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Elizabeth Berry-Kravis |
Treatment of CLN1 with TSHA-118 for AAV-9-Mediated PPT1 Gene Transfer Through an Expanded Access Program |
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Angela Schulz |
LTS-101: An Intracerebroventricular Delivered AAV Gene Therapy Using a Novel Capsid Variant for the Treatment of CLN2 Batten Disease |
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Ewa A Ziółkowska |
Dual delivery of gene therapy via intraventricular and intravenous routes in CLN2 disease |
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Evan Waldo Kleinboehl |
Engineered cell micropharmacies for in vivo production of TPP1 as enzyme replacement therapy in CLN2 Batten disease |
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Jonathan D Cooper |
Improving the efficacy of enzyme replacement and gene therapy approaches for CLN2 disease via S1S3 phosphotransferase mediated hyperphosphorylation |
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Miriam MA Aurilia |
ESRRA: a new druggable target to tackle Batten disease |
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Jill M Weimer |
Adenine Base Editors: A Novel Approach to Treat CLN2 Disease |
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Ewa A Ziółkowska |
Investigating the pathophysiological basis of dysphagia in CLN1, CLN2, and CLN3 mouse models and the therapeutic effects of gene therapy |
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Llinos Honeybun |
From cell phenotypes to candidate therapies: high-content screening in CLN3 disease |
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Magnar Bjørås |
CLN3 patient-derived organoids as preclinical platforms for drug screening and gene therapy testing |
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Clarissa D Booth |
Deep, Untargeted Multi-omics Biomarker Landscape in Large Animal Models of CLN1, -2, -3, -5,and -6 Batten Disease |
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Joelle T Anderson |
CLN2 Batten Disease biomarker profiling reveals cardiovascular-associated metabolic dysfunction |
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Lottie D Morison |
Speech, Language and Non-verbal Communication in CLN2 and CLN3 Batten Disease |
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Wendy E Heywood |
Urinary Glycerophosphoinositol is a potentially non-invasive clinically applicable biomarker for CLN3 disease |
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Miriam Nickel |
Assessment of cognitive decline in CLN3 using the Vineland Adaptive Behavior Scale and the CLN3 Disease Staging System |
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An N Dang Do |
MRI-derived parenchymal volume as a robust biomarker for CLN3 disease progression at the individual level: a longitudinal assessment |
Be ready for bus departure at 17:40
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