Bone marrow transplantation increases sulfatase activity in somatic tissues in a multiple sulfatase deficiency mouse model
Our results indicate that HSCT could be a suitable approach to treat MSD-pathology affecting peripheral organs, however that benefit to CNS pathology might be limited.
Authors
- Pierre-Alexandre Piec (ORCID: https://orcid.org/0009-0009-6315-7279)
- Maximiliano Presa (ORCID: https://orcid.org/0000-0002-0160-6528)
- Troy C. Lund (ORCID: https://orcid.org/0000-0001-9174-8919)
- Cathleen M. Lutz (ORCID: https://orcid.org/0000-0003-2502-1900)
- Vi Pham (ORCID: https://orcid.org/0000-0002-4647-2885)
- Somdatta Ray
- Jennifer D. Ryan (ORCID: https://orcid.org/0000-0001-9244-7851)
- Harold Coombs
- Lars Schlotawa (ORCID: https://orcid.org/0000-0002-7415-4905)
- Rebecca C. Ahrens‐Nicklas (ORCID: https://orcid.org/0000-0001-8243-7123)
- Timothy Billings
Institutions
- University of Minnesota (US)
- Children's Hospital of Philadelphia (US)
- Fraunhofer Institute for Translational Medicine and Pharmacology (DE)
- Jackson Laboratory (US)
- University of Pennsylvania (US)
Publication Details
- Journal
- Communications Medicine
- Published
- 2024-10-24
- DOI
- https://doi.org/10.1038/s43856-024-00648-y
- Primary Topic
- Lysosomal Storage Disorders Research
- Type
- article
- Field-Weighted Citation Impact
- 0.00
Funders
- National Institutes of Health