Gene therapy restores dopamine transporter expression and ameliorates pathology in iPSC and mouse models of infantile parkinsonism

Author:

Ng Joanne12ORCID,Barral Serena2ORCID,De La Fuente Barrigon Carmen3,Lignani Gabriele4,Erdem Fatma A.25ORCID,Wallings Rebecca6ORCID,Privolizzi Riccardo12ORCID,Rossignoli Giada2,Alrashidi Haya3ORCID,Heasman Sonja2ORCID,Meyer Esther2,Ngoh Adeline2ORCID,Pope Simon7ORCID,Karda Rajvinder1ORCID,Perocheau Dany1ORCID,Baruteau Julien13ORCID,Suff Natalie18ORCID,Antinao Diaz Juan1ORCID,Schorge Stephanie49ORCID,Vowles Jane10ORCID,Marshall Lucy R.11,Cowley Sally A.10ORCID,Sucic Sonja5ORCID,Freissmuth Michael5,Counsell John R.12ORCID,Wade-Martins Richard6ORCID,Heales Simon J. R.37,Rahim Ahad A.9ORCID,Bencze Maximilien1213ORCID,Waddington Simon N.114ORCID,Kurian Manju A.215

Affiliation:

1. Gene Transfer Technology Group, EGA-Institute for Women’s Health, University College London, London, WC1E 6HX, UK.

2. Developmental Neurosciences, Zayed Centre for Research into Rare Disease in Children, GOS-Institute of Child Health, University College London, London, WC1N 1DZ, UK.

3. Genetics and Genomic Medicine, GOS-Institute of Child Health, University College London, London, WC1N 1EH, UK.

4. Clinical and Experimental Epilepsy, Queen Square Institute of Neurology, University College London, London, WC1N 3BG, UK.

5. Institute of Pharmacology and Gaston H. Glock Laboratories for Exploratory Drug Research, Centre of Physiology and Pharmacology, Medical University of Vienna, 1090 Vienna, Austria.

6. Oxford Parkinson’s Disease Centre, Department of Physiology, Anatomy and Genetics, University of Oxford, Oxford, OX1 3PT, UK.

7. Neurometabolic Unit, National Hospital for Neurology and Neurosurgery, Queen Square, London, WC1N 3BG, UK.

8. Department of Women and Children’s Health, King’s College London, London, WC2R 2LS, UK.

9. Pharmacology, School of Pharmacy, University College London, London, WC1N 1AX, UK.

10. James Martin Stem Cell Facility, Sir William Dunn School of Pathology, University of Oxford, Oxford, OX1 3RE, UK.

11. Infection, Immunity, Inflammation, GOS-Institute of Child Health, University College London, London, WC1N 1EH, UK.

12. Developmental Neurosciences, GOS-Institute of Child Health, University College London, London, WC1N 1EH, UK.

13. University Paris Est Creteil, INSERM, IMRB, 94000 Creteil, France.

14. Wits/SAMRC Antiviral Gene Therapy Research Unit, Faculty of Health Sciences, University of the Witwatersrand, 2193 Johannesburg, South Africa.

15. Department of Neurology, Great Ormond Street Hospital for Children, London, WC1N 3JH, UK.

Abstract

Viral vectors restore dopamine transporter function and ameliorate neuropathology in iPSC-derived neurons and a mouse model of infantile parkinsonism.

Funder

Wellcome Trust

University of Kentucky

Wellcome

Horizon 2020 Framework Programme

MicroResearch

Texas Academy of Science

Medical Research Council

LifeArc

National Institute for Health Research

Great Ormond Street Hospital for Children

Department of Health, Australian Government

Epilepsy Research UK

Great Ormond Street Hospital Charity

Royal Society

Rosetrees Trust

Austrian Science Fund

Wellbeing of Women

Parkinson’s UK

Action Medical Research

Sparks

National Centre for the Replacement Refinement and Reduction of Animals in Research

Mauritius Research Council

UCLH Biomedical Research Centre

NIHR Great Ormond Street Hospital Biomedical Research Centre

Brunei Research Council

Marie Curie Fellowship

NIHR

Versus Arthritis

CONICYT Becas Chile Doctoral Fellowship Program

Association Française contre les Myopathies

NC3Rs

European Union’s Horizon 2020

Marie Skłodowska-Curie Actions European Union’s Seventh Framework Programme FP7

Publisher

American Association for the Advancement of Science (AAAS)

Subject

General Medicine

Reference52 articles.

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