Accelerated Endosomal Escape of Splice-Switching Oligonucleotides Enables Efficient Hepatic Splice Correction.

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Tác giả: Fatih Alioglu, Volker Baumann, Denise Benisch, Theresa Birkfeld, Auke Boersma, Simon Decker, Alina Duran, Enes Dusinovic, Helene Fasching, Levente Ferencz, Nadine Follrich, Laura Bocanegra Gómez, Filip Ilievski, Christoph Kugler, Ulrich Lächelt, Dajana Mihalicokova, Sandra Milenkovic, Manfred Ogris, Daniel Paunov, Thomas Rülicke, Haider Sami, Karla Singeorzan, Silvia Weiss, Nikolaus Zehetmayer, Dejan Zivanonvic

Ngôn ngữ: eng

Ký hiệu phân loại: 539.732 High-voltage accelerators

Thông tin xuất bản: United States : ACS applied materials & interfaces , 2025

Mô tả vật lý:

Bộ sưu tập: NCBI

ID: 61712

Splice-switching oligonucleotides (SSOs) can restore protein functionality in pathologies and are promising tools for manipulating the RNA-splicing machinery. Delivery vectors can considerably improve SSO functionality in vivo and allow dose reduction, thereby addressing the challenges of RNA-targeted therapeutics. Here, we report a biocompatible SSO nanocarrier, based on redox-responsive disulfide cross-linked low-molecular-weight linear polyethylenimine (cLPEI), for overcoming multiple biological barriers from subcellular compartments to en-route serum stability and finally in vivo delivery challenges. Intracellularly responsive cross-links of cLPEI significantly accelerated the endosomal escape and offered efficient SSO release to the cell's nucleus, thereby leading to high splice correction in vitro. In vivo performance of cLPEI-SSOs was investigated in a novel transgenic mouse model for splice correction, spatiotemporal tracking of SSO delivery in wild-type mice, and biodistribution in a colorectal cancer peritoneal metastasis model. A single intravenous application of 5 mg kg
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