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Molecular Therapy
Published by Elsevier.
ISSN 1525-0016 / 1525-0024
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Aims & scope
Molecular Therapy publishes peer-reviewed research. Manuscripts pass a technical check and a scope check by the handling editor before going to independent reviewers.
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News from biotech
DOI 10.1016/j.ymthe.2026.10.011 Authors not listed · 2026
Breaking the delivery barrier: Advancing gene therapy for adult-onset hearing loss
DOI 10.1016/j.ymthe.2026.08.044 Jerusha Naidoo · 2026
In vivo-directed evolution identifies AAV-WM04 as a next-generation vector for potent and sustained hearing restoration in DFNB9
DOI 10.1016/j.ymthe.2026.07.021 Yong Tao, Cenfeng Chu, Zhenzhe Cheng, Shan Yin, Yilin Sun, Ying Chen, Huifang Zhang, Shuyue Bao, Boyu Yang, Baoyi Feng, Xianyu Huang, Yao Lu, Qiuxiang Yang, Xuegao Mao, Qifang Zhou, Chenxi Jin, Zhuo Duan, Guisheng Zhong, Hao Wu · 2026
Protein trans-splicing: Solving the challenge of gene therapy for large genes
DOI 10.1016/j.ymthe.2026.09.017 Rachel R. Stine, James J. Dowling · 2026
Breakthroughs in cell and gene therapy: Innovations in therapeutic modalities for the CNS, targeted delivery, translation, and CMC
DOI 10.1016/j.ymthe.2026.09.026 Allison M. Bradbury · 2026
Christopher D. Porada, PhD (1969–2026): In memoriam
DOI 10.1016/j.ymthe.2026.08.023 Roland W. Herzog, Graça Almeida-Porada · 2026
Targeted antisense oligonucleotide therapy rescues PRPF31 expression in retinitis pigmentosa caused by a splicing mutation
DOI 10.1016/j.ymthe.2026.05.033 Poulami Banik, Felix Zimmann, Prasoon K. Thakur, Lubica Dudakova, Kateřina Večerková, Ondrej Kostov, Marvin H. Caruthers, Michal Kolář, Inka Krejčířová, Marie Vajter, Petra Liskova, Tomáš Bárta, David Staněk · 2026
Sustained relief of chronic pain via a Nav1.7-targeting ASO-siRNA conjugate in rats
DOI 10.1016/j.ymthe.2026.09.039 Bin Ren, Chang Yu, Weiqin Yin, Zhuangchuan Yuan, Hongyu Chen, Yicao Liu, Bin Fang, Chen Zhang, Jianming Wu, Suyuan Liu, Lianchao Gao, Zhiwei Cao, Qingqing Yu, Xin Qiu, Peixia Yu, Guohao Wang · 2026
Closed-loop inhibitory metabotropic glutamate receptor gene therapy for focal refractory epilepsy
DOI 10.1016/j.ymthe.2026.10.023 Laura V. Ussingkær, Dimitri M. Kullmann · 2026
Five decades of gene therapy innovation and regulation in the US and EU: a historical analysis
DOI 10.1016/j.ymthe.2026.10.009 Thomas Wessel van de Voort, Jarno Hoekman, Elisabeth Henrica Maria Moors, Lourens Theo Bloem · 2026
Reprogramming immunosuppressive TMEs with Salmonella-delivered sialidase and COX-2 blockade synergistically enhances cancer immunotherapy
DOI 10.1016/j.ymthe.2026.08.036 Yongjie Zhu, Ming Cheng, Jinling Liu, Mengqi Xiong, Jun Lan, Kang Han, Shengen Yi, Sheng-Nan Jiang, Wenzhi Tan, Jin Hai Zheng · 2026
Inhaled nanosilica orchestrates a pulmonary macrophage-NK cell axis for memory-like NK programming toward synergistic cancer immunotherapy
DOI 10.1016/j.ymthe.2026.08.046 Feng Yuan, Qi Li, Yingnan Liu, Fangfang Liu, Wenhe Luo, Qingle Song, Yanqiao Zeng, Jiacheng Bi, Guofang Zhang, Yang Li · 2026
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