We found a match
Your institution may have access to this item. Find your institution then sign in to continue.
- Title
A validated cellular biobank for β-thalassemia.
- Authors
Cosenza, Lucia Carmela; Breda, Laura; Breveglieri, Giulia; Zuccato, Cristina; Finotti, Alessia; Lampronti, Ilaria; Borgatti, Monica; Chiavilli, Francesco; Gamberini, Maria Rita; Satta, Stefania; Manunza, Laura; De Martis, Franca Rosa; Moi, Paolo; Rivella, Stefano; Gambari, Roberto; Bianchi, Nicoletta
- Abstract
<bold>Background: </bold>Cellular biobanking is a key resource for collaborative networks planning to use same cells in studies aimed at solving a variety of biological and biomedical issues. This approach is of great importance in studies on β-thalassemia, since the recruitment of patients and collection of specimens can represent a crucial and often limiting factor in the experimental planning.<bold>Methods: </bold>Erythroid precursor cells were obtained from 72 patients, mostly β-thalassemic, expanded and cryopreserved. Expression of globin genes was analyzed by real time RT-qPCR. Hemoglobin production was studied by HPLC.<bold>Results: </bold>In this paper we describe the production and validation of a Thal-Biobank constituted by expanded erythroid precursor cells from β-thalassemia patients. The biobanked samples were validated for maintenance of their phenotype after (a) cell isolation from same patients during independent phlebotomies, (b) freezing step in different biobanked cryovials, (c) thawing step and analysis at different time points. Reproducibility was confirmed by shipping the frozen biobanked cells to different laboratories, where the cells were thawed, cultured and analyzed using the same standardized procedures. The biobanked cells were stratified on the basis of their baseline level of fetal hemoglobin production and exposed to fetal hemoglobin inducers.<bold>Conclusion: </bold>The use of biobanked cells allows stratification of the patients with respect to fetal hemoglobin production and can be used for determining the response to the fetal hemoglobin inducer hydroxyurea and to gene therapy protocols with reproducible results.
- Subjects
THALASSEMIA; GENE therapy; PHLEBOTOMISTS; REPRODUCIBLE research; ERYTHROCYTES; MEDICAL research
- Publication
Journal of Translational Medicine, 2016, Vol 14, p1
- ISSN
1479-5876
- Publication type
journal article
- DOI
10.1186/s12967-016-1016-4