Sophie Ramadier

Weight based dosing of alemtuzumab as part of an enhanced lymphodepletion regimen may mitigate the risks of over and underexposure and improve response rates : Analysis of the nathali-01 trial

Xenia Naj, Laurent Poirot, Vivian Dai, P Vermeulen et autres

Abstract Introduction: Chimeric Antigen Receptor T-cell (CAR-T) therapy has revolutionized treatment of refractory hematologic cancers, particularly B-cell malignancies. A critical preparative step for CAR-T infusion is lymphodepletion (LD) which creates a more permissive immune environment to improve CAR-T expansion, engraftment and efficacy. …

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Trial in progress: Open-label dose-finding and dose-expansion study to evaluate the safety, expansion, persistence, and clinical activity of UCART20x22 in subjects with relapsed or refractory B-cell non-Hodgkin lymphoma (B-NHL) nathali-01

Jeremy S. Abramson, Aravind Ramakrishnan, Emmanuel Bachy, Laurent Poirot et autres

Abstract Background Despite significant advances in the treatment of B-cell non-Hodgkin's lymphoma, new treatment approaches are needed for patients with relapsed/refractory (R/R) disease, especially for those with chemo-refractory NHL who fail to achieve a response or relapse early after the standard of …

us, fr (code pays fourni par la source)

Novel lentiviral vectors for gene therapy of sickle cell disease combining gene addition and gene silencing strategies

Mégane Brusson, Anne Chalumeau, Pierre Martinucci, Oriana Romano et autres

Sickle cell disease (SCD) is due to a mutation in the β-globin gene causing production of the toxic sickle hemoglobin (HbS; α 2 β S 2 ). Transplantation of autologous hematopoietic stem and progenitor cells (HSPCs) transduced with lentiviral vectors (LVs) expressing …

fr, it, us (code pays fourni par la source)

Novel lentiviral vectors for gene therapy of sickle cell disease combining gene addition and gene silencing strategies

Mégane Brusson, Anne Chalumeau, Pierre Martinucci, Oriana Romano et autres

Abstract Sickle cell disease (SCD) is due to a mutation in the β-globin ( HBB ) gene causing the production of the toxic sickle hemoglobin (HbS, a 2 β S 2 ). Transplantation of autologous hematopoietic stem/progenitor cells (HSPCs) transduced with lentiviral …

fr, it, us (code pays fourni par la source)

Combination of lentiviral and genome editing technologies for the treatment of sickle cell disease

Sophie Ramadier, Anne Chalumeau, Tristan Félix, Nadia Othman et autres

Sickle cell disease (SCD) is caused by a mutation in the β-globin gene leading to polymerization of the sickle hemoglobin (HbS) and deformation of red blood cells. Autologous transplantation of hematopoietic stem/progenitor cells (HSPCs) genetically modified using lentiviral vectors (LVs) to express …

fr, it (code pays fourni par la source)

Combination of lentiviral vector and genome editing technologies for the treatment of ∩-hemoglobinopathies

Sophie Ramadier

Combination of lentiviral vector and genome editing technologies for the treatment of beta-hemoglobinopathies

Sophie Ramadier

Combinaison des technologies de vecteur lentiviral et d'édition du génome pour le traitement des bêta-hémoglobinopathies La drépanocytose et la bêta-thalassémie sont causées par des mutations affectant le gène de la globine-bêta (HBB). Dans la drépanocytose, la substitution d'un acide aminé dans la …

fr (code pays fourni par la source)

A Case of Conjunctival Bee Sting Injury with Review of the Literature on Ocular Bee Stings

Axelle Semler-Collery, George Hayek, Sophie Ramadier, Jean‐Marc Perone

BACKGROUND Ocular bee stings have been rarely described in the literature, and their management is controversial. A case of conjunctival bee sting with retention of the stinger for 48 hours is presented with a review of the literature on the complications and …

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Optimization of CRISPR/Cas9 Delivery to Human Hematopoietic Stem and Progenitor Cells for Therapeutic Genomic Rearrangements

Annalisa Lattanzi, Vasco Meneghini, Giulia Pavani, Fatima Amor et autres

Editing the β-globin locus in hematopoietic stem cells is an alternative therapeutic approach for gene therapy of β-thalassemia and sickle cell disease. Using the CRISPR/Cas9 system, we genetically modified human hematopoietic stem and progenitor cells (HSPCs) to mimic the large rearrangements in …

fr, us, it (code pays fourni par la source)