Compositions and methods for treating neurological disorders
JP2026508803APending Publication Date: 2026-03-13KINGS COLLEGE LONDON
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Patent Information
- Authority / Receiving Office
- JP · JP
- Patent Type
- Applications
- Current Assignee / Owner
- Filing Date
- 2023-12-14
- Publication Date
- 2026-03-13
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Figure 2026508803000008 
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Abstract
This disclosure features compositions and methods for treating disorders associated with the expression of wild-type or mutant superoxide dismutase 1 (SOD1) RNA transcripts that may result in pathological phenotypes. Inhibitory RNA constructs that suppress SOD1 expression, as well as viral vectors such as adeno-associated viral vectors encoding such inhibitory RNA molecules, are disclosed herein.
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