<
https://arstechnica.com/science/2024/06/researchers-do-gene-editing-in-lung-stem-cells-target-cystic-fibrosis/>
"The development of gene editing tools, which enable the specific targeting and
correction of mutations, hold the promise of allowing us to correct those
mutations that cause genetic diseases. However, the technology has been around
for a while now—two researchers were critical to its development in 2020—and
there have been only a few cases where gene editing has been used to target
diseases.
One of the reasons for that is the challenge of targeting specific cells in a
living organism. Many genetic diseases affect only a specific cell type, such
as red blood cells in sickle-cell anemia, or specific tissue. Ideally, to limit
potential side effects, we'd like to ensure that enough of the editing takes
place in the affected tissue to have an impact, while minimizing editing
elsewhere to limit side effects. But our ability to do so has been limited.
Plus, a lot of the cells affected by genetic diseases are mature and have
stopped dividing. So, we either need to repeat the gene editing treatments
indefinitely or find a way to target the stem cell population that produces the
mature cells.
On Thursday, a US-based research team said that they've done gene editing
experiments that targeted a high-profile genetic disease: cystic fibrosis.
Their technique largely targets the tissue most affected by the disease (the
lung), and occurs in the stem cell populations that produce mature lung cells,
ensuring that the effect is stable."
Via
Fix the News:
<
https://fixthenews.com/good-news-sleeping-sickness-chad-forests-ukraine-amur-leopards-in-siberia/>
Cheers,
*** Xanni ***
--
mailto:xanni@xanadu.net Andrew Pam
http://xanadu.com.au/ Chief Scientist, Xanadu
https://glasswings.com.au/ Partner, Glass Wings
https://sericyb.com.au/ Manager, Serious Cybernetics
