Successful RAG1-SCID gene therapy depends on the level of RAG1 expression

Affiliations

¹ Department of Immunohematology and Blood Transfusion, Leiden University Medical Center, Leiden, The Netherlands.
² Institute of Experimental Hematology, Hannover Medical School, Hannover, Germany.
³ Department of Pediatrics, Leiden University Medical Center, Leiden, The Netherlands.
⁴ Département de Biothérapie, Hôpital Necker-Enfants Malades, Paris, France.
⁵ Department of Pediatrics, Erasmus Medical Center, Rotterdam, The Netherlands.
⁶ Molecular Immunology Unit, UCL Institute of Child Health, London, United Kingdom.
⁷ Department of Molecular Cell Biology, Leiden University Medical Center, Leiden, The Netherlands.
⁸ Department of Pharmacy, Leiden University Medical Center, Leiden, The Netherlands.
⁹ Department of Immunology, Erasmus Medical Center, Rotterdam, The Netherlands.
¹⁰ Department of Immunohematology and Blood Transfusion, Leiden University Medical Center, Leiden, The Netherlands. Electronic address: f.j.t.staal@lumc.nl.

Comment

Karin Pike-Overzet et al. J Allergy Clin Immunol. 2014 Jul.

. 2014 Jul;134(1):242-3.

doi: 10.1016/j.jaci.2014.04.033. Epub 2014 Jun 26.

¹ Department of Immunohematology and Blood Transfusion, Leiden University Medical Center, Leiden, The Netherlands.
² Institute of Experimental Hematology, Hannover Medical School, Hannover, Germany.
³ Department of Pediatrics, Leiden University Medical Center, Leiden, The Netherlands.
⁴ Département de Biothérapie, Hôpital Necker-Enfants Malades, Paris, France.
⁵ Department of Pediatrics, Erasmus Medical Center, Rotterdam, The Netherlands.
⁶ Molecular Immunology Unit, UCL Institute of Child Health, London, United Kingdom.
⁷ Department of Molecular Cell Biology, Leiden University Medical Center, Leiden, The Netherlands.
⁸ Department of Pharmacy, Leiden University Medical Center, Leiden, The Netherlands.
⁹ Department of Immunology, Erasmus Medical Center, Rotterdam, The Netherlands.
¹⁰ Department of Immunohematology and Blood Transfusion, Leiden University Medical Center, Leiden, The Netherlands. Electronic address: f.j.t.staal@lumc.nl.

No abstract available

Comment in

Reply: To PMID 24332219.
van Til NP, Cortes P, Danos O, Cassani B, Poliani PL, Villa A, Wagemaker G. van Til NP, et al. J Allergy Clin Immunol. 2014 Jul;134(1):243-4. doi: 10.1016/j.jaci.2014.04.032. Epub 2014 Jun 26. J Allergy Clin Immunol. 2014. PMID: 25117804 No abstract available.

Recombination-activating gene 1 (Rag1)-deficient mice with severe combined immunodeficiency treated with lentiviral gene therapy demonstrate autoimmune Omenn-like syndrome.
van Til NP, Sarwari R, Visser TP, Hauer J, Lagresle-Peyrou C, van der Velden G, Malshetty V, Cortes P, Jollet A, Danos O, Cassani B, Zhang F, Thrasher AJ, Fontana E, Poliani PL, Cavazzana M, Verstegen MM, Villa A, Wagemaker G. van Til NP, et al. J Allergy Clin Immunol. 2014 Apr;133(4):1116-23. doi: 10.1016/j.jaci.2013.10.009. Epub 2013 Dec 9. J Allergy Clin Immunol. 2014. PMID: 24332219