Magnetic Resonance Imaging as a Readout of CLN5 Gene Therapy Efficacy in Sheep
This study has demonstrated that MRI, a clinically relevant outcome measure, can be successfully utilized to assess therapeutic efficacy in a large animal model of CLN5 NCL, both in a longitudinal study and a cross-sectional study when robust natural history data is available for comparison.
Authors
- Tracy R. Melzer (ORCID: https://orcid.org/0000-0003-0621-212X)
- Daniel J. Myall (ORCID: https://orcid.org/0000-0002-0032-6567)
- Nadia L. Mitchell (ORCID: https://orcid.org/0000-0003-4801-2804)
- Arsène Ella
- David N. Palmer (ORCID: https://orcid.org/0000-0002-9703-5351)
- Reza Shoorangiz (ORCID: https://orcid.org/0000-0002-3653-9644)
- Ashley R. Deane (ORCID: https://orcid.org/0000-0003-4030-0123)
- Matthieu Keller (ORCID: https://orcid.org/0000-0002-5445-7431)
- Simon A. Felton
- Mustafa M. Almuqbel (ORCID: https://orcid.org/0000-0003-4878-6973)
- Nickolas J. Palmer
- Samantha J. Murray (ORCID: https://orcid.org/0000-0002-4847-2205)
Institutions
- Université de Tours (FR)
- Centre National de la Recherche Scientifique (FR)
- Lincoln University (NZ)
- University of Canterbury (NZ)
- Institut National de Recherche pour l'Agriculture, l'Alimentation et l'Environnement (FR)
- Pacific Radiology (New Zealand) (NZ)
- New Zealand Brain Research Institute (NZ)
- Physiologie de la Reproduction et des Comportements (FR)
- University of Otago (NZ)
Publication Details
- Journal
- Brain and Behavior
- Published
- 2025-04-01
- DOI
- https://doi.org/10.1002/brb3.70431
- Citations
- 1
- Primary Topic
- Lysosomal Storage Disorders Research
- Type
- article
- Field-Weighted Citation Impact
- 1.16
Funders
- Batten Disease Support and Research Association
- University of Otago
- Canterbury Medical Research Foundation
- Neurological Foundation of New Zealand
- Batten Disease Support and Research Association Australia