Targeted Intracellular Delivery via Precision Programming of ARRDC1‐Mediated Microvesicles

Efficient and cell-specific delivery remains a major barrier to realising the full therapeutic potential of modalities such as mRNA and CRISPR-based gene editors. Here, we report a versatile delivery platform based on engineered ARRDC1-mediated microvesicles (ARMMs) capable of delivering cargo to defined cell populations. By decorating ARMMs with engineered Nipah virus (NiV)-derived fusion and attachment proteins conjugated to cell-specific ligands, we enable selective binding and membrane fusion-mediated cargo release. ARMMs functionalized with anti-CD8 single-chain variable fragment (scFv) delivered protein, mRNA, or CRISPR-Cas9 base editor selectively to CD8⁺ T cells. Similarly, ARMMs displaying a designed ankyrin repeat protein (DARPin) targeting the GluA4 receptor enabled delivery to parvalbumin-positive (PV⁺) neurons. In vivo, administration of targeted ARMMs resulted in functional delivery to CD8⁺ splenocytes and PV⁺ cortical neurons in mice. These findings establish surface-engineered ARMMs as a programmable and modular system for precision delivery of therapeutic macromolecules, with broad applicability in gene and RNA-based medicine.

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Publication Details

Journal
Journal of Extracellular Vesicles
Published
2025-12-01
DOI
https://doi.org/10.1002/jev2.70199
Citations
2
Primary Topic
Extracellular vesicles in disease
Type
article
Field-Weighted Citation Impact
1.61
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article

Targeted Intracellular Delivery via Precision Programming of ARRDC1‐Mediated Microvesicles

Takao K. Hensch, Zhiping Yang, Samuel A. Theuerkauf, Sengjin Choi et al.
2 citations
Journal of Extracellular Vesicles
Extracellular vesicles in disease
1.61
article

Targeted Intracellular Delivery via Precision Programming of ARRDC1‐Mediated Microvesicles

Takao K. Hensch, Zhiping Yang, Samuel A. Theuerkauf, Sengjin Choi, Joseph F. Nabhan, Christian J. Buchholz, Ramón María Alvargonzález Rodríguez, Zunwei Chen, Quan Lu, Qiyu Wang, Zhi Qiao
article en
2 citations

Abstract

Efficient and cell-specific delivery remains a major barrier to realising the full therapeutic potential of modalities such as mRNA and CRISPR-based gene editors. Here, we report a versatile delivery platform based on engineered ARRDC1-mediated microvesicles (ARMMs) capable of delivering cargo to defined cell populations. By decorating ARMMs with engineered Nipah virus (NiV)-derived fusion and attachment proteins conjugated to cell-specific ligands, we enable selective binding and membrane fusion-mediated cargo release. ARMMs functionalized with anti-CD8 single-chain variable fragment (scFv) delivered protein, mRNA, or CRISPR-Cas9 base editor selectively to CD8⁺ T cells. Similarly, ARMMs displaying a designed ankyrin repeat protein (DARPin) targeting the GluA4 receptor enabled delivery to parvalbumin-positive (PV⁺) neurons. In vivo, administration of targeted ARMMs resulted in functional delivery to CD8⁺ splenocytes and PV⁺ cortical neurons in mice. These findings establish surface-engineered ARMMs as a programmable and modular system for precision delivery of therapeutic macromolecules, with broad applicability in gene and RNA-based medicine.

Journal of Extracellular VesiclesVol. 14(12)
Boston Children's Hospital (US), Harvard University (US), NewGen Therapeutics (United States) (US), Paul Ehrlich Institut (DE)
Openalex Percentile: Top 14%
Extracellular vesicles in disease
1.61
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