Publications by authors named "A Chalumeau"
Article Synopsis
- - Sickle cell disease (SCD) is caused by mutations in the hemoglobin beta chain gene, leading to the production of abnormal hemoglobin (HbS), which results in symptoms like anemia and painful vaso-occlusive events.
- - Gene therapy shows promise for treating SCD, with two specific therapies already on the market, but there is a need to enhance their effectiveness, particularly with strategies that involve transferring a corrective beta-globin gene.
- - Research demonstrates that using cyclosporin H (CsH) during the gene therapy process can improve transgene expression and the number of gene copies in stem cells from cord blood, making it a potential effective combination for treating SCD.
Fetal hemoglobin (HbF) reactivation expression through CRISPR-Cas9 is a promising strategy for the treatment of sickle cell disease (SCD). Here, we describe a genome editing strategy leading to reactivation of HbF expression by targeting the binding sites (BSs) for the lymphoma-related factor (LRF) repressor in the γ-globin promoters. CRISPR-Cas9 treatment in healthy donor (HD) and patient-derived HSPCs resulted in a high frequency of LRF BS disruption and potent HbF synthesis in their erythroid progeny.
View Article and Find Full Text PDFSickle cell disease is a devastating blood disorder that originates from a single point mutation in the HBB gene coding for hemoglobin. Here, we develop a GMP-compatible TALEN-mediated gene editing process enabling efficient HBB correction via a DNA repair template while minimizing risks associated with HBB inactivation. Comparing viral versus non-viral DNA repair template delivery in hematopoietic stem and progenitor cells in vitro, both strategies achieve comparable HBB correction and result in over 50% expression of normal adult hemoglobin in red blood cells without inducing β-thalassemic phenotype.
View Article and Find Full Text PDFArticle Synopsis
- Pulsar Timing Array experiments are used to investigate the existence of ultralight dark matter particles by observing an ensemble of millisecond radio pulsars over several years.
- The second data release from the European Pulsar Timing Array focuses on a scenario where dark matter only interacts with regular matter through gravity.
- The findings indicate that while ultralight dark matter particles in a specific mass range cannot make up all of the local dark matter density, they can exist at a maximum local density of around 0.3 GeV/cm³.
Article Synopsis
- * A new treatment involves using special medicine (lentiviral vectors) to help make a better type of hemoglobin and reduce the bad HbS, which is getting better results than previous methods.
- * This new treatment is safe, doesn't harm blood cell development, and combines two strategies to help the body fight SCD more effectively.