Cargando…

A Rational Approach to Drug Repositioning in β-thalassemia: Induction of Fetal Hemoglobin by Established Drugs

Drug repositioning and the relevance of orphan drug designation for β-thalassemia is reviewed. Drug repositioning and similar terms ('drug repurposing', 'drug reprofiling', 'drug redirecting', ‘drug rescue’, ‘drug re-tasking’ and/or 'drug rediscovery') have ga...

Descripción completa

Detalles Bibliográficos
Autores principales:	Prosdocimi, Marco, Zuccato, Cristina, Cosenza, Lucia Carmela, Borgatti, Monica, Lampronti, Ilaria, Finotti, Alessia, Gambari, Roberto
Formato:	Online Artículo Texto
Lenguaje:	English
Publicado:	F1000 Research Limited 2022
Materias:	Review
Acceso en línea:	https://www.ncbi.nlm.nih.gov/pmc/articles/PMC9453112/ https://www.ncbi.nlm.nih.gov/pubmed/36110836 http://dx.doi.org/10.12688/wellcomeopenres.17845.3

Ejemplares similares

Treatment of Erythroid Precursor Cells from β-Thalassemia Patients with Cinchona Alkaloids: Induction of Fetal Hemoglobin Production
por: Zuccato, Cristina, et al.
Publicado: (2021)

5613398 A DRUG REPOSITIONING PROJECT FOR Β-THALASSEMIA: SIROLIMUS AND CINCHONA ALKALOIDS
por: Michele Lipucci Di Paola, M.L.P., et al.
Publicado: (2023)

An Aγ-globin G->A gene polymorphism associated with β(0)39 thalassemia globin gene and high fetal hemoglobin production
por: Breveglieri, Giulia, et al.
Publicado: (2017)

Fetal Hemoglobin Inducers from the Natural World: A Novel Approach for Identification of Drugs for the Treatment of β-Thalassemia and Sickle-Cell Anemia
por: Bianchi, Nicoletta, et al.
Publicado: (2009)

The rs368698783 (G>A) Polymorphism Affecting LYAR Binding to the Aγ-Globin Gene Is Associated with High Fetal Hemoglobin (HbF) in β-Thalassemia Erythroid Precursor Cells Treated with HbF Inducers
por: Zuccato, Cristina, et al.
Publicado: (2023)

Co-Treatment of Erythroid Cells from β-Thalassemia Patients with CRISPR-Cas9-Based β(0)39-Globin Gene Editing and Induction of Fetal Hemoglobin
por: Cosenza, Lucia Carmela, et al.
Publicado: (2022)

A combined approach for β-thalassemia based on gene therapy-mediated adult hemoglobin (HbA) production and fetal hemoglobin (HbF) induction
por: Zuccato, Cristina, et al.
Publicado: (2012)

Effects of Sirolimus Treatment on Fetal Hemoglobin Production and Response to SARS-CoV-2 Vaccination: A Case Report Study
por: Gamberini, Maria Rita, et al.
Publicado: (2023)

The Long Scientific Journey of Sirolimus (Rapamycin): From the Soil of Easter Island (Rapa Nui) to Applied Research and Clinical Trials on β-Thalassemia and Other Hemoglobinopathies
por: Gambari, Roberto, et al.
Publicado: (2023)

5613423 TREATMENT OF ERYTHROID PRECURSOR CELLS FROM β-THALASSEMIC PATIENTS WITH ISOXAZOLE DERIVATIVES: POTENT INDUCTION OF FETAL HEMOGLOBIN
por: Cristina Zuccato, C.Z., et al.
Publicado: (2023)

5613417 CO-TREATMENT OF ERYTHROID CELLS ISOLATED FROM Β039-THALASSEMIA PATIENTS WITH CRISPR-CAS9 GENE EDITING AND FETAL HEMOGLOBIN INDUCTION
por: Lipucci di Paola, M.L.P., et al.
Publicado: (2023)

Expression of γ-globin genes in β-thalassemia patients treated with sirolimus: results from a pilot clinical trial (Sirthalaclin)
por: Zuccato, Cristina, et al.
Publicado: (2022)

A validated cellular biobank for β-thalassemia
por: Cosenza, Lucia Carmela, et al.
Publicado: (2016)

Decrease in α-Globin and Increase in the Autophagy-Activating Kinase ULK1 mRNA in Erythroid Precursors from β-Thalassemia Patients Treated with Sirolimus
por: Zurlo, Matteo, et al.
Publicado: (2023)

Efficient CRISPR-Cas9-based genome editing of β-globin gene on erythroid cells from homozygous β(0)39-thalassemia patients
por: Cosenza, Lucia Carmela, et al.
Publicado: (2021)

Discovery of Novel Fetal Hemoglobin Inducers through Small Chemical Library Screening
por: Breveglieri, Giulia, et al.
Publicado: (2020)

Development and characterization of K562 cell clones expressing BCL11A-XL: Decreased hemoglobin production with fetal hemoglobin inducers and its rescue with mithramycin
por: Finotti, Alessia, et al.
Publicado: (2015)

