Some genetic diseases (like certain muscular dystrophies) are caused by mutations that effect the production of functioning protein. Most of the treatments being investigated try to tackle this problem at a genetic level (exon skipping, RNA, Viral Vectors, etc.)
My question is - why can't you inject the missing protein directly into the muscle cell? Like how a diabetic gets insulin? Assuming the only problem here is the production of the protein.
My question is - why can't you inject the missing protein directly into the muscle cell? Like how a diabetic gets insulin? Assuming the only problem here is the production of the protein.
