A5039834600- Virus-based gene therapy research
- CRISPR and Genetic Engineering
- Neurogenesis and neuroplasticity mechanisms
- Pluripotent Stem Cells Research
- Hemoglobinopathies and Related Disorders
- Neuroinflammation and Neurodegeneration Mechanisms
- Nerve injury and regeneration
- Viral gastroenteritis research and epidemiology
- Neurogenetic and Muscular Disorders Research
- Glioma Diagnosis and Treatment
- Parvovirus B19 Infection Studies
- Signaling Pathways in Disease
- Xenotransplantation and immune response
- Genetics and Neurodevelopmental Disorders
- Neuroscience and Neural Engineering
- Autism Spectrum Disorder Research
- Nuclear Receptors and Signaling
- Erythrocyte Function and Pathophysiology
- Axon Guidance and Neuronal Signaling
- RNA Interference and Gene Delivery
Inserm
2011-2024
Sorbonne Université
2020-2024
Institut du Cerveau
2020-2024
Centre National de la Recherche Scientifique
2022-2024
Université Paris-Saclay
2011-2022
Université Paris-Sud
2022
Assistance Publique – Hôpitaux de Paris
2022
Pitié-Salpêtrière Hospital
2022
CEA Paris-Saclay - Etablissement de Fontenay-aux-roses
2010-2017
Commissariat à l'Énergie Atomique et aux Énergies Alternatives
2010-2017
A previously published clinical trial demonstrated the benefit of autologous CD34+ cells transduced with a selfinactivating lentiviral vector (HPV569) containing an engineered β-globin gene (βA-T87Q-globin) in subject β thalassemia major. This has been modified to increase transduction efficacy without compromising safety. In vitro analyses indicated that changes resulted both increased titers (3 4 fold) and (2 3 fold). An vivo study which 58 β-thalassemic mice were transplanted vector- or...
Hematopoietic stem cell transplantation (HSCT) is a therapy used for multiple malignant and nonmalignant diseases, with chemotherapy pretransplantation myeloablation. The post-HSCT brain contains peripheral engrafted parenchymal macrophages, despite their absence in the normal brain, engraftment mechanism still undefined. Here we show that HSCT broadly disrupts mouse regenerative populations, including permanent loss of adult neurogenesis. Microglial density was halved, causing microglial...
A lentiviral vector encoding β-globin flanked by insulator elements has been used to treat β-thalassemia (β-Thal) successfully in one human subject. However, a clonal expansion was observed after integration the HMGA2 locus, raising question of how commonly would be associated with possible insertional activation. Here, we report correcting β-Thal murine model using same and busulfan-conditioning regimen, allowing us investigate efficacy evolution at 9.2 months transplantation bone marrow...
Rett syndrome (RTT) is a rare neurodevelopmental disorder caused by mutation in the X-linked gene methyl-CpG-binding protein 2 (Mecp2), ubiquitously expressed transcriptional regulator. RTT results mental retardation and developmental regression that affects approximately 1 10,000 females. Currently, there no curative treatment for RTT. Thus, it crucial to develop new therapeutic approaches children suffering from Several studies suggested linked with defects cholesterol homeostasis, but...
Although gene transfer to hematopoietic stem cells (HSCs) has shown therapeutic efficacy in recent trials for several individuals with inherited disorders, transduction incompleteness of the HSC population remains a hurdle yield cure all patients reasonably low integrated vector numbers. In previous attempts at selection, massive loss transduced HSCs, contamination non-transduced cells, or lack applicability large cell populations rendered procedures out reach human applications. Here, we...
Wireless powered optogenetic cell-based implant provides a strategy to deliver subcutaneously therapeutic proteins. Immortalize Human Mesenchymal Stem Cells (hMSC-TERT) expressing the bacteriophytochrome diguanylate cyclase (DGCL) were validated for controlled interferon-β delivery (Optoferon cells) in bioelectronic implant. Optoferon cells transcriptomic profiling was used elaborate an in-silico model of recombinant production. optoelectronic device integration developed using additive...
ABSTRACT Preventing neurodegeneration-associated disability progression in patients with multiple sclerosis (MS) remains an unmet therapeutic need. As remyelination prevents degeneration of demyelinated axons, promoting this process might halt the development permanent disability. In demyelinating mouse lesions, local overexpression Semaphorin 3F (Sema3F), oligodendrocyte progenitor cell (OPC) attractant, increases OPC recruitment and remyelination. However, molecular targeting to MS lesions...