UPF1 silenced cellular model systems for screening of read-through agents active on β(0)39 thalassemia point mutation
por: Salvatori, Francesca, et al.
Publicado: (2018)

Combined approaches for increasing fetal hemoglobin (HbF) and de novo production of adult hemoglobin (HbA) in erythroid cells from β-thalassemia patients: treatment with HbF inducers and CRISPR-Cas9 based genome editing
por: Finotti, Alessia, et al.
Publicado: (2023)

Effects of Sirolimus treatment on patients with β‐Thalassemia: Lymphocyte immunophenotype and biological activity of memory CD4 (+) and CD8 (+) T cells
por: Zurlo, Matteo, et al.
Publicado: (2023)

Generation and Characterization of a Transgenic Mouse Carrying a Functional Human β-Globin Gene with the IVSI-6 Thalassemia Mutation
por: Breveglieri, Giulia, et al.
Publicado: (2015)

MicroRNAs and Long Non-coding RNAs in Genetic Diseases
por: Finotti, Alessia, et al.
Publicado: (2019)

Increase of microRNA-210, Decrease of Raptor Gene Expression and Alteration of Mammalian Target of Rapamycin Regulated Proteins following Mithramycin Treatment of Human Erythroid Cells
por: Bianchi, Nicoletta, et al.
Publicado: (2015)

Role of Cystic Fibrosis Bronchial Epithelium in Neutrophil Chemotaxis
por: Cabrini, Giulio, et al.
Publicado: (2020)

Effects of Mithramycin on BCL11A Gene Expression and on the Interaction of the BCL11A Transcriptional Complex to γ-Globin Gene Promoter Sequences
por: Finotti, Alessia, et al.
Publicado: (2023)

Recent trends in the gene therapy of β-thalassemia
por: Finotti, Alessia, et al.
Publicado: (2015)

Targeting miR-155-5p and miR-221-3p by peptide nucleic acids induces caspase-3 activation and apoptosis in temozolomide-resistant T98G glioma cells
por: Milani, Roberta, et al.
Publicado: (2019)

Teaching during COVID-19 pandemic in practical laboratory classes of applied biochemistry and pharmacology: A validated fast and simple protocol for detection of SARS-CoV-2 Spike sequences
por: Gasparello, Jessica, et al.
Publicado: (2022)

Production and Characterization of K562 Cellular Clones Hyper-Expressing the Gene Encoding α-Globin: Preliminary Analysis of Biomarkers Associated with Autophagy
por: Zurlo, Matteo, et al.
Publicado: (2023)

PCR detection of segmented filamentous bacteria in the terminal ileum of patients with ulcerative colitis
por: Finotti, Alessia, et al.
Publicado: (2017)

Pro-apoptotic activity of novel synthetic isoxazole derivatives exhibiting inhibitory activity against tumor cell growth in vitro
por: Lampronti, Ilaria, et al.
Publicado: (2020)

Peptide nucleic acids targeting β-globin mRNAs selectively inhibit hemoglobin production in murine erythroleukemia cells
por: MONTAGNER, GIULIA, et al.
Publicado: (2015)

Postnatal and non-invasive prenatal detection of β-thalassemia mutations based on Taqman genotyping assays
por: Breveglieri, Giulia, et al.
Publicado: (2017)

Induction of erythroid differentiation and increased globin mRNA production with furocoumarins and their photoproducts
por: Salvador, Alessia, et al.
Publicado: (2013)

BCL11A mRNA Targeting by miR-210: A Possible Network Regulating γ-Globin Gene Expression
por: Gasparello, Jessica, et al.
Publicado: (2017)

Detection of the sickle hemoglobin allele using a surface plasmon resonance based biosensor
por: Breveglieri, Giulia, et al.
Publicado: (2019)

The Cystic Fibrosis Transmembrane Conductance Regulator Gene (CFTR) Is under Post-Transcriptional Control of microRNAs: Analysis of the Effects of agomiRNAs Mimicking miR-145-5p, miR-101-3p, and miR-335-5p
por: Papi, Chiara, et al.
Publicado: (2023)

Modulation of the Expression of the Proinflammatory IL-8 Gene in Cystic Fibrosis Cells by Extracts Deriving from Olive Mill Waste Water
por: Lampronti, Ilaria, et al.
Publicado: (2013)

Effects of decoy molecules targeting NF-kappaB transcription factors in Cystic fibrosis IB3–1 cells: Recruitment of NF-kappaB to the IL-8 gene promoter and transcription of the IL-8 gene
por: Finotti, Alessia, et al.
Publicado: (2012)

A Peptide Nucleic Acid (PNA) Masking the miR-145-5p Binding Site of the 3′UTR of the Cystic Fibrosis Transmembrane Conductance Regulator (CFTR) mRNA Enhances CFTR Expression in Calu-3 Cells
por: Sultan, Shaiq, et al.
Publicado: (2020)

Cannot write session to /tmp/vufind_sessions/sess_cpar5iqpk16dilh6v04e32ov0k