"Why study genetic diseases when you cannot treat them?" We've seen these comments from grants' reviewers way too many times
Wrong! Genetics offers a myriad of possibilities for targeted treatments: here is one and it's happening now!
@asnpublications.bsky.social

https://journals.lww.com/jasn/fulltext/2024/12000/treating_the_untreatable__antisense.19.aspx