Add to ORKGThe general aim of this proposal is to analyse the gene expression of erythroid progenitor cells from beta−thalassemia patients, for the identification of approaches enhabling the induction of fetal hemoglobin (HbF) and production of adult hemoglobin (HbA), of possible interest for the therapy of beta−thalassemia. The objective of Task 1 is the recruitment and genetic characterization of the beta−thalassemia patients involved in this study. The objective of Task 2 is the characterization of the molecular mechanisms of action of novel HbF inducers and their use in combination with gene therapy employing lentiviral vectors. The objective of Task 3 is the study of gene expression in HPFH patients and in beta−thalassemia patients produci...
The research described in the present PhD Thesis has been conducted in the context of a multicenter ...
Based upon the lack of clinical samples available for research in many laboratories worldwide, a sig...
Based upon the lack of clinical samples available for research in many laboratories worldwide, a sig...
During the last few years genome-wide association studies identified new loci and variants affecting...
textabstractThe hematopoietic system provides one of the most attractive systems for studies on dev...
beta-Thalassemia and sickle cell disease both display a great deal of phenotypic heterogeneity, desp...
β-thalassemias are caused by nearly 300 mutations of the β-globin gene, leading to a low or absent p...
In vitro erythroid differentiation from primary human cells is valuable to develop genetic strategie...
The development of cellular systems for the screening of molecules able to induce the production of ...
Background and Aim: Understanding the molecular mechanisms involved in the increased levels of HbF i...
Gene therapy might fall short in achieving a complete reversion of the β-thalassemic phenotype due t...
The identification of molecules useful for pharmacologically-mediated regulation of the expression o...
Fundação de Amparo à Pesquisa do Estado de São Paulo (FAPESP)Conselho Nacional de Desenvolvimento Ci...
Beta-thalassemia is a common monogenic disorder due to mutations in the beta-globin gene and gene th...
<div><p>Based upon the lack of clinical samples available for research in many laboratories worldwid...
The research described in the present PhD Thesis has been conducted in the context of a multicenter ...
Based upon the lack of clinical samples available for research in many laboratories worldwide, a sig...
Based upon the lack of clinical samples available for research in many laboratories worldwide, a sig...
During the last few years genome-wide association studies identified new loci and variants affecting...
textabstractThe hematopoietic system provides one of the most attractive systems for studies on dev...
beta-Thalassemia and sickle cell disease both display a great deal of phenotypic heterogeneity, desp...
β-thalassemias are caused by nearly 300 mutations of the β-globin gene, leading to a low or absent p...
In vitro erythroid differentiation from primary human cells is valuable to develop genetic strategie...
The development of cellular systems for the screening of molecules able to induce the production of ...
Background and Aim: Understanding the molecular mechanisms involved in the increased levels of HbF i...
Gene therapy might fall short in achieving a complete reversion of the β-thalassemic phenotype due t...
The identification of molecules useful for pharmacologically-mediated regulation of the expression o...
Fundação de Amparo à Pesquisa do Estado de São Paulo (FAPESP)Conselho Nacional de Desenvolvimento Ci...
Beta-thalassemia is a common monogenic disorder due to mutations in the beta-globin gene and gene th...
<div><p>Based upon the lack of clinical samples available for research in many laboratories worldwid...
The research described in the present PhD Thesis has been conducted in the context of a multicenter ...
Based upon the lack of clinical samples available for research in many laboratories worldwide, a sig...
Based upon the lack of clinical samples available for research in many laboratories worldwide, a sig...