Paper 1 is pretty interesting and is related to one of the methods of brain delivery I’ve looked at before.
I’m not sure we really want to have a lot of T cells floating around inside the central nervous system, but a friend of mine who spent a few days looking into this earlier this year thought we might be able to repurpose the same tech with synthetic circuits to use microglia cells in the brain to do delivery.
Microglia (and to a lesser extent oligodendrocytes and astrocytes) naturally migrate around the brain. If you could get them to deliver RNA, DNA or RNP payloads to cells when they encounter a tissue-specific cell surface receptor, that might actually solve the brain delivery issue better than any other method.
This would take a lot of work to develop, but if you managed to get it working you could potentially continuously dose a patient’s brain with an arbitrary protein for an indefinite time period. That could be pretty damn valuable.
Alternatively you might be able to use a conditionally activated system like Tet-On to temporarily turn on expression of gene editors or gene editor RNA for some time period to do editing.
https://www.science.org/doi/10.1126/science.adl4237 https://www.science.org/doi/10.1126/science.adt9921
Paper 1 is pretty interesting and is related to one of the methods of brain delivery I’ve looked at before.
I’m not sure we really want to have a lot of T cells floating around inside the central nervous system, but a friend of mine who spent a few days looking into this earlier this year thought we might be able to repurpose the same tech with synthetic circuits to use microglia cells in the brain to do delivery.
Microglia (and to a lesser extent oligodendrocytes and astrocytes) naturally migrate around the brain. If you could get them to deliver RNA, DNA or RNP payloads to cells when they encounter a tissue-specific cell surface receptor, that might actually solve the brain delivery issue better than any other method.
This would take a lot of work to develop, but if you managed to get it working you could potentially continuously dose a patient’s brain with an arbitrary protein for an indefinite time period. That could be pretty damn valuable.
Alternatively you might be able to use a conditionally activated system like Tet-On to temporarily turn on expression of gene editors or gene editor RNA for some time period to do editing.