A5003912397- Hemoglobinopathies and Related Disorders
- Virus-based gene therapy research
- Prenatal Screening and Diagnostics
- Hematopoietic Stem Cell Transplantation
- CRISPR and Genetic Engineering
- CAR-T cell therapy research
- Parvovirus B19 Infection Studies
- Acute Lymphoblastic Leukemia research
- Acute Myeloid Leukemia Research
- Iron Metabolism and Disorders
- Immunodeficiency and Autoimmune Disorders
- Cancer Genomics and Diagnostics
- Single-cell and spatial transcriptomics
- Pluripotent Stem Cells Research
- Erythrocyte Function and Pathophysiology
- Cytomegalovirus and herpesvirus research
- T-cell and Retrovirus Studies
- RNA Research and Splicing
- Protein Tyrosine Phosphatases
- Immune Cell Function and Interaction
- Myeloproliferative Neoplasms: Diagnosis and Treatment
- Blood groups and transfusion
- Mesenchymal stem cell research
- DNA Repair Mechanisms
- Liver physiology and pathology
Assistance Publique – Hôpitaux de Paris
2016-2023
Hôpital Necker-Enfants Malades
2016-2023
Sorbonne Université
2022
Pitié-Salpêtrière Hospital
2022
Inserm
2016-2021
Université Paris Cité
2018-2021
Institut des Maladies Génétiques Imagine
2016-2021
Hôpitaux Universitaires Paris-Ouest
2017-2020
Centre d'Investigation Clinique Biothérapies Necker
2018-2019
Lucile Packard Children's Hospital
2018
Sickle cell disease results from a homozygous missense mutation in the β-globin gene that causes polymerization of hemoglobin S. Gene therapy for patients with this disorder is complicated by complex cellular abnormalities and challenges achieving effective, persistent inhibition We describe our first patient treated lentiviral vector-mediated addition an antisickling into autologous hematopoietic stem cells. Adverse events were consistent busulfan conditioning. Fifteen months after...
Donor availability and transplantation-related risks limit the broad use of allogeneic hematopoietic-cell transplantation in patients with transfusion-dependent β-thalassemia. After previously establishing that lentiviral transfer a marked β-globin (βA-T87Q) gene could substitute for long-term red-cell transfusions patient β-thalassemia, we wanted to evaluate safety efficacy such therapy
Abstract 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 ) for eight patients WAS having undergone phase lentiviral vector-based trials (nos. NCT01347346 and NCT01347242 ), focus on thrombocytopenia...
Sickle cell disease is characterized by chronic anemia and vaso-occlusive crises, which eventually lead to multi-organ damage premature death. Hematopoietic stem transplantation the only curative treatment but it limited toxicity poor availability of HLA-compatible donors. A gene therapy approach based on autologous lentiviral-corrected hematopoietic progenitor cells was shown be efficacious in one patient. However, alterations bone marrow environment properties red blood hamper harvesting...
Summary Relapse remains the leading cause of treatment failure in children with acute lymphoblastic leukaemia ( ALL ) undergoing allogeneic haematopoietic stem cell transplantation HSCT ). We retrospectively investigated prognostic role minimal residual disease MRD before and after 119 transplanted complete remission CR was measured by polymerase chain reaction bone marrow samples collected pre‐ during first third trimesters (post‐ 1 post‐ 3). The overall event‐free survival EFS 50%....
Seventy-five percent of patients with beta thalassemia (β-thalassemia) do not have human leukocyte antigen-matched siblings and until recently had no access to a curative treatment. Gene therapy is promising treatment that can be proposed these patients. This study estimates its cost efficacy. In monocentric retrospective cost-efficacy analysis, this compared the two-year outcomes costs β-thalassemia treated by gene hematopoietic stem-cell transplantation (HSCT). Grade III grade IV...
Although studies of mixed chimerism following hematopoietic stem cell transplantation in patients with sickle disease (SCD) may provide insights into the engraftment needed to correct and immunological reconstitution, an extensive multilineage analysis is lacking. We analyzed simultaneously peripheral erythroid granulomonocytic precursors/progenitors, highly purified B T lymphocytes, monocytes, granulocytes red blood cells (RBC). Thirty-four ≥12 months follow-up were included. A selective...
Autologous transplantation of hematopoietic stem cells transduced with a lentiviral vector (LV) expressing an anti-sickling HBB variant is potential treatment for sickle cell disease (SCD). With clinical trial as our ultimate goal, we generated LV constructs containing transgene (HBBAS3), minimal promoter, and different combinations DNase I hypersensitive sites (HSs) from the locus control region (LCR). Hematopoietic progenitor (HSPCs) SCD patients were LVs either HS2 HS3 (β-AS3) or HS2,...
The association of the SH3 (Src homology 3) domain SFKs family kinases) with protein partners bearing proline-rich motifs has been implicated in regulation SFK activity, and described as a possible mechanism relocalization to subcellular compartments. We demonstrate present study for first time that p13, an accessory encoded by HTLV-1 (human T-cell leukaemia virus type 1), binds via its C-terminal motif, forming stable heterodimer translocates mitochondria virtue N-terminal mitochondrial...
Congenital erythropoietic porphyria (CEP) is a rare disease characterized by erosive photosensitivity and chronic hemolysis due to defect of the enzyme uroporphyrinogen-III-synthase (UROS). To date, hematopoietic stem cell transplantation (HSCT) only curative therapy for devastating early severe form disease. We describe 6 patients with CEP treated HSCT (3 them twice after failure first graft) between 1994 2016 in our center, including 2 very living more than 20 years ago. Four are doing...
Although the risk of developing lymphoma has decreased in highly active antiretroviral therapy era, this cancer remains major cause mortality HIV-infected patients. Autologous hematopoietic stem cell transplantation (ASCT) outcome does not differ for versus HIV-uninfected We propose to develop a new treatment HIV-associated high-risk based on autologous two genetically modified products: CD4+ T lymphocytes and CD34+ cells (HSPCs). The will be transduced ex vivo with Cal-1 lentiviral vector...
A 4‐year‐old male with the diagnosis of T‐cell acute lymphoblastic leukemia (T‐ALL) relapsed after 19 months an myeloid (AML). Immunoglobulin and receptor gene rearrangements analyses reveal that both leukemias were rearranged a clonal relationship between them. Comparative genomic hybridization (Array‐CGH) whole‐exome sequencing samples suggest this may have originated from common T/myeloid progenitor. The presence homozygous deletion p16/INK4A, p14/ARF, p15/INK4B, heterozygous WT1 locus...
Abstract Background Autologous and allogeneic hematopoietic stem cell transplantation of cytokine‐mobilized peripheral blood cells (PBSCs) is increasingly used to treat patients with hematologic disorders. Different types vascular access have been exploited for the apheresis procedure, including veins (PV) central venous catheter (CVC). In some cases, PV unavailable. There are few published data on efficiency quality harvesting different access. This study brings out complications morbidity...