MATERIALS AND METHODS FOR THE TREATMENT OF DUCHENNE MUSCULAR DYSTROPHY
DE602016092572T2Active Publication Date: 2025-06-11VERTEX PHARMACEUTICALS INC
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Patent Information
- Application Number
- DE602016092572
- Authority / Receiving Office
- DE · DE
- Patent Type
- Patents
- Current Assignee / Owner
- Filing Date
- 2016-10-28
- Publication Date
- 2025-06-11
- Estimated Expiration
- 2036-10-28
Abstract
The present application provides materials and methods for treating a patient with Duchenne Muscular Dystrophy (DMD) both ex vivo and in vivo. In addition, the present application provides materials and methods for editing a dystrophin gene in a cell by genome editing.
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