Methods And Compositions For The Treatment Of Monogenic Diseases
Patent No. EP3196301 (titled "Methods And Compositions For The Treatment Of Monogenic Diseases") was filed by Sangamo Biosciences on Mar 15, 2013. The application was issued on Oct 17, 2018.
Patent Summary
A method to treat lysosomal storage diseases like Gaucher's disease by genetically modifying cells to produce the missing enzyme. The method involves inserting a corrective gene encoding the enzyme into cells using engineered nucleases like TALENs or CRISPR/Cas systems. The inserted gene is integrated into the genome and expressed, allowing the modified cells to produce the enzyme and treat the disease. The nucleases are designed to specifically target safe harbor genes like albumin to avoid disruption of essential genes. This enables production of therapeutic proteins at therapeutically relevant levels when the modified cells are administered to patients.
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Date
Description
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EP3196301
- Application Number
- EP16198961A
- Filing Date
- Mar 15, 2013
- Status
- Revoked
- Jul 3, 2020
- Publication Date
- Oct 17, 2018
- External Links
- Slate, Register , Google Patents