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Page 1
Gene Therapy for β-Hemoglobinopathies.
Cavazzana M, Antoniani C, Miccio A. Cavazzana M, et al. Mol Ther. 2017 May 3;25(5):1142-1154. doi: 10.1016/j.ymthe.2017.03.024. Epub 2017 Apr 1. Mol Ther. 2017. PMID: 28377044 Free PMC article. Review.
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. Among authors: cavazzana m. 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
Gene therapy for inherited immunodeficiency.
Touzot F, Hacein-Bey-Abina S, Fischer A, Cavazzana M. Touzot F, et al. Among authors: cavazzana m. Expert Opin Biol Ther. 2014 Jun;14(6):789-98. doi: 10.1517/14712598.2014.895811. Epub 2014 Mar 8. Expert Opin Biol Ther. 2014. PMID: 24823313 Review.
Gene therapy for primary immunodeficiencies.
Fischer A, Hacein-Bey Abina S, Touzot F, Cavazzana M. Fischer A, et al. Among authors: cavazzana m. Clin Genet. 2015 Dec;88(6):507-15. doi: 10.1111/cge.12576. Epub 2015 Mar 23. Clin Genet. 2015. PMID: 25708106 Review.
Faster T-cell development following gene therapy compared with haploidentical HSCT in the treatment of SCID-X1.
Touzot F, Moshous D, Creidy R, Neven B, Frange P, Cros G, Caccavelli L, Blondeau J, Magnani A, Luby JM, Ternaux B, Picard C, Blanche S, Fischer A, Hacein-Bey-Abina S, Cavazzana M. Touzot F, et al. Among authors: cavazzana m. Blood. 2015 Jun 4;125(23):3563-9. doi: 10.1182/blood-2014-12-616003. Epub 2015 Apr 13. Blood. 2015. PMID: 25869287 Free article. Clinical Trial.
Outcomes following gene therapy in patients with severe Wiskott-Aldrich syndrome.
Hacein-Bey Abina S, Gaspar HB, Blondeau J, Caccavelli L, Charrier S, Buckland K, Picard C, Six E, Himoudi N, Gilmour K, McNicol AM, Hara H, Xu-Bayford J, Rivat C, Touzot F, Mavilio F, Lim A, Treluyer JM, Héritier S, Lefrère F, Magalon J, Pengue-Koyi I, Honnet G, Blanche S, Sherman EA, Male F, Berry C, Malani N, Bushman FD, Fischer A, Thrasher AJ, Galy A, Cavazzana M. Hacein-Bey Abina S, et al. Among authors: cavazzana m. JAMA. 2015 Apr 21;313(15):1550-63. doi: 10.1001/jama.2015.3253. JAMA. 2015. PMID: 25898053 Free PMC article. Clinical Trial.
Lymphopoiesis in transgenic mice over-expressing Artemis.
Rivera-Munoz P, Abramowski V, Jacquot S, André P, Charrier S, Lipson-Ruffert K, Fischer A, Galy A, Cavazzana M, de Villartay JP. Rivera-Munoz P, et al. Among authors: cavazzana m. Gene Ther. 2016 Feb;23(2):176-86. doi: 10.1038/gt.2015.95. Epub 2015 Oct 1. Gene Ther. 2016. PMID: 26361272
173 results