Actualités : Presse, Congrès & Publications scientifiques

Publications scientifiques 21 février 2023

Severe hematopoietic stem cell inflammation compromises chronic granulomatous disease gene therapy.

X-linked chronic granulomatous disease (CGD) is associated with defective phagocytosis, life-threatening infections, and inflammatory complications. We performed a clinical trial of lentivirus-based gene therapy in four patients (NCT02757911). Two patients show stable engraftment and clinical benefits, whereas the other two [...]

ActualitésPresse 7 février 2023

Granulomatose septique chronique : des bio-marqueurs pour prédire l’efficacité de la thérapie génique

Les services cliniques de l’Hôpital Necker-Enfants malades AP-HP en collaboration étroite avec des équipes de l'AP-HP, de l'Inserm et d'Université Paris Cité, au sein de l’Institut Imagine ont mis en évidence 51 biomarqueurs qui permettraient de prédire le succès d’une [...]

Publications scientifiques 1 janvier 2022

Long-term outcomes of lentiviral gene therapy for the β-hemoglobinopathies: the HGB-205 trial.

Sickle cell disease (SCD) and transfusion-dependent β-thalassemia (TDT) are the most prevalent monogenic disorders worldwide. Trial HGB-205 ( NCT02151526 ) aimed at evaluating gene therapy by autologous CD34 cells transduced ex vivo with lentiviral vector BB305 that encodes the anti-sickling [...]

Publications scientifiques 1 janvier 2022

A new step in understanding stem cell mobilization in patients with Fanconi anemia: A bridge to gene therapy.

Fanconi anemia (FA) is an inherited disorder characterized clinically by congenital abnormalities, progressive bone marrow failure (BMF), and a predisposition to malignancy. Gene therapy (GT) of FA, via the infusion of gene-corrected peripheral blood (PB) autologous hematopoietic stem cells (HSCs), [...]

Publications scientifiques 1 janvier 2022

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

Patients with Wiskott-Aldrich syndrome (WAS) lacking a human leukocyte antigen-matched donor may benefit from gene therapy through the provision of gene-corrected, autologous hematopoietic stem/progenitor cells. Here, we present comprehensive, long-term follow-up results (median follow-up, 7.6 years) (phase I/II trial no. NCT02333760 [...]

Publications scientifiques 29 avril 2021

A combination of cyclophosphamide and interleukin-2 allows CD4+ T cells converted to Tregs to control scurfy syndrome.

Immunodysregulation, polyendocrinopathy, enteropathy, X-linked (IPEX) syndrome is caused by mutations in forkhead box P3 (FOXP3), which lead to the loss of function of regulatory T cells (Tregs) and the development of autoimmune manifestations early in life. The selective induction of [...]

Publications scientifiques 1 janvier 2021

Bayesian Modeling Immune Reconstitution Apply to CD34+ Selected Stem Cell Transplantation for Severe Combined Immunodeficiency.

Severe combined immunodeficiencies (SCIDs) correspond to the most severe form of primary immunodeficiency. Allogeneic hematopoietic stem cell transplantation (HSCT) and gene therapy are curative treatments, depending on the donor's availability and molecular diagnostics. A partially human leukocyte antigen (HLA)-compatible donor [...]

Biotherapy for Innovation in Orphan Disease

Biotherapy for Innovation in Orphan Disease

Severe hematopoietic stem cell inflammation compromises chronic granulomatous disease gene therapy.

Granulomatose septique chronique : des bio-marqueurs pour prédire l’efficacité de la thérapie génique

Long-term outcomes of lentiviral gene therapy for the β-hemoglobinopathies: the HGB-205 trial.

A new step in understanding stem cell mobilization in patients with Fanconi anemia: A bridge to gene therapy.

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

A combination of cyclophosphamide and interleukin-2 allows CD4+ T cells converted to Tregs to control scurfy syndrome.

Bayesian Modeling Immune Reconstitution Apply to CD34+ Selected Stem Cell Transplantation for Severe Combined Immunodeficiency.