The production and validation of a cellular thalassemia Biobank was conducted within the multicenter European Project THALAMOSS (THalassemia MOdular Stratification System for personalized therapy of β-thalassemia). The cellular Biobank contains hematopoietic stem cells isolated from the peripheral blood, expanded, freezed and cryopreserved. The use of biobanked samples allows to compare potentially therapeutic treatments on several samples growing in parallel. Freezing, cryopreservation and thawing steps do not affect the erythroid differentiation potential of the cells in terms of kinetics and types of hemoglobin produced by the cells obtained from different biobanked cryovials of the same patient and thawed with intervals reaching several months. The validation the cellular Biobank was further performed by sending and culturing the crypreserved cells from the same patients in different laboratories. Finally, we demonstrated that the biobanked cells can be used for determining the response to different inducers, such as mithramycin, hydroxyurea and resveratrol. Moreover, the biobanked cells can be used to test their response to gene therapy protocols. At present, the THALAMOSS cellular Biobank is composed by samples from 135 β-thalassemia patients and 19 healthy donors for a totale of more than 750 cryovials. The most represented genotypes are β039/β039 (51 patients), β+IVSI-6/β+IVSI-110 (31 patients), β039/β+IVSI-110 (21 patients) and β+IVSI-110/β+IVSI-110 (9 patients). In conclusion, this research activity will allow patients stratification taking into account all the phenotypic/genotypic characteristics of the single individual, and the response to potential therapeutic treatments.
The multicenter THALAMOSS cellular Biobank for β-Thalassemia
COSENZA, Lucia Carmela;BREDA, Laura;BREVEGLIERI, Giulia;ZUCCATO, Cristina;FINOTTI, Alessia;LAMPRONTI, Ilaria;BORGATTI, Monica;GAMBARI, Roberto;BIANCHI, Nicoletta
2016
Abstract
The production and validation of a cellular thalassemia Biobank was conducted within the multicenter European Project THALAMOSS (THalassemia MOdular Stratification System for personalized therapy of β-thalassemia). The cellular Biobank contains hematopoietic stem cells isolated from the peripheral blood, expanded, freezed and cryopreserved. The use of biobanked samples allows to compare potentially therapeutic treatments on several samples growing in parallel. Freezing, cryopreservation and thawing steps do not affect the erythroid differentiation potential of the cells in terms of kinetics and types of hemoglobin produced by the cells obtained from different biobanked cryovials of the same patient and thawed with intervals reaching several months. The validation the cellular Biobank was further performed by sending and culturing the crypreserved cells from the same patients in different laboratories. Finally, we demonstrated that the biobanked cells can be used for determining the response to different inducers, such as mithramycin, hydroxyurea and resveratrol. Moreover, the biobanked cells can be used to test their response to gene therapy protocols. At present, the THALAMOSS cellular Biobank is composed by samples from 135 β-thalassemia patients and 19 healthy donors for a totale of more than 750 cryovials. The most represented genotypes are β039/β039 (51 patients), β+IVSI-6/β+IVSI-110 (31 patients), β039/β+IVSI-110 (21 patients) and β+IVSI-110/β+IVSI-110 (9 patients). In conclusion, this research activity will allow patients stratification taking into account all the phenotypic/genotypic characteristics of the single individual, and the response to potential therapeutic treatments.I documenti in SFERA sono protetti da copyright e tutti i diritti sono riservati, salvo diversa indicazione.
