VCN Analysis Using Droplet Digital PCR Method in Hematopoietic Stem Cells and T Lymphocytes after Lentiviral Transduction: Optimization and Limitations

Autor: Aude Parcelier, Julien Buisset, Emilie Neveu, Bastien Kauffmann, Sandrine Fraboulet, Peggy Sanatine, Samia Martin, C?drick Rousseaux, Nadia Avenier, Sabine Charrier

Publikováno v: Re:GEN Open, Vol 2, Iss 1, Pp 28-36 (2022)

Background: In the context of ex vivo gene therapy or chimeric antigen receptor T cell (CAR-T) cell therapy, vector copy number (VCN) analysis in transduced cells by lentiviral vectors enables the assessment of risk and therapeutic efficiency in pati

Externí odkaz: https://doaj.org/article/1891b6ea0fe24f2fbad1c22dc45dcd7f

Pre-clinical Development of a Lentiviral Vector Expressing the Anti-sickling βAS3 Globin for Gene Therapy for Sickle Cell Disease

Autor: Valentina Poletti, Fabrizia Urbinati, Sabine Charrier, Guillaume Corre, Roger P. Hollis, Beatriz Campo Fernandez, Samia Martin, Michael Rothe, Axel Schambach, Donald B. Kohn, Fulvio Mavilio

Publikováno v: Molecular Therapy: Methods & Clinical Development, Vol 11, Iss , Pp 167-179 (2018)

Sickle cell disease (SCD) is caused by a mutation (E6V) in the hemoglobin (Hb) β-chain that induces polymerization of Hb tetramers, red blood cell deformation, ischemia, anemia, and multiple organ damage. Gene therapy is a potential alternative to h

Externí odkaz: https://doaj.org/article/eb2c06bff7d64ce5a72279d990d278a9

Wiskott-Aldrich syndrome protein-deficient hematopoietic cells can be efficiently mobilized by granulocyte colony-stimulating factor

Autor: Sabine Charrier, Michael Blundell, Gregory Cédrone, Fawzia Louache, William Vainchenker, Adrian J. Thrasher, Anne Galy

Publikováno v: Haematologica, Vol 98, Iss 8 (2013)

The Wiskott-Aldrich syndrome protein is an essential cytoskeleton regulator found in cells of the hematopoietic lineage and controls the motility of leukocytes. The impact of WAS gene deficiency on the mobilization of hematopoietic progenitor/stem ce

Externí odkaz: https://doaj.org/article/cd49dbcf1c44494894b00ee0a08857c0

Biosafety Studies of a Clinically Applicable Lentiviral Vector for the Gene Therapy of Artemis-SCID

Autor: Grégory Cédrone, Alain Fischer, Sabine Charrier, Axel Schambach, Michael Rothe, Chantal Lagresle-Peyrou, Bernard Gjata, Salima Hacein-Bey-Abina, Jean-Pierre de Villartay, Fulvio Mavilio, Anne Galy, Valentina Poletti, Marina Cavazzana

Publikováno v: Mol Ther Methods Clin Dev
Mol Ther Methods Clin Dev, 2019, 15, pp.232-245. ⟨10.1016/j.omtm.2019.08.014⟩
Molecular Therapy. Methods & Clinical Development
Molecular Therapy-Methods & Clinical Development
Molecular Therapy: Methods & Clinical Development, Vol 15, Iss, Pp 232-245 (2019)

Genetic deficiency of the nuclease DCLRE1C/Artemis causes radiosensitive severe combined immunodeficiency (RS-SCID) with lack of peripheral T and B cells and increased sensitivity to ionizing radiations. Gene therapy based on transplanting autologous

Externí odkaz: https://explore.openaire.eu/search/publication?articleId=doi_dedup___::95eb6abbec6734dd7e1265579a3a67bb
https://hal.archives-ouvertes.fr/hal-02880790

Engraftment and in vivo proliferation advantage of gene-corrected mobilized CD34(+) cells from Fanconi anemia patients

Autor: Parinda A. Mehta, Julián Sevilla, Maria Roser Pujol, José A. Casado, Anne Galy, Sabine Charrier, María L. Lamana, Rebeca Sanchez-Dominguez, Cristina Díaz de Heredia, Jordi Surrallés, Rosa Yañez, Guillermo Guenechea, José C. Segovia, Susana Navarro, Juan A. Bueren, Paula Río

Publikováno v: Blood
Blood, 2017, 130 (13), pp.1535-1542. ⟨10.1182/blood-2017-03-774174⟩
Blood, American Society of Hematology, 2017, 130 (13), pp.1535-1542. ⟨10.1182/blood-2017-03-774174⟩
BLOOD
r-IIB SANT PAU. Repositorio Institucional de Producción Científica del Instituto de Investigación Biomédica Sant Pau
instname

International audience; Previous Fanconi anemia (FA) gene therapy studies have failed to demonstrate engraftment of gene-corrected hematopoietic stem and progenitor cells (HSPCs) from FA patients, either after autologous transplantation or infusion i

Externí odkaz: https://explore.openaire.eu/search/publication?articleId=doi_dedup___::8cf37e2a2bf00b94877944883f51001b
https://hal.science/hal-02881013