Cultures of Erythroid Precursors in the Screening of Gamma-Globin mRNA and Fetal Haemoglobin Inducers used for a new Approach in Thalassemia and Sickle Cell Anemia Therapy
Poster Feb 27, 2006
Nicoletta Bianchi, Cristina Zucatto, Carlo Mischiati, Ilaria Lampronti, Monica Borgatti, Eugenia Pruss, Eitan Fibach and Roberto Gambari
Our group studied several inducers of erythroid differentiation. In the present study we demonstrate that rapamycin induces an increase of HbFproduction in erythroid precursors. This compound is able to increase gammaglobin gene expression in stem cells from blood of normal subjects as well asbeta-thalassemia patients.
We utilized paired synthetic crRNAs coupled with our synthetic tracrRNA in cells transduced with lentiviral Cas9 to perform a functional knockout on hsa-miR-221. This three-part system (crRNA, tracrRNA and Cas9) has demonstrated efficient gene editing when used with only one guide RNA, but the goal was to use two crRNAs to remove the entire stem-loop.READ MORE