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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