I discussed this project with GeneSmith and I think it is promising, though very challenging to implement in practice. The hardest part will be safely and efficiently delivering the editing agent to a large fraction of the cells in the brain.
Some other points:
CAR T-cell therapy, a treatment for certain types of cancer, requires the removal of white blood cells via IV, genetic modification of those cells outside the body, culturing of the modified cells, chemotherapy to kill off most of the remaining unmodified cells in the body, and reinjection of the genetically engineered ones. The price is $500,000 to $1,000,000.
And it only modifies a single gene.
This makes it sound like CAR-T is gene editing, but it isn’t. Instead of editing a gene, it introduces a new one (a chimeric T-cell receptor). Although some companies are working on gene editing to enhance CAR-Ts.
I also know of a PHD student in George Church’s lab that was able to make several thousand edits in the same cell at the same time by targeting a gene that has several thousand copies spread throughout the genome.
Thanks! I’ve added a link to the paper by Cory Smith and others to the relevant section and updated the section about CAR T-cell therapy to read “adds a single gene” rather than “modifies a single gene”
Consolidating my previous comments:
I discussed this project with GeneSmith and I think it is promising, though very challenging to implement in practice. The hardest part will be safely and efficiently delivering the editing agent to a large fraction of the cells in the brain.
Some other points:
This makes it sound like CAR-T is gene editing, but it isn’t. Instead of editing a gene, it introduces a new one (a chimeric T-cell receptor). Although some companies are working on gene editing to enhance CAR-Ts.
The paper reporting this was here: https://www.ncbi.nlm.nih.gov/pmc/articles/PMC7229841/
Thanks! I’ve added a link to the paper by Cory Smith and others to the relevant section and updated the section about CAR T-cell therapy to read “adds a single gene” rather than “modifies a single gene”