Cargando…

Gene therapy restores the transcriptional program of hematopoietic stem cells in Fanconi anemia

Clinical trials have shown that lentiviral-mediated gene therapy can ameliorate bone marrow failure (BMF) in non-conditioned Fanconi anemia (FA) patients resulting from the proliferative advantage of corrected FA hematopoietic stem and progenitor cells (HSPC). However, it is not yet known if gene th...

Descripción completa

Detalles Bibliográficos
Autores principales:	Lasaga, Miren, Río, Paula, Vilas-Zornoza, Amaia, Planell, Nuria, Navarro, Susana, Alignani, Diego, Fernández-Varas, Beatriz, Mouzo, Daniel, Zubicaray, Josune, Pujol, Roser M., Nicoletti, Eileen, Schwartz, Jonathan D., Sevilla, Julián, Ainciburi, Marina, Ullate-Agote, Asier, Surrallés, Jordi, Perona, Rosario, Sastre, Leandro, Prosper, Felipe, Gomez-Cabrero, David, Bueren, Juan A.
Formato:	Online Artículo Texto
Lenguaje:	English
Publicado:	Fondazione Ferrata Storti 2023
Materias:	Article - Bone Marrow Failure
Acceso en línea:	https://www.ncbi.nlm.nih.gov/pmc/articles/PMC10542844/ https://www.ncbi.nlm.nih.gov/pubmed/37021532 http://dx.doi.org/10.3324/haematol.2022.282418

Ejemplares similares

Genotype-phenotype and outcome associations in patients with Fanconi anemia: the National Cancer Institute cohort
por: Altintas, Burak, et al.
Publicado: (2022)

Sars-Cov-2 Infection Associated with Aplastic Anemia and Pure Red Cell Aplasia
por: Lee, Nicholas C.J., et al.
Publicado: (2021)

Sars-Cov-2 Vaccination in Patients with Paroxysmal Nocturnal Hemoglobinuria: An Italian Multicenter Survey
por: Giannotta, Juri Alessandro, et al.
Publicado: (2021)

Acquired Aplastic Anemia in Immunocompetent Pediatric Patients with Asymptomatic COVID-19 Infection: A Case Series
por: Fritch, Jordan E., et al.
Publicado: (2021)

Transforming growth factor-β signaling modifies the hematopoietic acute inflammatory response to drive bone marrow failure
por: Javier, Jose, et al.
Publicado: (2021)

Syndromes predisposing to leukemia are a major cause of inherited cytopenias in children
por: Gilad, Oded, et al.
Publicado: (2022)

Impaired immunosuppressive role of myeloid-derived suppressor cells in acquired aplastic anemia
por: Dong, Peiyuan, et al.
Publicado: (2022)

Somatic genetic alterations predict hematological progression in GATA2 deficiency
por: Largeaud, Laetitia, et al.
Publicado: (2023)

Effects of nandrolone decanoate on telomere length and clinical outcome in patients with telomeropathies: a prospective trial
por: Clé, Diego V., et al.
Publicado: (2022)

Conditional survival and standardized mortality ratios of patients with severe aplastic anemia surviving at least one year after hematopoietic cell transplantation or immunosuppressive therapy
por: Nakamura, Ryotaro, et al.
Publicado: (2023)

Gonadal function in pediatric Fanconi anemia patients treated with hematopoietic stem cell transplant
por: Koo, Jane, et al.
Publicado: (2023)

Mosaicism in Fanconi anemia: concise review and evaluation of published cases with focus on clinical course of blood count normalization
por: Nicoletti, Eileen, et al.
Publicado: (2020)

TALEN mediated gene editing in a mouse model of Fanconi anemia
por: Pino-Barrio, Maria José, et al.
Publicado: (2020)

Revealing cell populations catching the early stages of human embryo development in naive pluripotent stem cell cultures
por: Moya-Jódar, Marta, et al.
Publicado: (2022)

Reptilian Transcriptomes v2.0: An Extensive Resource for Sauropsida Genomics and Transcriptomics
por: Tzika, Athanasia C., et al.
Publicado: (2015)

Improved collection of hematopoietic stem cells and progenitors from Fanconi anemia patients for gene therapy purposes
por: Sevilla, Julián, et al.
Publicado: (2021)

Somitic positional information guides self-organized patterning of snake scales
por: Tzika, Athanasia C., et al.
Publicado: (2023)

