Leanne K. Winner
Bedford Park, SA, Australia
Last Updated: 09/26/2026
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Found 10 publications
Evaluation of neuroretina following i.v. or intra-CSF AAV9 gene replacement in mice with MPS IIIA, a childhood dementia.
Journal: CNS neuroscience & therapeutics
Published: January 15, 2024
Repetitive, non-invasive imaging of neurodegeneration, and prevention of it with gene replacement, in mice with Sanfilippo syndrome.
Journal: Experimental neurology
Published: May 24, 2023
Biomarkers for predicting disease course in Sanfilippo syndrome: An urgent unmet need in childhood-onset dementia.
Journal: Journal of neurochemistry
Published: March 03, 2023
The ovine Type II Gaucher disease model recapitulates aspects of human brain disease.
Journal: Biochimica et biophysica acta. Molecular basis of disease
Published: July 18, 2022
AAVrh10 Vector Corrects Disease Pathology in MPS IIIA Mice and Achieves Widespread Distribution of SGSH in Large Animal Brains.
Journal: Molecular therapy. Methods & clinical development
Published: August 13, 2019
Evaluation of Disease Lesions in the Developing Canine MPS IIIA Brain.
Journal: JIMD reports
Published: May 02, 2017
A Preclinical Study Evaluating AAVrh10-Based Gene Therapy for Sanfilippo Syndrome.
Journal: Human gene therapy
Published: March 16, 2016
Intracisternal enzyme replacement therapy in lysosomal storage diseases: dispersal pathways, regional enzyme concentrations and the effect of posttreatment posture.
Journal: Neuropathology and applied neurobiology
Published: July 08, 2012
Neonatal Bone Marrow Transplantation in MPS IIIA Mice.
Journal: JIMD reports
Published: April 13, 2012
Showing 1-10 of 10
Last Updated: 09/26/2026