Prime editing can efficiently rewrite the genetic mutation causing sickle cell disease, in haematopoietic stem cells from patients. Sébastien Levesque Daniel E. Bauer News & Views19 May 2023Nature Biomedical Engineering Volume: 7, P: 605-606 ...
Stem-cell transplantation, which has been used to reverse sickle-cell disease in hundreds of children, is being adapted so that adults can benefit (S14). And the advance of gene-editing techniques could soon allow scientists to perform molecular microsurgery, repairing the genetic error that ...
Sickle cell disease (SCD) is a group of inherited disorders caused by mutations in HBB, which encodes haemoglobin subunit β. The incidence is estimated to be between 300,000 and 400,000 neonates globally each year, the majority in sub-Saharan Africa. Haemoglobin molecules that include mutant ...
Sickle cell disease (SCD) is a group of inherited disorders caused by mutations inHBB, which encodes haemoglobin subunit β. The incidence is estimated to be between 300,000 and 400,000 neonates globally each year, the majority in sub-Saharan Africa. Haemoglobin molecules that include mutant sic...
Nature文章:Base editing of haematopoietic stem cells rescues sickle cell disease in mice $Beam Therapeutics(BEAM)$ 2021年6月2日,David Liu 实验室又在Nature杂志上发表了一篇单碱基编辑器治疗小鼠SCD的论文。 之前我的文章有讲到过BEAM在研发BEAM-102时PAM还是有很大的限制。那时PAM主要有NGG,NGC和NGA三种。
The promise of new treatments for sickle cell disease, a group of inherited red blood cell disorders, has never been so great, but it will only be realized if there is constant investment in health policies such as programs for screening newborns, especi
In the present paper, biosynthesis studies performed on reticulocytes from heterozygotes and homozygotes for the Hb S give further indications of the mild nature of sickle-cell disease in Arabia. Comparison of two affected families, from Saudi Arabia and Jordan, showed that clinical manifestation ...
The role of this compound in the therapy of sickle cell disease also remains to be determined. While the reports of urea and cyanate have excited a good deal of interest, other forms of therapy for sickle cell disease and for some of its complications also merit investigation. The efficacy ...
New research on sickle cell disease (SCD) has found that using antibiotics to deplete the body's microbiome may prevent acute sickle cell crisis and could offer the first effective strategy for warding off the disease's long-term complications, such as organ failure. The study, conducted by sc...
Cell-free hemoglobin limits nitric oxide bioavailability in sickle-cell disease. Although the deleterious vasoconstrictive effects of cell-free, -based have been appreciated, the systemic effects of chronic on nitric bioavailability hav... CD Reiter,X Wang,JE Tanus-Santos,... - 《Nature Medicine》...