The 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 producing very high levels of HbF. The objective of Task 4 is the analysis of micro RNA network in HPFH cells and in erythroid progenitors induced to HbF production. The objective of Task 5 is the application of the read−through approach on HbA production by beta°39 thalassemic cells and transgenic animals. The objective of Task 6 is the study of the effects of siRNAs or antisense DNA or DNA−PNA chimeras on target mRNA sequences. Task 7 is dedicated to exploitation, for the identification of Biotech companies interested in the developed protocols and patents.
Hemoglobin production in beta-thalassemia erythroid cells following alteration of biomolecular pathways regulating globin gene expression. Durata: 24 mesi (dal 26/11/2010 al 26/11/2012). Progetto n°: GGP10214. Finanziato da: TELETHON 2010.
GAMBARI, Roberto
2010
Abstract
The 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 producing very high levels of HbF. The objective of Task 4 is the analysis of micro RNA network in HPFH cells and in erythroid progenitors induced to HbF production. The objective of Task 5 is the application of the read−through approach on HbA production by beta°39 thalassemic cells and transgenic animals. The objective of Task 6 is the study of the effects of siRNAs or antisense DNA or DNA−PNA chimeras on target mRNA sequences. Task 7 is dedicated to exploitation, for the identification of Biotech companies interested in the developed protocols and patents.I documenti in SFERA sono protetti da copyright e tutti i diritti sono riservati, salvo diversa indicazione.
