Published in
American Society of Hematology, Blood, 13(130), p. 1535-1542
DOI: 10.1182/blood-2017-03-774174
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Engraftment and in vivo proliferation advantage of gene-corrected mobilized CD34+ cells from Fanconi anemia patients
Journal article published in 2017 by
Paula Río 
,
Susana Navarro ,
Guillermo Guenechea ,
Rebeca Sánchez-Domínguez ,
Maria Luisa Lamana,
Rosa Yañez ,
Jose A. Casado ,
Parinda A. Mehta,
Maria Roser Pujol ,
Jordi Surrallés ,
Sabine Charrier,
Anne Galy ,
José C. Segovia ,
Cristina Díaz de Heredia ,
Julián Sevilla
and other authors.
,
Susana Navarro ,
Guillermo Guenechea ,
Rebeca Sánchez-Domínguez ,
Maria Luisa Lamana,
Rosa Yañez ,
Jose A. Casado ,
Parinda A. Mehta,
Maria Roser Pujol ,
Jordi Surrallés ,
Sabine Charrier,
Anne Galy ,
José C. Segovia ,
Cristina Díaz de Heredia ,
Julián Sevilla
and other authors.
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Abstract
Key Points First evidence of phenotypic correction in FA hematopoietic repopulating cells by optimized collection and short transduction of CD34+ cells. Optimized ex vivo gene therapy of FA CD34+ cells confers proliferation advantage to phenotypically corrected repopulating cells.