An engineered lentivirus delivers functional copies of a modified form of the β-globin gene (βA-T87Q-globin gene or LentiGlobin) into a sickle cell disease (SCD) patient’s own haematopoietic stem cells. Once patients have the βA-T87Q-globin gene, they can make functional red blood cells, with the goal of reducing sickled red blood cells, haemolysis, and ensuing complications . In patients who were at least 6 months post-treatment with lentiviral LentiGlobin for SCD, the median level of abnormal sickle haemoglobin was reduced to ≤50% of total haemoglobin. At up to 15 months post-treatment with LentiGlobin, no serious vaso-occlusive crisis (VOEs) or acute chest syndrome were reported in this cohort.
In the plenary session, Dr Olivier Hermine (Necker Hospital, France) presented the ongoing, phase 1/2 HGB-206 study. Adults and children living with SCD experience unpredictable episodes of pain due...
please login to read the entire article:
You need to register to read the entire article, please do so now.
« Letter from the Editor Next Article
Guadecitabine vs treatment of choice in 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.