Results
author = "Río, Paula"
1
Found 8 documents, displaying page 1 of 1
Disease-corrected haematopoietic progenitors from Fanconi anemia induced pluripotent stem cells
Author(s) :
Rodríguez Pizà, Ignasi
,
Verma, Inder M.
,
Veiga, Anna
,
Aasen, Trond
,
Izpisúa Belmonte, J. C.
,
Bueren, Juan
,
Garreta, Elena
Description :
The generation of induced pluripotent stem (iPS) cells has enabled the derivation of patient-specific pluripotent cells and
provided valuable experimental platforms to model human disease. Patient-specific iPS cells are also thought to hold great
therapeutic potential, although direct evidence for t...
Repository :
e-Repositori UPF
Language(s) :
English
Deficiency of Bruton's tyrosine kinase in B cell precursor leukemia cells
Author(s) :
Feldhahn, Niklas
,
Río, Paula
,
Soh, Bonaventure Ndikung Bejeng
,
Liedtke, Stefanie
,
Sprangers, Mieke
,
Klein, Florian
Description :
Bruton's tyrosine kinase (BTK) deficiency results in a differentiation block at the pre-B cell stage. Likewise, acute lymphoblastic leukemia cells are typically arrested at early stages of B cell development. We therefore investigated BTK function in B cell precursor leukemia cells carrying a BCR-AB...
Repository :
Europe PubMed Central
Language(s) :
English
Lentiviral-mediated Genetic Correction of Hematopoietic and Mesenchymal Progenitor Cells From Fanconi Anemia Patients
Author(s) :
Jacome, Ariana
,
Navarro, Susana
,
Río, Paula
,
Yañez, Rosa M
,
González-Murillo, Africa
,
Luz Lozano, M
,
Lamana, Maria Luisa
Description :
Previous clinical trials based on the genetic correction of purified CD34+ cells with γ-retroviral vectors have demonstrated clinical efficacy in different monogenic diseases, including X-linked severe combined immunodeficiency, adenosine deaminase deficient severe combined immunodeficiency and chro...
Repository :
Europe PubMed Central
Language(s) :
English
Bcr/Abl Interferes with the Fanconi Anemia/BRCA Pathway: Implications in the Chromosomal Instability of Chronic Myeloid Leukemia Cells
Author(s) :
Valeri, Antonio
,
Alonso-Ferrero, Maria Eugenia
,
Río, Paula
,
Pujol, María Roser
,
Casado, José A.
,
Pérez, Laura
,
Jacome, Ariana
Description :
Chronic myeloid leukemia (CML) is a malignant clonal disorder of the hematopoietic system caused by the expression of the BCR/ABL fusion oncogene. Although it is well known that CML cells are genetically unstable, the mechanisms accounting for this genomic instability are still poorly understood. Be...
Repository :
Europe PubMed Central
Language(s) :
English
Transforming and Tumorigenic Activity of JAK2 by Fusion to BCR: Molecular Mechanisms of Action of a Novel BCR-JAK2 Tyrosine-Kinase
Author(s) :
Cuesta-Domínguez, Álvaro
,
Ortega, Mara
,
Ormazábal, Cristina
,
Santos-Roncero, Matilde
,
Galán-Díez, Marta
,
Steegmann, Juan Luis
Description :
Chromosomal translocations in tumors frequently produce fusion genes coding for chimeric proteins with a key role in oncogenesis. Recent reports described a BCR-JAK2 fusion gene in fatal chronic and acute myeloid leukemia, but the functional behavior of the chimeric protein remains uncharacterized. ...
Repository :
Europe PubMed Central
Language(s) :
English
Gene Therapy for Fanconi Anemia: One Step Closer to the Clinic
Author(s) :
Tolar, Jakub
,
Becker, Pamela S.
,
Clapp, D. Wade
,
Hanenberg, Helmut
,
de Heredia, Cristina Díaz
,
Kiem, Hans-Peter
Description :
Repository :
Europe PubMed Central
Language(s) :
English
A comprehensive strategy for the subtyping of patients with Fanconi anaemia: conclusions from the Spanish Fanconi Anemia Research Network
Author(s) :
Casado, José Antonio
,
Callén, Elsa
,
Jacome, Ariana
,
Río, Paula
,
Castella, Maria
,
Lobitz, Stephan
,
Ferro, Teresa
,
Muñoz, Arturo
Description :
Repository :
Europe PubMed Central
Language(s) :
English
Stem Cell Gene Therapy for Fanconi Anemia: Report from the 1st International Fanconi Anemia Gene Therapy Working Group Meeting
Author(s) :
Tolar, Jakub
,
Adair, Jennifer E
,
Antoniou, Michael
,
Bartholomae, Cynthia C
,
Becker, Pamela S
,
Blazar, Bruce R
,
Bueren, Juan
Description :
Survival rates after allogeneic hematopoietic cell transplantation (HCT) for Fanconi anemia (FA) have increased dramatically since 2000. However, the use of autologous stem cell gene therapy, whereby the patient's own blood stem cells are modified to express the wild-type gene product, could potenti...
Repository :
Europe PubMed Central
Language(s) :
English
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Found 8 documents, displaying page 1 of 1