Single-cut gene therapy in a one-step generated rhesus monkey model of Duchenne muscular dystrophy

Progress in Duchenne muscular dystrophy (DMD) treatment is hindered by the lack of animal models that closely replicate human pathology and enable the evaluation of therapy efficacy and safety based on these models. To address this need, we optimize the generation of nonhuman primate DMD models, reducing the development time from 6 to 7 years to under 1 year, enabling the rapid generation of DMD monkey models. These models closely mimic human DMD pathology and motor dysfunction, making them suitable for testing gene therapies. Using these models, we develop a single-cut gene therapy strategy that can be directly applied to humans. This treatment restores dystrophin expression, improves pathological features, and enhances motor abilities in DMD monkeys, with effects lasting at least 1.5 years. In conclusion, we achieve the rapid generation of DMD monkey models and demonstrate that our gene therapy approach is effective and holds significant potential for clinical application.

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Publication Details

Journal
Cell Reports Medicine
Published
2025-03-27
DOI
https://doi.org/10.1016/j.xcrm.2025.102037
Citations
1
Primary Topic
Muscle Physiology and Disorders
Type
article
Field-Weighted Citation Impact
0.81
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article

Single-cut gene therapy in a one-step generated rhesus monkey model of Duchenne muscular dystrophy

Yue Ma, Yongchang Chen, Wenjie Sun, Junyu Zhang et al.
1 citations
Cell Reports Medicine
Muscle Physiology and Disorders
0.81
article

Single-cut gene therapy in a one-step generated rhesus monkey model of Duchenne muscular dystrophy

Yue Ma, Yongchang Chen, Wenjie Sun, Junyu Zhang, Puhao Xiao, Weizhi Ji, Raoxian Bai, Jie Liu, Yang Jiao, Chaoran Zhou, Shangang Li, Shuaiwei Ren, Siyu Liu, Ting Zhang, Shu Zhang, Wenting Guo, Shiwen Wu, Hong Wang
article en
1 citations

Abstract

Progress in Duchenne muscular dystrophy (DMD) treatment is hindered by the lack of animal models that closely replicate human pathology and enable the evaluation of therapy efficacy and safety based on these models. To address this need, we optimize the generation of nonhuman primate DMD models, reducing the development time from 6 to 7 years to under 1 year, enabling the rapid generation of DMD monkey models. These models closely mimic human DMD pathology and motor dysfunction, making them suitable for testing gene therapies. Using these models, we develop a single-cut gene therapy strategy that can be directly applied to humans. This treatment restores dystrophin expression, improves pathological features, and enhances motor abilities in DMD monkeys, with effects lasting at least 1.5 years. In conclusion, we achieve the rapid generation of DMD monkey models and demonstrate that our gene therapy approach is effective and holds significant potential for clinical application.

Cell Reports MedicineVol. 6(4)
Kunming University of Science and Technology (CN), Southwest Forestry University (CN), Chinese PLA General Hospital (CN), Yunnan Yingmao Biotechnology (China) (CN)
Zero hunger
Openalex Percentile: Top 30%
Muscle Physiology and Disorders
0.81
DOIOpen Access PDFOpenAlex
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