Engineered brain‐targeting exosome for reprogramming immunosuppressive microenvironment of glioblastoma

Abstract The immunosuppressive microenvironment of glioblastoma multiforme (GBM) severely impacts the response to various treatments, including systemic chemotherapy. Targeted reprogramming of immunosuppressive GBM microenvironment using RNA interference (RNAi) is largely restricted by poor brain de...

Full description

Saved in:
Bibliographic Details
Main Authors: Jun Yang, Yong Li, Shaoping Jiang, Yuxin Tian, Mengjie Zhang, Shuai Guo, Pengfei Wu, Jianan Li, Lin Xu, Wenpei Li, Yushu Wang, Huile Gao, Yuanyu Huang, Yuhua Weng, Shaobo Ruan
Format: Article
Language:English
Published: Wiley 2025-04-01
Series:Exploration
Subjects:
Online Access:https://doi.org/10.1002/EXP.20240039
Tags: Add Tag
No Tags, Be the first to tag this record!
_version_ 1850179553380532224
author Jun Yang
Yong Li
Shaoping Jiang
Yuxin Tian
Mengjie Zhang
Shuai Guo
Pengfei Wu
Jianan Li
Lin Xu
Wenpei Li
Yushu Wang
Huile Gao
Yuanyu Huang
Yuhua Weng
Shaobo Ruan
author_facet Jun Yang
Yong Li
Shaoping Jiang
Yuxin Tian
Mengjie Zhang
Shuai Guo
Pengfei Wu
Jianan Li
Lin Xu
Wenpei Li
Yushu Wang
Huile Gao
Yuanyu Huang
Yuhua Weng
Shaobo Ruan
author_sort Jun Yang
collection DOAJ
description Abstract The immunosuppressive microenvironment of glioblastoma multiforme (GBM) severely impacts the response to various treatments, including systemic chemotherapy. Targeted reprogramming of immunosuppressive GBM microenvironment using RNA interference (RNAi) is largely restricted by poor brain delivery efficiency and targeting specificity. Herein, an acid‐cleavable transferrin (Tf) decorated engineering exosome‐based brain‐targeting delivery system (ACTE) was proposed to efficiently deliver small interference RNA towards transform growth factor‐β (siTGF‐β) and doxorubicin (DOX) to GBM site for combination chemo‐immunotherapy. The siTGF‐β and DOX co‐loaded ACTE, termed as DOX&siTGF‐β@ACTE (Ds@ACTE), is designed to specifically recognize the Tf receptor (TfR) on the blood‐brain barrier (BBB). Subsequently, Ds@ACTE undergoes acid‐responsive detachment of Tf within lysosome of brain capillary endothelial cells, leading to the separation of DOX&siTGF‐β@Exo (Ds@Exo) from the Tf‐TfR complex and enhanced BBB transcytosis. After crossing BBB, the separated Ds@Exo can further target GBM cells via the homing effect. In vivo studies validated that Ds@ACTE significantly downregulated the TGF‐β expression to reprogram the immunosuppressive microenvironment, and thereby reinforce the chemotherapeutic effect of DOX and DOX‐induced anti‐tumor immune response. The effectiveness of this strategy not only can provide thinking for designing a more intelligent brain‐targeting system based on engineered exosomes but also explore an effective treatment regimen for GBM.
