E-resources
Peer reviewed
-
Río, Paula; Navarro, Susana; Wang, Wei; Sánchez-Domínguez, Rebeca; Pujol, Roser M; Segovia, José C; Bogliolo, Massimo; Merino, Eva; Wu, Ning; Salgado, Rocío; Lamana, María L; Yañez, Rosa M; Casado, José A; Giménez, Yari; Román-Rodríguez, Francisco J; Álvarez, Lara; Alberquilla, Omaira; Raimbault, Anna; Guenechea, Guillermo; Lozano, M Luz; Cerrato, Laura; Hernando, Miriam; Gálvez, Eva; Hladun, Raquel; Giralt, Irina; Barquinero, Jordi; Galy, Anne; García de Andoín, Nagore; López, Ricardo; Catalá, Albert; Schwartz, Jonathan D; Surrallés, Jordi; Soulier, Jean; Schmidt, Manfred; Díaz de Heredia, Cristina; Sevilla, Julián; Bueren, Juan A
Nature medicine, 09/2019, Volume: 25, Issue: 9Journal Article
Fanconi anemia (FA) is a DNA repair syndrome generated by mutations in any of the 22 FA genes discovered to date . Mutations in FANCA account for more than 60% of FA cases worldwide . Clinically, FA is associated with congenital abnormalities and cancer predisposition. However, bone marrow failure is the primary pathological feature of FA that becomes evident in 70-80% of patients with FA during the first decade of life . In this clinical study (ClinicalTrials.gov, NCT03157804 ; European Clinical Trials Database, 2011-006100-12), we demonstrate that lentiviral-mediated hematopoietic gene therapy reproducibly confers engraftment and proliferation advantages of gene-corrected hematopoietic stem cells (HSCs) in non-conditioned patients with FA subtype A. Insertion-site analyses revealed the multipotent nature of corrected HSCs and showed that the repopulation advantage of these cells was not due to genotoxic integrations of the therapeutic provirus. Phenotypic correction of blood and bone marrow cells was shown by the acquired resistance of hematopoietic progenitors and T lymphocytes to DNA cross-linking agents. Additionally, an arrest of bone marrow failure progression was observed in patients with the highest levels of gene marking. The progressive engraftment of corrected HSCs in non-conditioned patients with FA supports that gene therapy should constitute an innovative low-toxicity therapeutic option for this life-threatening disorder.
Author
![loading ... loading ...](themes/default/img/ajax-loading.gif)
Shelf entry
Permalink
- URL:
Impact factor
Access to the JCR database is permitted only to users from Slovenia. Your current IP address is not on the list of IP addresses with access permission, and authentication with the relevant AAI accout is required.
Year | Impact factor | Edition | Category | Classification | ||||
---|---|---|---|---|---|---|---|---|
JCR | SNIP | JCR | SNIP | JCR | SNIP | JCR | SNIP |
Select the library membership card:
If the library membership card is not in the list,
add a new one.
DRS, in which the journal is indexed
Database name | Field | Year |
---|
Links to authors' personal bibliographies | Links to information on researchers in the SICRIS system |
---|
Source: Personal bibliographies
and: SICRIS
The material is available in full text. If you wish to order the material anyway, click the Continue button.