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Journal article : Review

A systematic review of adeno-associated virus gene therapies in neurology: the need for consistent safety monitoring of a promising treatment

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 ...

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Publication status:
Published
Peer review status:
Peer reviewed

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Publisher copy:
10.1136/jnnp-2022-329431

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Role:
Author
ORCID:
0000-0002-3712-3026
Publisher:
BMJ Publishing Group
Journal:
Journal of Neurology, Neurosurgery, and Psychiatry More from this journal
Volume:
93
Issue:
12
Pages:
1276-1288
Place of publication:
England
Publication date:
2022-09-08
Acceptance date:
2022-06-20
DOI:
EISSN:
1468-330X
ISSN:
0022-3050
Pmid:
36190933
Language:
English
Keywords:
Subtype:
Review
Pubs id:
1280170
Local pid:
pubs:1280170
Deposit date:
2023-02-06

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