Adeno-associated virus vectors for treatment of rett syndrome
NZ836045APending Publication Date: 2026-08-28SAREPTA THERAPEUTICS INC
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Patent Information
- Application Number
- NZ836045
- Authority / Receiving Office
- NZ · NZ
- Patent Type
- Applications
- Current Assignee / Owner
- Priority Date
- 2020-08-19
- Filing Date
- 2021-08-19
- Publication Date
- 2026-08-28
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Figure 1_ABST
Abstract
The disclosure provides nucleic acids (comprising AAV expression cassettes), AAV vectors, and compositions for use in methods for treating and / or delaying the onset of diseases associated with mutations in the mecp2 gene, such as Rett Syndrome. Also, provided herein are methods for treating and / or delaying the onset of brain-derived neurotrophic factor (BDNF)-associated diseases.
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