First Patients to Get Crispr Gene-Editing Treatment Continue to Thrive(npr.org)
npr.org
First Patients to Get Crispr Gene-Editing Treatment Continue to Thrive
https://www.npr.org/sections/health-shots/2020/12/15/944184405/1st-patients-to-get-crispr-gene-editing-treatment-continue-to-thrive
3 comments
An aside: the text-only version of the NPR website is wonderfully clear and legible. Here is the original article in the text version: https://text.npr.org/2020/12/15/944184405/1st-patients-to-ge...
This is amazing and honestly it makes me a bit emotional that we have this technology now. It might be seen as a breakthrough on the level of the development of antibiotics, not yet but if the technology can be expanded and adapted, it could.
For the treatment, doctors remove stem cells from the patients' bone marrow and use CRISPR to edit a gene in the cells, activating the production of fetal hemoglobin. That protein is produced by fetuses in the womb but usually shuts off shortly after birth.
The patients then undergo a grueling round of chemotherapy to destroy most of their bone marrow to make room for the gene-edited cells, billions of which are then infused into their bodies.
This means that it wasn't in vivo modification, which would be truly revolutionary for many genetic diseases.
Still, it's spectacular progress that will help millions eventually.