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Charrier S, Lagresle-Peyrou C, Poletti V, et al. Biosafety Studies of a Clinically Applicable Lentiviral Vector for the Gene Therapy of Artemis-SCID. Mol Ther Methods Clin Dev. 2019;15:232-245doi: 10.1016/j.omtm.2019.08.014.
Charrier, S., Lagresle-Peyrou, C., Poletti, V., Rothe, M., Cédrone, G., Gjata, B., Mavilio, F., Fischer, A., Schambach, A., de Villartay, J. P., Cavazzana, M., Hacein-Bey-Abina, S., & Galy, A. (2019). Biosafety Studies of a Clinically Applicable Lentiviral Vector for the Gene Therapy of Artemis-SCID. Molecular therapy. Methods & clinical development, 15232-245. https://doi.org/10.1016/j.omtm.2019.08.014
Charrier, Sabine, et al. "Biosafety Studies of a Clinically Applicable Lentiviral Vector for the Gene Therapy of Artemis-SCID." Molecular therapy. Methods & clinical development vol. 15 (2019): 232-245. doi: https://doi.org/10.1016/j.omtm.2019.08.014
Charrier S, Lagresle-Peyrou C, Poletti V, Rothe M, Cédrone G, Gjata B, Mavilio F, Fischer A, Schambach A, de Villartay JP, Cavazzana M, Hacein-Bey-Abina S, Galy A. Biosafety Studies of a Clinically Applicable Lentiviral Vector for the Gene Therapy of Artemis-SCID. Mol Ther Methods Clin Dev. 2019 Sep 13;15:232-245. doi: 10.1016/j.omtm.2019.08.014. eCollection 2019 Dec 13. PMID: 31720302; PMCID: PMC6838972.
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Mol Ther Methods Clin Dev. 2019 Sep 13;15:232-245. doi: 10.1016/j.omtm.2019.08.014. eCollection 2019 Dec 13.

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

Molecular therapy. Methods & clinical development

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

Affiliations

  1. Genethon and UMR_S951, INSERM, Université Evry, Université Paris Saclay, Evry, 91002 Evry, France.
  2. Biotherapy Clinical Investigation Center, Groupe Hospitalier Universitaire Ouest, Assistance Publique-Hôpitaux de Paris, INSERM, Paris, France.
  3. Laboratory of Human Lymphohematopoiesis, UMR 1163, INSERM, Université Paris Descartes Sorbonne Paris Cité, Imagine Institute, Paris, France.
  4. Institute of Experimental Hematology, Hannover Medical School, Hannover, Germany.
  5. INSERM, UMR 1163, Paris Descartes University-Sorbonne Paris Cité, Paris, France.
  6. Imagine Institute, Paris, France.
  7. Immunology Pediatric Department, Hopital Necker-Enfants Malades, AP-HP, Paris, France.
  8. Collège de France, Paris, France.
  9. Laboratory of Genome Dynamics in the Immune System, UMR1163, INSERM, Université Paris Descartes Sorbonne Paris Cité, Imagine Institute, Paris, France.
  10. Biotherapy Department, Necker Children's Hospital, Assistance Publique-Hôpitaux de Paris, Paris, France.

PMID: 31720302 PMCID: PMC6838972 DOI: 10.1016/j.omtm.2019.08.014

Abstract

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 gene-modified hematopoietic stem cells could significantly improve the health of patients with RS-SCID by correcting their immune system. A lentiviral vector expressing physiological levels of human ARTEMIS mRNA from an EF1a promoter without post-transcriptional regulation was developed as a safe clinically applicable candidate for RS-SCID gene therapy. The vector was purified in GMP-comparable conditions and was not toxic

© 2019.

Keywords: Artemis; B cell; SCID; T cell; gene therapy; mouse; preclinical study; primary immunodeficiency

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