format Article
id doaj-art-6a12a8e9abd64699b9b840d7fc9f4fe1
institution OA Journals
issn 2766-8509
2766-2098
language English
publishDate 2025-04-01
publisher Wiley
record_format Article
series Exploration
spelling doaj-art-6a12a8e9abd64699b9b840d7fc9f4fe12025-08-20T02:18:28ZengWileyExploration2766-85092766-20982025-04-0152n/an/a10.1002/EXP.20240039Engineered brain‐targeting exosome for reprogramming immunosuppressive microenvironment of glioblastomaJun Yang0Yong Li1Shaoping Jiang2Yuxin Tian3Mengjie Zhang4Shuai Guo5Pengfei Wu6Jianan Li7Lin Xu8Wenpei Li9Yushu Wang10Huile Gao11Yuanyu Huang12Yuhua Weng13Shaobo Ruan14School of Life Science Advanced Research Institute of Multidisciplinary Science Laboratory of Molecular Medicine and Biotherapy. Beijing Institute of Technology Beijing ChinaSchool of Life Science Advanced Research Institute of Multidisciplinary Science Laboratory of Molecular Medicine and Biotherapy. Beijing Institute of Technology Beijing ChinaSchool of Life Science Advanced Research Institute of Multidisciplinary Science Laboratory of Molecular Medicine and Biotherapy. Beijing Institute of Technology Beijing ChinaSchool of Life Science Advanced Research Institute of Multidisciplinary Science Laboratory of Molecular Medicine and Biotherapy. Beijing Institute of Technology Beijing ChinaSchool of Life Science Advanced Research Institute of Multidisciplinary Science Laboratory of Molecular Medicine and Biotherapy. Beijing Institute of Technology Beijing ChinaSchool of Life Science Advanced Research Institute of Multidisciplinary Science Laboratory of Molecular Medicine and Biotherapy. Beijing Institute of Technology Beijing ChinaSchool of Life Science Advanced Research Institute of Multidisciplinary Science Laboratory of Molecular Medicine and Biotherapy. Beijing Institute of Technology Beijing ChinaSchool of Life Science Advanced Research Institute of Multidisciplinary Science Laboratory of Molecular Medicine and Biotherapy. Beijing Institute of Technology Beijing ChinaSchool of Life Science Advanced Research Institute of Multidisciplinary Science Laboratory of Molecular Medicine and Biotherapy. Beijing Institute of Technology Beijing ChinaSchool of Life Science Advanced Research Institute of Multidisciplinary Science Laboratory of Molecular Medicine and Biotherapy. Beijing Institute of Technology Beijing ChinaDepartment of Biomedical Engineering Tufts University Medford Massachusetts USAWest China School of Pharmacy Sichuan University Chengdu ChinaSchool of Life Science Advanced Research Institute of Multidisciplinary Science Laboratory of Molecular Medicine and Biotherapy. Beijing Institute of Technology Beijing ChinaSchool of Life Science Advanced Research Institute of Multidisciplinary Science Laboratory of Molecular Medicine and Biotherapy. Beijing Institute of Technology Beijing ChinaSchool of Life Science Advanced Research Institute of Multidisciplinary Science Laboratory of Molecular Medicine and Biotherapy. Beijing Institute of Technology Beijing ChinaAbstract The immunosuppressive microenvironment of glioblastoma multiforme (GBM) severely impacts the response to various treatments, including systemic chemotherapy. Targeted reprogramming of immunosuppressive GBM microenvironment using RNA interference (RNAi) is largely restricted by poor brain delivery efficiency and targeting specificity. Herein, an acid‐cleavable transferrin (Tf) decorated engineering exosome‐based brain‐targeting delivery system (ACTE) was proposed to efficiently deliver small interference RNA towards transform growth factor‐β (siTGF‐β) and doxorubicin (DOX) to GBM site for combination chemo‐immunotherapy. The siTGF‐β and DOX co‐loaded ACTE, termed as DOX&siTGF‐β@ACTE (Ds@ACTE), is designed to specifically recognize the Tf receptor (TfR) on the