A novel adeno-associated virus capsid with enhanced neurotropism corrects a lysosomal transmembrane enzyme deficiency

Author:

Tordo Julie1,O’Leary Claire2,Antunes André S L M1,Palomar Nuria1,Aldrin-Kirk Patrick3,Basche Mark4,Bennett Antonette5,D’Souza Zelpha2,Gleitz Hélène2,Godwin Annie2,Holley Rebecca J2,Parker Helen2,Liao Ai Yin2,Rouse Paul2,Youshani Amir Saam2,Dridi Larbi6,Martins Carla6,Levade Thierry7,Stacey Kevin B8,Davis Daniel M8,Dyer Adam1,Clément Nathalie9,Björklund Tomas3,Ali Robin R4,Agbandje-McKenna Mavis5,Rahim Ahad A10,Pshezhetsky Alexey6,Waddington Simon N1112,Linden R Michael1,Bigger Brian W2,Henckaerts Els1

Affiliation:

1. Department of Infectious Diseases, School of Immunology and Microbial Sciences, King’s College London, London, UK

2. Stem Cell and Neurotherapies, Division of Cell Matrix Biology and Regenerative Medicine, School of Biological Sciences, Faculty of Biology Medicine and Health, University of Manchester, Manchester, UK

3. Molecular Neuromodulation, Wallenberg Neuroscience Center, Lund University, Lund, Sweden

4. Department of Genetics, UCL Institute of Ophthalmology, London, UK

5. Department of Biochemistry and Molecular Biology, Center for Structural Biology, McKnight Brain Institute, College of Medicine, University of Florida, Gainesville, FL, USA

6. CHU Ste-Justine, University of Montreal, Montreal, Canada

7. Centre Hospitalo-Universitaire de Toulouse, Institut Fédératif de Biologie, Laboratoire de Biochimie Métabolique, and Unité Mixte de Recherche (UMR) 1037 Institut National de la Santé et de la Recherche Médicale (INSERM), Centre de Recherche en Cancérologie de Toulouse, Toulouse, France

8. Manchester Collaborative Centre for Inflammation Research, Division of Infection, Immunity and Respiratory Medicine, School of Biological Sciences, Faculty of Biology Medicine and Health, University of Manchester, Manchester, UK

9. Department of Pediatrics, Powell Gene Therapy Center, University of Florida, Gainesville, FL, USA

10. Department of Pharmacology, UCL School of Pharmacy, University College London, London, UK

11. Gene Transfer Technology Group, Institute for Women’s Health, University College London, London, UK

12. Wits/SAMRC Antiviral Gene Therapy Research Unit, Faculty of Health Sciences, University of the Witwatersrand, Johannesburg, South Africa

Funder

Action Medical Research

Swedish Research Council

Publisher

Oxford University Press (OUP)

Subject

Clinical Neurology

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