Preclinical use of a clinically-relevant scAAV9/SUMF1 vector for the treatment of multiple sulfatase deficiency
These preclinical studies highlight the potential of our AAV9/SUMF1 vector, the design of which is directly translatable for clinical use, as a gene replacement therapy for MSD patients.
Authors
- Harold F. Combs
- Steven J. Gray (ORCID: https://orcid.org/0000-0002-6240-8621)
- Pierre-Alexandre Piec (ORCID: https://orcid.org/0009-0009-6315-7279)
- Lauren Bailey (ORCID: https://orcid.org/0009-0000-4065-4541)
- Maximiliano Presa (ORCID: https://orcid.org/0000-0002-0160-6528)
- Saurabh Tata
- Cathleen M. Lutz (ORCID: https://orcid.org/0000-0003-2502-1900)
- Tara Murphy
- Somdatta Ray
- Rachel M. Bailey (ORCID: https://orcid.org/0000-0002-5909-5548)
Institutions
- Southwestern Medical Center (US)
- Jackson Laboratory (US)
- The University of Texas Southwestern Medical Center (US)
Publication Details
- Journal
- Communications Medicine
- Published
- 2025-01-27
- DOI
- https://doi.org/10.1038/s43856-025-00734-9
- Primary Topic
- Lysosomal Storage Disorders Research
- Type
- article
- Field-Weighted Citation Impact
- 0.00
Funders
- National Institutes of Health