Accessing the CNS in MPS disorders remains elusive. Despite recent advances, significant challenges remain. However, some progress is being made.
This article provides a comprehensive review of recent advances, describing the challenges and how they might be overcome. Particular attention is paid to gene editing and base editing, and importantly how these therapies can be delivered to the CNS.
Cell and Gene Therapies for Mucopolysaccharidoses – base editing and therapeutic delivery to the CNS
For a more detailed review of recent advances in gene editing, here is a good recent review.
Sharpening the Molecular Scissors Advances in Gene Editing Technology
