Evaluation of neuroretina following i.v. or intra‐<scp>CSF AAV9</scp> gene replacement in mice with <scp>MPS IIIA</scp>, a childhood dementia
Confirmatory studies are needed in adult mice; however, these data indicate that i.v. AAV9-sulfamidase infusion leads to superior outcomes in neuroretina, and cerebrospinal fluid-delivered AAV9 may need to be supplemented with another therapeutic approach for optimal patient quality of life.
Authors
- Andrew P. Shoubridge (ORCID: https://orcid.org/0000-0002-5148-4063)
- Kim M. Hemsley (ORCID: https://orcid.org/0000-0003-1038-9884)
- Paul J. Trim (ORCID: https://orcid.org/0000-0001-8734-3433)
- Adeline A. Lau (ORCID: https://orcid.org/0000-0002-4485-7714)
- Leanne K. Winner (ORCID: https://orcid.org/0000-0001-5175-4839)
- Emma J. Parkinson-Lawrence (ORCID: https://orcid.org/0000-0002-3845-4847)
- Helen Beard (ORCID: https://orcid.org/0000-0003-2042-1438)
- Siti Nurul Mubarokah
- Marten F. Snel (ORCID: https://orcid.org/0000-0002-8502-7274)
- Tabitha‐Rose Lance
- William Scott
- Barbara King (ORCID: https://orcid.org/0000-0002-2715-1787)
Institutions
- Flinders University (AU)
- University of South Australia (AU)
- South Australian Health and Medical Research Institute (AU)
Publication Details
- Journal
- CNS Neuroscience & Therapeutics
- Published
- 2024-08-01
- DOI
- https://doi.org/10.1111/cns.14919
- Primary Topic
- Lysosomal Storage Disorders Research
- Type
- article
- Field-Weighted Citation Impact
- 0.00
Funders
- University of Adelaide
- National Health and Medical Research Council