Gene therapy for Rett syndrome: prospects and challenges

Gadalla, K. K. E. , Ross, P. D., Hector, R. D., Bahey, N. G., Bailey, M. E.S. and Cobb, S. R. (2015) Gene therapy for Rett syndrome: prospects and challenges. Future Neurology, 10(5), pp. 467-484. (doi:10.2217/fnl.15.29)

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Abstract

Rett syndrome (RTT) is a neurological disorder that affects females and is caused by loss-of-function mutations in the X-linked gene MECP2. Deletion of Mecp2 in mice results in a constellation of neurological features that resemble those seen in RTT patients. Experiments in mice have demonstrated that restoration of MeCP2, even at adult stages, reverses several aspects of the RTT-like pathology suggesting that the disorder may be inherently treatable. This has provided an impetus to explore several therapeutic approaches targeting RTT at the level of the gene, including gene therapy, activation of MECP2 on the inactive X chromosome and read-through and repair of RTT-causing mutations. Here, we review these different strategies and the challenges of gene-based approaches in RTT.

Item Type:Articles
Status:Published
Refereed:Yes
Glasgow Author(s) Enlighten ID:Gadalla, Dr Kamal and Cobb, Dr Stuart and Hector, Dr Ralph and Bailey, Dr Mark and Bahey, Mrs Noha
Authors: Gadalla, K. K. E., Ross, P. D., Hector, R. D., Bahey, N. G., Bailey, M. E.S., and Cobb, S. R.
College/School:College of Medical Veterinary and Life Sciences
College of Medical Veterinary and Life Sciences > Institute of Neuroscience and Psychology
Journal Name:Future Neurology
Publisher:Future Medicine
ISSN:1479-6708
ISSN (Online):1748-6971

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Project CodeAward NoProject NamePrincipal InvestigatorFunder's NameFunder RefLead Dept
644651Developing and evaluating novel gene therapy approaches in Rett syndromeStuart CobbScottish Executive Health Department (SEHHD-CSO)ETM/334INP - CENTRE FOR NEUROSCIENCE
302221Grant for Group Status in Medical HistoryM CrowtherWellcome Trust (WELLCOME)065751/Z/01/Z/JCOLLEGE OF SOCIAL SCIENCES ADMIN