Antisense‐mediated therapeutic pseudoexon skipping in TMEM165‐CDG

Deficiencies in glycosyltransferases, glycosidases or nucleotide‐sugar transporters involved in protein glycosylation lead to congenital disorders of glycosylation ( CDG ), a group of genetic diseases mostly showing multisystem phenotype. Despite recent advances in the biochemical and molecular knowledge of these diseases, no effective therapy exists for most. Efforts are now being directed toward therapies based on identifying new targets,...