3 ms·

I may be way out of my depth, but until we can edit all the cells of a living organism, isn't CRISPR limited to working with pre-embryos/embryos? If so, other t

by pdevr 5y ago

I may be way out of my depth, but until we can edit all the cells of a living organism, isn't CRISPR limited to working with pre-embryos/embryos? If so, other than creating a potential super race, what is it of benefit to the living individuals with "imperfect" genes? Just to clarify, that is a real question, not rhetoric.

furstenheim 5y ago
They applied crispr to bone marrow. Extract it, apply crispr. Cleanse the body to remove existing one and apply modified barrow. Basically an auto transplant with marrow
- BizarroLand 5y ago
  I wonder if that can be used as a treatment in conjunction with finding the gene that gives some people immunity to HIV
  - ritwikr 5y ago
    That is being tried, and worked well on one patient it seems, and a second more recent one: https://www.nature.com/articles/d41586-019-00798-3 https://www.nature.com/articles/d41586-019-00798-3
dtech 5y ago
Gene editing has a lot of medical potential outside of the sci-fi utopia/dystopia stuff. Especially diseases caused by a straightforward well-known genetic cause [1], such as sickle-cell disease, might very well be permanently treatable in the next decade or 2. There's also more far-out ideas that might be viable involving permanently affecting the bodies biological machinery to improve it, like adding extra insulin producing capability to cells in diabetic patients. [1] https://www.ncbi.nlm.nih.gov/books/NBK132154/ https://www.ncbi.nlm.nih.gov/books/NBK132154/
axg11 5y ago
(Context: I have a PhD in genomics - but not necessarily an expert in this sub-field) It is possible, but still difficult, to edit cells in-place. One example is the liver, a highly vascularized tissue (lots of blood vessels). This means that you can use a blood-based delivery method, e.g. blood infusion of CRISPR components + other compounds that allow these components to enter cells. Over time, more of the liver cells are edited and you can reach a reasonable proportion of the cells using this method using current techniques (40-90%). Note that for many diseases you would not have to reach 100% of the cells in order to have a positive effect on health. There are a few labs working on better targeted delivery methods. In general the approach is to "wrap" CRISPR components in an envelope that is able to target specific cell-surface proteins for the cell type of interest.
- pdevr 5y ago
  >>the approach is to "wrap" CRISPR components in an envelope that is able to target specific cell-surface proteins for the cell type of interest. Fascinating, thanks.

by pdevr 5y ago

furstenheim 5y ago

They applied crispr to bone marrow. Extract it, apply crispr. Cleanse the body to remove existing one and apply modified barrow. Basically an auto transplant with marrow

BizarroLand 5y ago
I wonder if that can be used as a treatment in conjunction with finding the gene that gives some people immunity to HIV
- ritwikr 5y ago
  That is being tried, and worked well on one patient it seems, and a second more recent one: https://www.nature.com/articles/d41586-019-00798-3 https://www.nature.com/articles/d41586-019-00798-3

dtech 5y ago

Gene editing has a lot of medical potential outside of the sci-fi utopia/dystopia stuff. Especially diseases caused by a straightforward well-known genetic cause [1], such as sickle-cell disease, might very well be permanently treatable in the next decade or 2. There's also more far-out ideas that might be viable involving permanently affecting the bodies biological machinery to improve it, like adding extra insulin producing capability to cells in diabetic patients. [1] https://www.ncbi.nlm.nih.gov/books/NBK132154/ https://www.ncbi.nlm.nih.gov/books/NBK132154/

axg11 5y ago

(Context: I have a PhD in genomics - but not necessarily an expert in this sub-field) It is possible, but still difficult, to edit cells in-place. One example is the liver, a highly vascularized tissue (lots of blood vessels). This means that you can use a blood-based delivery method, e.g. blood infusion of CRISPR components + other compounds that allow these components to enter cells. Over time, more of the liver cells are edited and you can reach a reasonable proportion of the cells using this method using current techniques (40-90%). Note that for many diseases you would not have to reach 100% of the cells in order to have a positive effect on health. There are a few labs working on better targeted delivery methods. In general the approach is to "wrap" CRISPR components in an envelope that is able to target specific cell-surface proteins for the cell type of interest.

pdevr 5y ago
>>the approach is to "wrap" CRISPR components in an envelope that is able to target specific cell-surface proteins for the cell type of interest. Fascinating, thanks.