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Long-term efficacy for an immunodeficiency gene therapy

The Genethon research unit Integrare and its French and British partners have demonstrated the long-term efficacy of a gene therapy for Wiskott-Aldrich syndrome, an immune deficiency disorder. The clinical trial, published in Nature Medicine, monitored eight patients over an average of seven years.
Published in Nature Medecine 2022 >
INtegrare / Généthon - Efficacité à long terme de la thérapie génique d’un déficit immunitaire INtegrare / Généthon - Efficacité à long terme de la thérapie génique d’un déficit immunitaire

Work done by Anne Galy’s Integrare (Integrated genetic approaches and new therapies for rare diseases; Inserm, University of Évry-Paris Saclay, Genethon) team has resulted in an ex vivo* gene therapy for the treatment of Wiskott-Aldrich syndrome, a rare immune deficiency. Genethon conceived the vector, produced the clinical batches and designed an international clinical trial launched in 2010 at the Necker Children’s Hospital in Paris and at two hospitals in London, England.

Wiskott-Aldrich syndrome (WAS) is a rare genetic disease that severely alters the immune system of children. It causes bleeding, serious recurrent infections, severe eczema, autoimmune reactions and even cancers. The syndrome is caused by a mutation in the WAS gene, resulting in alterations to leukocyte function. Gene therapies thus hold the greatest promise for efficacious treatments. Integrare developed a lentiviral vector able to bring a functional WAS gene to the blood’s stem cells.

Earlier results published in 2015 showed a reestablishment of immune system function and improved clinical states in the first patients to have received the treatment. The recent article published 24 January 2022 in Nature Medicine reports the conclusive follow-up results from a long-term safety and efficacy trial. For it, eight patients aged 8 months to 30 years at the time of treatment were monitored over four to nine years.

Resolution of the main symptoms of the disease, such as recurrent severe infections or eczema, was observed for all of the patients. T lymphocyte function, which is vital for immune response, was completely restored. Bleeding and signs of auto-immune dysfunction also diminished significantly in all the patients and platelet counts had returned to normal in three of them. These positive effects were observed in the adult patients as well, confirming treatment efficacy in that population.

Furthermore, the authors observed no adverse effects in their study. And finally, using DNA sequencing, the team was able to determine the integration sites of the “gene-drug.” The results of this long-term follow-up study demonstrate the interest of lentiviral vectors for the safe and stable introduction of corrective genes.


Long-term safety and efficacy of lentiviral hematopoietic stem/progenitor cell gene therapy for Wiskott–Aldrich syndrome.

  • * Ex vivo gene therapy

    involves taking cells from a patient, cultivating them in vitro, transforming them genetically then reinjecting them back into the patient. This approach has the major advantage of reducing the risk of immune reactions because the viral vector is kept out of the organism and the cells are those of the patient.
    Also, ex vivo gene therapy can correct stem cells. Because these latter are able to multiply indefinitely, the therapeutic effect can remain active over time.
    Hematopoietic stem and progenitor cells are the starting points for all of the various cells in the blood (including those involved in the immune system) and compatible with ex vivo gene therapy techniques. They can be collected from the bone marrow and reinjected thereafter intravenously. Genetically modified progenitor cells can, once reintroduced into the organism, lead to the constitution of a corrected hematopoietic cell line.

Article posted on 18 February 2022



Alexis Biton and Paul Carouen - Genopole - visiting the Research Triangle Park (USA) accompanied by 3 Genopole's companies

Genopole strengthens its ties with the Research Triangle Park

From Monday March 20 to Thursday March 24, Genopole's Partnership & Business Development team visited the Research Triangle Park biopark in North Carolina to strengthen scientific and industrial collaborations.

l’équipe « Dystrophies musculaires progressives » du laboratoire Généthon

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A trio of genome sites associated with arterial hypertension

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Genoscope - CNS - Genopole's laboratory

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Equipe Sysfate de l'UMR 8030 - projet T-Fitness

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Couverture de la revue Cell Genomics - Le plancton

A feat in the study of planktonic life

A Genoscope research team was part of a pioneering study in environmental genomics. The researchers were able to at least partially reconstitute the genomes of close to 700 eukaryote species.

Logos Istem & Généthon

A two-drug combination to treat limb-girdle muscular dystrophies and cystic fibrosis

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Christophe Lanneau - Research & Platforms Department Director

News from the human genome

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CNRGH - Consequences of phthalate exposure during pregnancy

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Jörg Tost and his team of the National Center of Human Genomics Research (CNRGH; CEA) were part of a large collaborative study aimed at determining the genome-wide correlation between phthalate exposure during pregnancy and methylation of placental DNA.

I-Stem, IPSEN & LMN recreate the human muscle-nerve system

A cellular model to recreate the human muscle-nerve system

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Genoscope - CNS - Genopole's laboratory

Filling the gaps in the banana’s genome

Des chercheurs de Genoscope en collaboration avec une équipe du Cirad sont parvenus à reconstituer la séquence intégrale de chromosomes de bananier.

traitement des plaies diabétiques issu de peau et d’écailles de poissons

CERITD: Treating deep wounds with fish skin

CERITD and the South Île-de-France Medical Center are testing an innovative, natural fish-skin-based treatment for diabetic foot ulcers.


Sysfate Team: A tool to digitize organs

Sysfate at Genoscope has combined image analysis and genomics to create MULTILAYER, a tool to digitize biological tissues and organs.

muscle fiber

Signs of myopathy detected before muscle formation

An I-Stem team has revealed very early embryonic molecular signs of Duchenne muscular dystrophy occurring upstream of muscle development itself

Neurons representation - Cells affected by CMT

Gene therapy for Charcot-Marie-Tooth disease makes headway

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LAST - Makeup by Global Bioenergies

Global Bioenergies launches its makeup brand

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NEB France celebrate 10 years @ Genopole

New England Biolabs France Celebrates 10 Years!

Its French affiliate arrived at Genopole in 2011 and to mark its 10th anniversary in France, the company invites you to participate in monthly activities to be held throughout 2021.

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With the support from
Région île de France