blood‐brain barrier (BBB). Subsequently, Ds@ACTE undergoes acid‐responsive detachment of Tf within lysosome of brain capillary endothelial cells, leading to the separation of DOX&siTGF‐β@Exo (Ds@Exo) from the Tf‐TfR complex and enhanced BBB transcytosis. After crossing BBB, the separated Ds@Exo can further target GBM cells via the homing effect. In vivo studies validated that Ds@ACTE significantly downregulated the TGF‐β expression to reprogram the immunosuppressive microenvironment, and thereby reinforce the chemotherapeutic effect of DOX and DOX‐induced anti‐tumor immune response. The effectiveness of this strategy not only can provide thinking for designing a more intelligent brain‐targeting system based on engineered exosomes but also explore an effective treatment regimen for GBM.https://doi.org/10.1002/EXP.20240039chemo‐resistanceengineered exosomesglioblastomaimmunosuppressive microenvironmentRNA interference
spellingShingle Jun Yang
Yong Li
Shaoping Jiang
Yuxin Tian
Mengjie Zhang
Shuai Guo
Pengfei Wu
Jianan Li
Lin Xu
Wenpei Li
Yushu Wang
Huile Gao
Yuanyu Huang
Yuhua Weng
Shaobo Ruan
Engineered brain‐targeting exosome for reprogramming immunosuppressive microenvironment of glioblastoma
Exploration
chemo‐resistance
engineered exosomes
glioblastoma
immunosuppressive microenvironment
RNA interference
title Engineered brain‐targeting exosome for reprogramming immunosuppressive microenvironment of glioblastoma
title_full Engineered brain‐targeting exosome for reprogramming immunosuppressive microenvironment of glioblastoma
title_fullStr Engineered brain‐targeting exosome for reprogramming immunosuppressive microenvironment of glioblastoma
title_full_unstemmed Engineered brain‐targeting exosome for reprogramming immunosuppressive microenvironment of glioblastoma
title_short Engineered brain‐targeting exosome for reprogramming immunosuppressive microenvironment of glioblastoma
title_sort engineered brain targeting exosome for reprogramming immunosuppressive microenvironment of glioblastoma
topic chemo‐resistance
engineered exosomes
glioblastoma
immunosuppressive microenvironment
RNA interference
url https://doi.org/10.1002/EXP.20240039
work_keys_str_mv AT junyang engineeredbraintargetingexosomeforreprogrammingimmunosuppressivemicroenvironmentofglioblastoma
AT yongli engineeredbraintargetingexosomeforreprogrammingimmunosuppressivemicroenvironmentofglioblastoma
AT shaopingjiang engineeredbraintargetingexosomeforreprogrammingimmunosuppressivemicroenvironmentofglioblastoma
AT yuxintian engineeredbraintargetingexosomeforreprogrammingimmunosuppressivemicroenvironmentofglioblastoma
AT mengjiezhang engineeredbraintargetingexosomeforreprogrammingimmunosuppressivemicroenvironmentofglioblastoma
AT shuaiguo engineeredbraintargetingexosomeforreprogrammingimmunosuppressivemicroenvironmentofglioblastoma
AT pengfeiwu engineeredbraintargetingexosomeforreprogrammingimmunosuppressivemicroenvironmentofglioblastoma
AT jiananli engineeredbraintargetingexosomeforreprogrammingimmunosuppressivemicroenvironmentofglioblastoma
AT linxu engineeredbraintargetingexosomeforreprogrammingimmunosuppressivemicroenvironmentofglioblastoma
AT wenpeili engineeredbraintargetingexosomeforreprogrammingimmunosuppressivemicroenvironmentofglioblastoma
AT yushuwang engineeredbraintargetingexosomeforreprogrammingimmunosuppressivemicroenvironmentofglioblastoma
AT huilegao engineeredbraintargetingexosomeforreprogrammingimmunosuppressivemicroenvironmentofglioblastoma
AT yuanyuhuang engineeredbraintargetingexosomeforreprogrammingimmunosuppressivemicroenvironmentofglioblastoma
AT yuhuaweng engineeredbraintargetingexosomeforreprogrammingimmunosuppressivemicroenvironmentofglioblastoma
AT shaoboruan engineeredbraintargetingexosomeforreprogrammingimmunosuppressivemicroenvironmentofglioblastoma