Cargando…

Correction of β-thalassemia major by gene transfer in haematopoietic progenitors of pediatric patients

β-Thalassemia is a common monogenic disorder due to mutations in the β-globin gene and gene therapy, based on autologous transplantation of genetically corrected haematopoietic stem cells (HSCs), holds the promise to treat patients lacking a compatible bone marrow (BM) donor. We recently showed corr...

Descripción completa

Detalles Bibliográficos
Autores principales:	Roselli, Emanuela Anna, Mezzadra, Riccardo, Frittoli, Marta Claudia, Maruggi, Giulietta, Biral, Erika, Mavilio, Fulvio, Mastropietro, Fabrizio, Amato, Antonio, Tonon, Giovanni, Refaldi, Chiara, Cappellini, Maria Domenica, Andreani, Marco, Lucarelli, Guido, Roncarolo, Maria Grazia, Marktel, Sarah, Ferrari, Giuliana
Formato:	Online Artículo Texto
Lenguaje:	English
Publicado:	WILEY-VCH Verlag 2010
Materias:	Research Articles
Acceso en línea:	https://www.ncbi.nlm.nih.gov/pmc/articles/PMC3377331/ https://www.ncbi.nlm.nih.gov/pubmed/20665635 http://dx.doi.org/10.1002/emmm.201000083

Ejemplares similares

High incidence of severe cyclosporine neurotoxicity in children affected by haemoglobinopaties undergoing myeloablative haematopoietic stem cell transplantation: early diagnosis and prompt intervention ameliorates neurological outcome
por: Noè, Anna, et al.
Publicado: (2010)

Right in time: Mitapivat for the treatment of anemia in α- and β-thalassemia
por: Musallam, Khaled M., et al.
Publicado: (2022)

β-Thalassemia: New Therapeutic Modalities, Genetics, Complications, and Quality of Life
por: Karimi, Mehran, et al.
Publicado: (2012)

Beta Thalassemia: New Therapeutic Options Beyond Transfusion and Iron Chelation
por: Motta, Irene, et al.
Publicado: (2020)

Health related quality of life in Middle Eastern children with beta-thalassemia
por: Caocci, Giovanni, et al.
Publicado: (2012)

Non-Transfusion-Dependent Thalassemia: An Update on Complications and Management
por: Sleiman, Joseph, et al.
Publicado: (2018)

Coagulopathy in Beta-Thalassemia: Current Understanding and Future Perspectives
por: Cappellini, M. Domenica, et al.
Publicado: (2009)

2021 Thalassaemia International Federation Guidelines for the Management of Transfusion-dependent Thalassemia
por: Farmakis, Dimitrios, et al.
Publicado: (2022)

Iron Overload and Chelation Therapy in Non-Transfusion Dependent Thalassemia
por: Bou-Fakhredin, Rayan, et al.
Publicado: (2017)

Treating iron overload in patients with non-transfusion-dependent thalassemia
por: Taher, Ali T, et al.
Publicado: (2013)

Luspatercept for transfusion-dependent β-thalassemia: time to get real
por: Musallam, Khaled M., et al.
Publicado: (2023)

Therapeutic perspective for children and young adults living with thalassemia and sickle cell disease
por: Ferraresi, Marta, et al.
Publicado: (2023)

Pharmacological Induction of Fetal Hemoglobin in β-Thalassemia and Sickle Cell Disease: An Updated Perspective
por: Bou-Fakhredin, Rayan, et al.
Publicado: (2022)

BAT2 and BAT3 polymorphisms as novel genetic risk factors for rejection after HLA-related stem cell transplantation
por: Piras, Ignazio Stefano, et al.
Publicado: (2014)

EHA Research Roadmap on Hemoglobinopathies and Thalassemia: An Update
por: Iolascon, Achille, et al.
Publicado: (2019)

Pregnant women affected by thalassemia major: a controlled study of traits and personality
por: Messina, Giuseppina, et al.
Publicado: (2010)

Redox Balance in β-Thalassemia and Sickle Cell Disease: A Love and Hate Relationship
por: Bou-Fakhredin, Rayan, et al.
Publicado: (2022)

Untreated Anemia in Nontransfusion-dependent β-thalassemia: Time to Sound the Alarm
por: Musallam, Khaled M., et al.
Publicado: (2022)

