Dr Antonella Nai (San Raffaele Scientific Institute, Italy) and colleagues combined two experimental approaches to treat thalassemia in a mouse model . They first used TMPRSS6 gene-specific antisense oligonucleotides (ASOs) to decrease excess iron. In addition, they deleted the TFR2 gene in the bone marrow using gene therapy.
The ASO treatment is effective in degrading this TMPRSS6 gene product, a key regulator in iron homeostasis, allowing iron levels to decrease to a healthy level. Dr Nai pointed out that loss-of-function mutations in the TMPRSS6 gene cause an inherited cause of iron-refractory iron-deficient anaemia. This increases erythropoietin sensitivity in the bone marrow cells and improves red blood cell production. This two-pronged approach significantly improved anaemia and increased red blood cell and haemoglobin concentrations in the mouse model. Dr Nai explained that the treatment holds promise for some ...
please login to read the entire article:
You need to register to read the entire article, please do so now.
« Haematopoietic stem cell transplantation improves stroke risk in children with sickle cell anaemia Next Article
Gilteritinib prolongs overall survival in patients with FLT3-mutated relapsed/refractory AML »
Table of Contents: EHA 2019
Necessary cookies are absolutely essential for the website to function properly. This category only includes cookies that ensures basic functionalities and security features of the website. These cookies do not store any personal information.
Any cookies that may not be particularly necessary for the website to function and is used specifically to collect user personal data via analytics, ads, other embedded contents are termed as non-necessary cookies. It is mandatory to procure user consent prior to running these cookies on your website.