RNA-editing gene therapy approaches for treating myotonic dystrophy type 1 (DM1)
By employing CRISPR-associated proteins and guide nucleic acids to target and reduce expanded DMPK RNA transcripts, the treatment of Myotonic Dystrophy Type 1 (DM1) can be effectively addressed, providing a promising therapeutic option for this currently untreatable condition.
Patent Information
- Application Number
- PCT/US2024/048775
- Authority / Receiving Office
- WO · WO
- Patent Type
- Applications
- Current Assignee / Owner
- Priority Date
- 2023-09-28
- Filing Date
- 2024-09-27
- Publication Date
- 2025-04-03
AI Technical Summary
Current treatment options for Myotonic Dystrophy Type 1 (DM1) are limited due to the complexity of its pathogenesis, which involves the expression of toxic gain-of-function microsatellite repeat RNA, and there are no approved therapies to effectively address the disease.
The use of CRISPR-associated (Cas) proteins and guide nucleic acids to target and reduce the levels of expanded DMPK RNA transcripts, specifically through the administration of fusion proteins comprising a CRISPR-associated (Cas) protein and a cleavage or adenylation protein, along with a guide nucleic acid complementary to the target DMPK RNA.
This approach effectively reduces the levels of toxic DMPK RNA transcripts, potentially rescuing DMPK expression and preventing the transcription of downstream toxic CUG repeat RNA, thereby offering a novel therapeutic strategy for treating DM1.