Brain-Directed AAV Gene Therapy Rescues a Mouse Model of the CLN5 Form of Neuronal Ceroid Lipofuscinosis Disease and Normalizes a Blood Plasma Biomarker of Neurodegeneration
Year: 2025
Type: article
Language: en
Topic: Lysosomal Storage Disorders Research
Authors and affiliations:
raw_author_name: Wenfei Liu
raw_affiliation_strings: Department of Pharmacology, UCL School of Pharmacy, University College London, London, United Kingdom
raw_author_name: Amy F. Geard
raw_affiliation_strings: Department of Pharmacology, UCL School of Pharmacy, University College London, London, United Kingdom
raw_author_name: Giulia Massaro
raw_affiliation_strings: Department of Pharmacology, UCL School of Pharmacy, University College London, London, United Kingdom
raw_author_name: Michael P. Hughes
raw_affiliation_strings: Department of Pharmacology, UCL School of Pharmacy, University College London, London, United Kingdom
raw_author_name: Mikel Aristorena
raw_affiliation_strings: Centre for Gene Therapy and Regenerative Medicine, King’s College London, Guy’s Hospital, London, United Kingdom
UCL Great Ormond Street Institute of Child Health, University College London, London, United Kingdom
raw_author_name: Oliver Coombe-Tennant
raw_affiliation_strings: Department of Pharmacology, UCL School of Pharmacy, University College London, London, United Kingdom
raw_author_name: Liangyuan Xu
raw_affiliation_strings: Department of Pharmacology, UCL School of Pharmacy, University College London, London, United Kingdom
raw_author_name: Olha Semenyuk
raw_affiliation_strings: Centre for Gene Therapy and Regenerative Medicine, King’s College London, Guy’s Hospital, London, United Kingdom
raw_author_name: Reuben Bush
raw_affiliation_strings: Department of Pharmacology, University of Oxford, Oxford, United Kingdom
raw_author_name: Danielle te Vruchte
raw_affiliation_strings: Department of Pharmacology, University of Oxford, Oxford, United Kingdom
raw_author_name: David Priestman
raw_affiliation_strings: Department of Pharmacology, University of Oxford, Oxford, United Kingdom
raw_author_name: Rhiannon Laban
raw_affiliation_strings: Fluid Biomarker Laboratory and Biomarker Factory, UK Dementia Research Institute at UCL, London, United Kingdom
raw_author_name: Elena Veleva
raw_affiliation_strings: Fluid Biomarker Laboratory and Biomarker Factory, UK Dementia Research Institute at UCL, London, United Kingdom
raw_author_name: Amanda J. Heslegrave
raw_affiliation_strings: Fluid Biomarker Laboratory and Biomarker Factory, UK Dementia Research Institute at UCL, London, United Kingdom
raw_author_name: Henrik Zetterberg
raw_affiliation_strings: Fluid Biomarker Laboratory and Biomarker Factory, UK Dementia Research Institute at UCL, London, United Kingdom
raw_author_name: Frances M. Platt
raw_affiliation_strings: Department of Pharmacology, University of Oxford, Oxford, United Kingdom
raw_author_name: Alexander J. Smith
raw_affiliation_strings: Centre for Gene Therapy and Regenerative Medicine, King’s College London, Guy’s Hospital, London, United Kingdom
raw_author_name: Sara E. Mole
raw_affiliation_strings: UCL Great Ormond Street Institute of Child Health, University College London, London, United Kingdom
raw_author_name: Robin R. Ali
raw_affiliation_strings: Centre for Gene Therapy and Regenerative Medicine, King’s College London, Guy’s Hospital, London, United Kingdom
raw_author_name: Ahad A. Rahim
raw_affiliation_strings: Department of Pharmacology, UCL School of Pharmacy, University College London, London, United Kingdom