FORCE platform overcomes barriers of oligonucleotide delivery to muscle and corrects myotonic dystrophy features in preclinical models

FORCE platform overcomes barriers of oligonucleotide delivery to muscle and corrects myotonic dystrophy features in preclinical models

Abstract Background We developed the FORCETM platform to overcome limitations of oligonucleotide delivery to muscle and enable their applicability to neuromuscular disorders. The platform consists of an antigen-binding fragment, highly specific for the human transferrin receptor 1 (TfR1), conjugated...

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Main Authors: Timothy Weeden, Tyler Picariello, Brendan Quinn, Sean Spring, Pei-Yi Shen, Qifeng Qiu, Benjamin F. Vieira, Lydia Schlaefke, Ryan J. Russo, Ya-An Chang, Jin Cui, Monica Yao, Aiyun Wen, Nelson Hsia, Tama Evron, Katy Ovington, Pei-Ni Tsai, Nicholas Yoder, Bo Lan, Reshmii Venkatesan, John Hall, Cody A. Desjardins, Mo Qatanani, Scott Hilderbrand, John Najim, Zhenzhi Tang, Matthew K. Tanner, Romesh Subramanian, Charles A. Thornton, Oxana Ibraghimov-Beskrovnaya, Stefano Zanotti
Format: Article
Language:English
Published: Nature Portfolio 2025-01-01
Series:Communications Medicine
Online Access:https://doi.org/10.1038/s43856-025-00733-w
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