T cell-depleted hla-haploidentical stem cell transplantation in thalassemia young patients
por: Sodani, Pietro, et al.
Publicado: (2011)

Relationship between Iron deposition and T lymphocytes in children with β-thalassemia with haematopoietic stem cell transplantation
por: Zhou, Yuhang, et al.
Publicado: (2022)

Advances in mRNA Vaccines for Infectious Diseases
por: Zhang, Cuiling, et al.
Publicado: (2019)

Bone marrow harvesting from paediatric patients undergoing haematopoietic stem cell gene therapy
por: Tucci, Francesca, et al.
Publicado: (2019)

Profile of Luspatercept in the Treatment of Anemia in Adults with Non-Transfusion-Dependent β-Thalassemia (NTDT): Design, Development and Potential Place in Therapy
por: Musallam, Khaled M, et al.
Publicado: (2023)

High-Definition Mapping of Retroviral Integration Sites Defines the Fate of Allogeneic T Cells After Donor Lymphocyte Infusion
por: Cattoglio, Claudia, et al.
Publicado: (2010)

Efficacy and safety of iron-chelation therapy with deferoxamine, deferiprone, and deferasirox for the treatment of iron-loaded patients with nontransfusion-dependent thalassemia syndromes
por: Taher, Ali T, et al.
Publicado: (2016)

P1519: EXPLORING THE ROLE OF FGF23 IN BETA-THALASSEMIA: A NOVEL THERAPEUTIC TARGET TO AMELIORATE ALTERED INTERACTIONS BETWEEN HSC AND BONE MARROW NICHE
por: Raggi, L., et al.
Publicado: (2022)

Multiple Integrated Non-clinical Studies Predict the Safety of Lentivirus-Mediated Gene Therapy for β-Thalassemia
por: Lidonnici, Maria Rosa, et al.
Publicado: (2018)

CYP450 Mediates Reactive Oxygen Species Production in a Mouse Model of β-Thalassemia through an Increase in 20-HETE Activity
por: Bou-Fakhredin, Rayan, et al.
Publicado: (2021)

Bone marrow stromal cells from β-thalassemia patients have impaired hematopoietic supportive capacity
por: Crippa, Stefania, et al.
Publicado: (2019)

Generation of an immortalised erythroid cell line from haematopoietic stem cells of a haemoglobin E/β-thalassemia patient
por: Trakarnsanga, Kongtana, et al.
Publicado: (2020)

Bone marrow transplantation for thalassemia
por: Lucarelli, G., et al.
Publicado: (1993)

Exjade® (deferasirox, ICL670) in the treatment of chronic iron overload associated with blood transfusion
por: Cappellini, Maria Domenica
Publicado: (2007)

Advances in hemoglobin disorders
por: Cappellini, Maria Domenica
Publicado: (2018)

Validation of a patient‐reported outcomes symptom measure for patients with nontransfusion‐dependent thalassemia (NTDT‐PRO(©))
por: Taher, Ali, et al.
Publicado: (2018)

Recapitulative haematopoietic development of human pluripotent stem cells in the absence of exogenous haematopoietic cytokines
por: Philonenko, Elena S., et al.
Publicado: (2021)

Low-dose Synachten test with measurement of salivary cortisol in adult patients with β-thalassemia major
por: Ambrogio, Alberto G., et al.
Publicado: (2018)

Development of a patient‐reported outcomes symptom measure for patients with nontransfusion‐dependent thalassemia (NTDT‐PRO(©))
por: Taher, Ali, et al.
Publicado: (2018)

Tricuspid-valve regurgitant jet velocity as a risk factor for death in β-thalassemia
por: Derchi, Giorgio, et al.
Publicado: (2022)

P1466: EFFECT OF LUSPATERCEPT ON BONE MINERAL DENSITY IN PATIENTS WITH BETA‑THALASSEMIA ENROLLED IN THE PHASE 3 BELIEVE TRIAL
por: Coates, Thomas D., et al.
Publicado: (2023)

Clonality in haematopoietic stem cell ageing
por: Terradas-Terradas, Maria, et al.
Publicado: (2020)

Cannot write session to /tmp/vufind_sessions/sess_6mf3jf1qjdtshkmvtpaog1pnab