Cellular and Gene Therapies Targeting the Central Nervous System in MPS Disorders

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

Author: Ashok Vellodi

I have had a lifelong interest in lysosomal storage disorders. Having recently retired I now have time to pursue my passion for teaching and training of young doctors and scientists, and hopefully instil in them the passion for their patients that is so integral to working in this field.

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