Journal article
A systematic review of adeno-associated virus gene therapies in neurology: the need for consistent safety monitoring of a promising treatment
Journal of neurology, neurosurgery and psychiatry, Vol.93(12), pp.1276-1288
09/07/2022
DOI: 10.1136/jnnp-2022-329431
PMID: 36190933
Abstract
Adeno-associated virus (AAV) gene therapies are generating much excitement in the rare disease field, particularly for previously untreatable neurological conditions. Efficacy has been claimed for several gene therapy products and the number of trials is rapidly increasing. However, reports of severe treatment-related adverse reactions are emerging, including death. There is still insufficient knowledge about their aetiology, prevention and treatment. We therefore undertook to systematically review publicly available data on AAV gene therapies in order to collate existing information on both safety and efficacy. Here, we review emerging efficacy reports of these novel therapies, many of which show promise. We also collate an increasing number of adverse reactions. Overwhelmingly, these results make a case for unified reporting of adverse events. This is likely to be critical for improving the safety of these promising treatments.
Details
- Title: Subtitle
- A systematic review of adeno-associated virus gene therapies in neurology: the need for consistent safety monitoring of a promising treatment
- Creators
- Rebecca H. Horton - University of OxfordDimah Saade - University of IowaTheodora Markati - University of OxfordElinor Harriss - University of OxfordCarsten G. Bonnemann - NINDS, NIH, Bldg 36,Rm 4D04, Bethesda, MD 20892 USAFrancesco Muntoni - Great Ormond Street HospitalLaurent Servais - University of Oxford
- Resource Type
- Journal article
- Publication Details
- Journal of neurology, neurosurgery and psychiatry, Vol.93(12), pp.1276-1288
- DOI
- 10.1136/jnnp-2022-329431
- PMID
- 36190933
- NLM abbreviation
- J Neurol Neurosurg Psychiatry
- ISSN
- 0022-3050
- eISSN
- 1468-330X
- Publisher
- Bmj Publishing Group
- Number of pages
- 13
- Language
- English
- Date published
- 09/07/2022
- Academic Unit
- Stead Family Department of Pediatrics; Iowa Neuroscience Institute; Neurology (Pediatrics)
- Record Identifier
- 9984353936302771
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