Announcement of Paper Publication: EditForce Demonstrates Efficacy of Treatment for Myotonic Dystrophy Type 1 Using PPR Platform Technology

The results of this study open the way for the development of treatments for the Disease, which currently has no fundamental cure, and are evaluated as demonstrating the innovativeness and efficacy of EditForce's proprietary PPR platform technology. It will continue to strive in its R&D activities to deliver the treatment for the Disease to patients as quickly as possible.

Paper Information

- Journal: Science Translational Medicine

- Publication Date: Wednesday, April 16, 2025, 2:00 PM (Eastern Daylight Time)

- Title: Pentatricopeptide repeat protein targeting CUG repeat RNA ameliorates RNA toxicity in a myotonic dystrophy type 1 mouse model

- Authors: Takayoshi Imai1*, Maiko Miyai2*, Joe Nemoto3, Takayuki Tamai1, Masaru Ohta1, Yusuke Yagi1, Osamu Nakanishi1, Hideki Mochizuki2, and Masayuki Nakamori2,3**

Affiliations:

1. EditForce, Inc.

2. Graduate School of Medicine, University of Osaka, Department of Neurology

3. Graduate School of Medicine, Yamaguchi University, Clinical Neurology

*: These authors contributed equally to this work.

**: Corresponding author

DOI: 10.1126/scitranslmed.adq2005
https://www.science.org/doi/10.1126/scitranslmed.adq2005 

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