Effector proteins, compositions, systems and methods of use thereof for the treatment of dmpk-associated diseases and syndromes
EP4747383A1Pending Publication Date: 2026-05-27MAMMOTH BIOSCIENCES INC
Patent Information
- Authority / Receiving Office
- EP · EP
- Patent Type
- Applications
- Current Assignee / Owner
- MAMMOTH BIOSCIENCES INC
- Filing Date
- 2024-07-17
- Publication Date
- 2026-05-27
AI Technical Summary
Technical Problem
Current technologies are inadequate for effectively detecting and editing target nucleic acids associated with the DMPK gene, which are linked to various diseases and syndromes.
Method used
Compositions and systems comprising an effector protein and a guide nucleic acid are developed for the modification and detection of target nucleic acids of the DMPK gene, utilizing nucleic acid modifying activities for therapeutic applications.
Benefits of technology
These compositions and systems enable precise modification and detection of DMPK gene-associated nucleic acids, offering potential therapeutic benefits for associated diseases and syndromes.
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Abstract
Provided herein are compositions, systems, and methods comprising variants of effector proteins, and uses thereof. These effector proteins may be characterized as CRISPR-associated (Cas) proteins. Various compositions, systems, and methods of the present disclosure may leverage the activities of these effector proteins for the editing, detecting and / or engineering of nucleic acids.
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