Actualités : Presse, Congrès & Publications scientifiques

Publications scientifiques 1 avril 2025

Severe inflammation and lineage skewing are associated with poor engraftment of engineered hematopoietic stem cells in patients with sickle cell disease.

In sickle cell disease (SCD), the β6 substitution in the β-globin leads to red blood cell sickling. The transplantation of autologous, genetically modified hematopoietic stem and progenitor cells (HSPCs) is a promising treatment option for patients with SCD. We completed [...]

ActualitésCongrès 21 mars 2025

SFTGC – Bordeaux (France)

Dr. Jean-Sébastien Diana a présenté les travaux suivants : Gene therapy for familial hemophagocytic lymphohistiocytosis related to Munc 13-4 deficiency Boris Bessot a présenté les travaux suivants : Gene therapy for Artemis-SCID patients: preliminary results of the French ARTEGENE phase I/II [...]

Publications scientifiques 5 juillet 2024

Restoration of T and B Cell Differentiation after RAG1 Gene Transfer in Human RAG1 Defective Hematopoietic Stem Cells.

Recombinase-activating gene (RAG)-deficient SCID patients lack B and T lymphocytes due to the inability to rearrange immunoglobulin and T cell receptor genes. The two genes act as a required dimer to initiate gene recombination. Gene therapy is a valid treatment [...]

Publications scientifiques 25 août 2023

Sickle Cell Disease: From Genetics to Curative Approaches.

Sickle cell disease (SCD) is a monogenic blood disease caused by a point mutation in the gene coding for β-globin. The abnormal hemoglobin [sickle hemoglobin (HbS)] polymerizes under low-oxygen conditions and causes red blood cells to sickle. The clinical presentation [...]

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

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 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 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 [...]

Biotherapy for Innovation in Orphan Disease

Biotherapy for Innovation in Orphan Disease

Severe inflammation and lineage skewing are associated with poor engraftment of engineered hematopoietic stem cells in patients with sickle cell disease.

SFTGC – Bordeaux (France)

Restoration of T and B Cell Differentiation after RAG1 Gene Transfer in Human RAG1 Defective Hematopoietic Stem Cells.

Sickle Cell Disease: From Genetics to Curative Approaches.

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

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.

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

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