Shank3 gene therapy approaches
EP4669362A1Pending Publication Date: 2025-12-31MASSACHUSETTS INST OF TECH +1
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Patent Information
- Application Number
- EP2024761117
- Authority / Receiving Office
- EP · EP
- Patent Type
- Applications
- Current Assignee / Owner
- Priority Date
- 2023-02-24
- Filing Date
- 2024-02-23
- Publication Date
- 2025-12-31
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Figure US2024017176_29082024_PF_FP_ABST
Abstract
Aspects of the disclosure relate to expression cassettes encoding miniShank3 proteins, AAV vectors comprising the expression cassettes, and gene therapy methods. In particular, the expression cassettes is formulated to further comprise AAV9 capsids, and the gene therapy is useful for treating neuroveleopmental disorders such as autism spectrum disorder (ASD) and Phelan-McDermid syndrome.
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