Mapping brain networks in MPS I mice and their restoration following gene therapy

Wei Zhu, Li Ou, Lin Zhang, Isaac H. Clark, Ying Zhang, Xiao Hong Zhu, Chester B. Whitley, Perry B. Hackett, Walter C. Low, Wei Chen

Research output: Contribution to journalArticlepeer-review


Mucopolysaccharidosis type I (MPS I) is an inherited lysosomal disorder that causes syndromes characterized by physiological dysfunction in many organs and tissues. Despite the recognizable morphological and behavioral deficits associated with MPS I, neither the underlying alterations in functional neural connectivity nor its restoration following gene therapy have been shown. By employing high-resolution resting-state fMRI (rs-fMRI), we found significant reductions in functional neural connectivity in the limbic areas of the brain that play key roles in learning and memory in MPS I mice, and that adeno-associated virus (AAV)-mediated gene therapy can reestablish most brain connectivity. Using logistic regression in MPS I and treated animals, we identified functional networks with the most alterations. The rs-fMRI and statistical methods should be translatable into clinical evaluation of humans with neurological disorders.

Original languageEnglish (US)
Article number12716
JournalScientific reports
Issue number1
StatePublished - Dec 2023

Bibliographical note

Publisher Copyright:
© 2023, Springer Nature Limited.

PubMed: MeSH publication types

  • Journal Article
  • Research Support, Non-U.S. Gov't
  • Research Support, N.I.H., Extramural


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