论文发表公告：EditForce展示使用PPR平台技术治疗1型肌强直性营养不良的疗效-动脉网

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

CISION 等信源发布 2025-04-25 14:00

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FUKUOKA, Japan

福冈，日本

，

April 25, 2025

2025年4月25日

/PRNewswire/ -- EditForce, Inc. (Headquarters:

/PRNewswire/ -- EditForce公司（总部：

Fukuoka, Japan

日本福冈

) is pleased to announce that a research paper on the results of a joint study with the research group led by Professor

）高兴地宣布，与教授领导的研究小组联合研究的结果发表了一篇研究论文，

Masayuki Nakamori

中森正之

of the Department of Neurology of the Yamaguchi University Graduate School of Medicine, and Professor

山口大学医学研究生院神经内科系，教授

Hideki Mochizuki

望月秀树

of the Department of Neurology of the University of Osaka Graduate School of Medicine, was published in Science Translational Medicine on April 16,

大阪大学医学研究生院神经内科系于4月16日发表在《科学转化医学》杂志上，

2025 (Eastern Daylight Time

2025年（北美东部夏令时间）

）。

In this study, it was demonstrated that a single administration of the jointly developed PPR protein (CUG-PPR1), which specifically binds to the abnormal RNA causing Myotonic Dystrophy Type 1 (hereinafter referred to as 'the Disease'), resulted in long-term amelioration of muscle symptoms in mice, with minimal immune response and side effects..

本研究证实，单独使用联合开发的PPR蛋白（CUG-PPR1）（其特异性结合导致Ⅰ型肌强直性营养不良（以下简称“该疾病”）的异常RNA），可长期改善小鼠的肌肉症状，且免疫反应和副作用极小。

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..

本研究的结果为开发目前尚无根治方法的疾病的治疗手段开辟了道路，并且被评估为展示了EditForce独有的PPR平台技术的创新性和有效性。公司将继续全力以赴进行研发活动，以尽快将该疾病的治疗方法带给患者。

Paper Information

论文信息

- Journal: Science Translational Medicine

期刊：《科学转化医学》

- Publication Date:

- 出版日期：

Wednesday, April 16, 2025

2025年4月16日，星期三

，

2:00 PM (Eastern Daylight Time)

下午2:00（东部夏令时间）

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

- 标题：靶向CUG重复RNA的五肽重复蛋白在1型肌强直性营养不良小鼠模型中缓解RNA毒性

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

作者：今井孝佳1*，三宅舞子2*，根本丈雄3，田邉孝之1，太田贤治1，八木佑介1，中西修1，持木秀树2，中森雅之2,3**

Affiliations:

关联：

1. EditForce, Inc.

1. EditForce公司

2. Graduate School of Medicine, University of

2. 医学研究生院，大学

Osaka

大阪

, Department of Neurology

神经内科部

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