Delivering gene therapy for mucopolysaccharide diseases

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Shaun R Wood1, Brian W Bigger1

  • 1Stem Cell and Neurotherapies Group, School of Biological Sciences, Faculty of Biology, Medicine and Health, The University of Manchester, Manchester, United Kingdom.

Frontiers in Molecular Biosciences
|September 29, 2022
PubMed

Insights

Mucopolysaccharidoses (MPS) are inherited diseases causing GAG buildup. Gene therapy offers a promising new treatment strategy for these severe pediatric conditions, addressing limitations of current therapies.

Area of Science:

  • Biochemistry
  • Genetics
  • Pediatrics

Background:

  • Mucopolysaccharidoses (MPS) are inherited lysosomal storage diseases in children.
  • Enzyme deficiencies lead to glycosaminoglycan (GAG) accumulation, causing multisystemic symptoms and reduced lifespan.
  • Current treatments like enzyme replacement therapy (ERT) and hematopoietic stem cell transplant (HSCT) have limitations, especially for neurological aspects.

Purpose of the Study:

  • To review emerging gene therapy and gene-editing approaches for MPS.
  • To discuss the potential of these novel strategies in overcoming current treatment challenges.

Main Methods:

  • Review of pre-clinical and clinical research on gene therapy vectors for MPS.
  • Analysis of gene-editing technologies being developed for MPS treatment.

Main Results:

  • Gene therapy is an emerging strategy for MPS treatment.
  • Various vectors and gene-editing approaches are under development.
  • These novel therapies aim to improve efficacy, particularly for neurological symptoms.

Conclusions:

  • Gene therapy and gene editing represent promising therapeutic avenues for MPS.
  • Further research and clinical development are crucial to realize the full potential of these strategies for improving patient outcomes.

Keywords:
clinical trialsgene editinggene therapymucopolysaccharidosisvectors

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