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The Functional Effect of Repeated Cryopreservation on Transduced CD34(+) Cells from Patients with Thalassemia

Stable gene marking and effective engraftment of gene-modified CD34(+) hematopoietic stem cells is a prerequisite for gene therapy success but may be challenged by the inevitable cryopreservation of the final product prior to extensive quality assurance testing. We investigated the β-globin gene tra...

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Библиографические подробности
Опубликовано в: :Hum Gene Ther Methods
Главные авторы: Karponi, Garyfalia, Papayanni, Penelope-Georgia, Zervou, Fani, Bouinta, Asimina, Anagnostopoulos, Achilles, Yannaki, Evangelia
Формат: Artigo
Язык:Inglês
Опубликовано: Mary Ann Liebert, Inc., publishers 2018
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Online-ссылка:https://ncbi.nlm.nih.gov/pmc/articles/PMC6206548/
https://ncbi.nlm.nih.gov/pubmed/30079761
https://ncbi.nlm.nih.govhttp://dx.doi.org/10.1089/hgtb.2018.032
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