Improved collection of hematopoietic stem cells and progenitors from Fanconi anemia patients for gene therapy purposes

Improved collection of hematopoietic stem cells and progenitors from Fanconi anemia patients for gene therapy purposes

Difficulties in the collection of hematopoietic stem and progenitor cells (HSPCs) from Fanconi anemia (FA) patients have limited the gene therapy in this disease. We have investigated (ClinicalTrials.gov, NCT02931071) the safety and efficacy of filgrastim and plerixafor for mobilization of HSPCs and...

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Main Authors: Julián Sevilla, Susana Navarro, Paula Rio, Rebeca Sánchez-Domínguez, Josune Zubicaray, Eva Gálvez, Eva Merino, Elena Sebastián, Carmen Azqueta, José A. Casado, José C. Segovia, Omaira Alberquilla, Massimo Bogliolo, Francisco J. Román-Rodríguez, Yari Giménez, Lise Larcher, Rocío Salgado, Roser M. Pujol, Raquel Hladun, Ana Castillo, Jean Soulier, Sergi Querol, Jesús Fernández, Jonathan Schwartz, Nagore García de Andoín, Ricardo López, Albert Catalá, Jordi Surralles, Cristina Díaz-de-Heredia, Juan A. Bueren
Format: Article
Language:English
Published: Elsevier 2021-09-01
Series:Molecular Therapy: Methods & Clinical Development
Subjects:
Fanconi anemia
HSPC collection
gene therapy
Mozobil
mobilization
leukapheresis
Online Access:http://www.sciencedirect.com/science/article/pii/S2329050121001030

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http://www.sciencedirect.com/science/article/pii/S2329050121001030

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