led by the company Spark Therapeutics, using technologies developed at Genethon.

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