Vidcast:  https://youtu.be/Lad0r0yMmMw

 

 A single dose of genetically-altered blood-forming stem cells can eliminate the often disastrous complications of sickle cell disease for up to 3 years.  Clinical researchers at Columbia University in collaboration with those at the NIH now announce the exciting preliminary results of their phase 1/2 trial of gene therapy in an initial cohort of 35 patients.

 

Blood-forming stem cells are harvested from study participants with sickle cell disease, treated with a lentiviral vector that transmits the normalized genetic material into the stem cells, and then reinjected back into the patients in a process known as a LentiGlobulin infusion.  All 35 patients receiving their genetically-mended cells back accepted them.  Of the 25 patients that could be evaluated, all enjoyed resolution of their typically severe sickle cell events.

 

This process is not without risks as those under treatment must undergo a course of ablative chemotherapy to first destroy the sickle-type blood stem cells populating their bone marrows.  Longer term followup and more confirmatory studies are necessary, but the fact that this process works is exciting for those patients and their families who suffer with this generally poorly controllable disease.

 

https://www.nejm.org/doi/10.1056/NEJMoa2117175

https://www.sciencedaily.com/releases/2021/12/211213160126.htm

 

#sicklecelldisease #geneticengineering #lentivirus #lentigloblin