Mini-gde for the treatment of glycogen storage disease iii
Patent Information
- Application Number
- IL280570
- Authority / Receiving Office
- IL · IL
- Patent Type
- Applications
- Current Assignee / Owner
- Priority Date
- 2018-08-08
- Filing Date
- 2019-08-06
- Publication Date
- 2026-08-01
AI Technical Summary
Current gene therapy approaches for glycogen storage disease III (GSDIII) are hindered by the large size of the GDE transgene, which exceeds the size limit of most gene therapy vectors, necessitating the development of novel strategies for effective treatment.
The use of functional truncated human GDE polypeptides, specifically deleted versions of the GDE protein, that are small enough to be packaged into a single AAV vector, preserving enzymatic activity and enabling efficient glycogen degradation and muscle strength rescue.
These truncated GDE polypeptides, such as those comprising specific amino acid deletions, can be efficiently delivered via AAV vectors, effectively rescuing glycogen accumulation and muscle function in GSDIII models, offering a promising therapeutic option.
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