Biodrugs · 7 authors, 3 centres
This summary was generated by AI from a single paper. It has not been reviewed by a clinician and is not clinical advice. Verify against the source before acting on it.
The central finding is that splice-switching ASOs represent a promising individualized molecular therapy for IEMs, supported by the FDA-permitted N-of-1 study of milasen for Batten disease and preclinical evaluations in patient cells across multiple IEM subtypes.
The review highlights that eight ASOs received FDA marketing approval during the reviewed period, and that the 2018 FDA permission for milasen's N-of-1 study in CLN7 Batten disease represents a milestone for personalized ASO therapy. The authors suggest future directions including novel nucleotide chemistries, mixmer designs, and ASO cocktail strategies.
