Gene Therapy for Lysosomal Storage Disorders: Ongoing Studies and Clinical Development
Giulia Massaro,
Amy F. Geard,
Wenfei Liu,
Oliver Coombe-Tennant,
Simon N. Waddington,
Julien Baruteau,
Paul Gissen,
Ahad A. Rahim
Affiliations
Giulia Massaro
UCL School of Pharmacy, University College London, London WC1N 1AX, UK
Amy F. Geard
UCL School of Pharmacy, University College London, London WC1N 1AX, UK
Wenfei Liu
UCL School of Pharmacy, University College London, London WC1N 1AX, UK
Oliver Coombe-Tennant
UCL School of Pharmacy, University College London, London WC1N 1AX, UK
Simon N. Waddington
Wits/SAMRC Antiviral Gene Therapy Research Unit, Faculty of Health Sciences, University of the Witwatersrand, Johannesburg 2193, South Africa
Julien Baruteau
Metabolic Medicine Department, Great Ormond Street Hospital for Children NHS Foundation Trust, London WC1N 1EH, UK
Paul Gissen
Great Ormond Street Hospital Biomedical Research Centre, Great Ormond Street Institute of Child Health, National Institute of Health Research, University College London, London WC1N 1EH, UK
Ahad A. Rahim
UCL School of Pharmacy, University College London, London WC1N 1AX, UK
Rare monogenic disorders such as lysosomal diseases have been at the forefront in the development of novel treatments where therapeutic options are either limited or unavailable. The increasing number of successful pre-clinical and clinical studies in the last decade demonstrates that gene therapy represents a feasible option to address the unmet medical need of these patients. This article provides a comprehensive overview of the current state of the field, reviewing the most used viral gene delivery vectors in the context of lysosomal storage disorders, a selection of relevant pre-clinical studies and ongoing clinical trials within recent years.