Impact of genome architecture on the functional activation and repression of Hox regulatory landscapes
por: Rodríguez-Carballo, Eddie, et al.
Publicado: (2019)

Epigenetic Alterations in Fanconi Anaemia: Role in Pathophysiology and Therapeutic Potential
por: Belo, Hélio, et al.
Publicado: (2015)

Deconvolution of the hematopoietic stem cell microenvironment reveals a high degree of specialization and conservation
por: Ye, Jin, et al.
Publicado: (2022)

Bcr/Abl Interferes with the Fanconi Anemia/BRCA Pathway: Implications in the Chromosomal Instability of Chronic Myeloid Leukemia Cells
por: Valeri, Antonio, et al.
Publicado: (2010)

Functional and transcriptomic analysis of extracellular vesicles identifies calprotectin as a new prognostic marker in peripheral arterial disease (PAD)
por: Saenz-Pipaon, Goren, et al.
Publicado: (2020)

Upregulation of NKG2D ligands impairs hematopoietic stem cell function in Fanconi anemia
por: Casado, José A., et al.
Publicado: (2022)

One-Step In Vitro Generation of ETV2-Null Pig Embryos
por: Moya-Jódar, Marta, et al.
Publicado: (2022)

Early expression of CD94 and loss of CD96 on CD8+ T cells after allogeneic stem cell tranplantation is predictive of subsequent relapse and survival
por: Verma, Kriti, et al.
Publicado: (2022)

CD56(bright)CD16(-) natural killer cells as an important regulatory mechanism in chronic graft-versus-host disease
por: Lauener, Madeline P., et al.
Publicado: (2022)

A phase II, prospective, randomized, open-label study of defibrotide added to standard-of-care prophylaxis for the prevention of acute graft-versus-host disease after allogeneic hematopoietic cell transplantation
por: Hudspeth, Michelle, et al.
Publicado: (2022)

Transcriptome analysis in acute gastrointestinal graft-versus host disease reveals a unique signature in children and shared biology with pediatric inflammatory bowel disease
por: Khandelwal, Pooja, et al.
Publicado: (2023)

Clonal hematopoiesis in the donor does not adversely affect long-term outcomes following allogeneic hematopoietic stem cell transplantation: result from a 13-year follow-up
por: Kim, Kyoung Ha, et al.
Publicado: (2023)

Allogeneic transplantation in acute myelogenous leukemia: a comprehensive single institution's experience
por: Socie, Gerard, et al.
Publicado: (2023)

Targeted gene therapy and cell reprogramming in Fanconi anemia
por: Rio, Paula, et al.
Publicado: (2014)

Uncovering perturbations in human hematopoiesis associated with healthy aging and myeloid malignancies at single-cell resolution
por: Ainciburu, Marina, et al.
Publicado: (2023)

High content drug screening for Fanconi anemia therapeutics
por: Montanuy, Helena, et al.
Publicado: (2020)

Next-generation Sequencing in Bone Marrow Failure Syndromes and Isolated Cytopenias: Experience of the Spanish Network on Bone Marrow Failure Syndromes
por: Gálvez, Eva, et al.
Publicado: (2021)

Genome mapping of a LYST mutation in corn snakes indicates that vertebrate chromatophore vesicles are lysosome-related organelles
por: Ullate-Agote, Asier, et al.
Publicado: (2020)

Parvovirus B19 in the Context of Hematopoietic Stem Cell Transplantation: Evaluating Cell Donors and Recipients
por: Gama, Bianca E., et al.
Publicado: (2017)

Isolation of a Highly Purified HSC-enriched CD34(+)CD90(+)CD45RA(−) Cell Subset for Allogeneic Transplantation in the Nonhuman Primate Large-animal Model
por: Radtke, Stefan, et al.
Publicado: (2020)

CD94 Ex Vivo Cultures in a Bone Marrow Transplantation Setting
por: Abrams, Kraig, et al.
Publicado: (2020)

Multivariate Analysis of Immune Reconstitution and Relapse Risk Scoring in Children Receiving Allogeneic Stem Cell Transplantation for Acute Leukemias
por: Spadea, Manuela, et al.
Publicado: (2021)

Detection of Antibodies Against Human Leukocyte Antigen Class II in the Sera of Patients Receiving Intravenous Immunoglobulin
por: Takamatsu, Hiroyuki, et al.
Publicado: (2021)

Cannot write session to /tmp/vufind_sessions/sess_so1gpps1jmhjtp95q73iamm3fn