Gene Therapy With The Lentiviral Vector Tns9.3.55 Produces Long-Term Improvement In Severe ß-Thalassemia
Author(s): Aurelio Maggio, Michel Sadelain, Isabelle Riviere, Xiuyan Wang, Simona Raso, Santina Acuto, Rita Barone, Rosario Di Maggio, Lorella Pitrolo, Farid Boulad
EHA Library. Raso S. 06/12/20; 293982; EP1497
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β-thalassemia is the most prevalent monogenic disease for which gene therapy with autologous hematopoietic progenitor cells (HPC) modiﬁed to express ß-globin represents a potential therapeutic option. The most relevant clinical variables inﬂuencing success of gene therapy in ß-thalassemia are patient genotype, myeloablative conditioning regimen, number and type of progenitor cells, and vector copy number (VCN).
We report the results of a safety and efﬁcacy study of the use of gene therapy with the human globin TNS9.3.55 vector in three patients at Memorial Sloan Kettering Cancer Center between November 2012 and May 2015, with a follow-up of 5-7 years. The results of 3 patients treated and followed at Department of Hematology and Rare Diseases European Reference Network Center for Rare Hematological Diseases (Hospital Villa Soﬁa-V. Cervello) are reported here.
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