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.
Authors
- Takao K. Hensch (ORCID: https://orcid.org/0000-0003-0748-8152)
- Zhiping Yang (ORCID: https://orcid.org/0000-0003-0285-7633)
- Samuel A. Theuerkauf (ORCID: https://orcid.org/0000-0002-4767-6695)
- Sengjin Choi (ORCID: https://orcid.org/0000-0003-0379-1701)
- Joseph F. Nabhan (ORCID: https://orcid.org/0000-0002-9600-2347)
- Christian J. Buchholz (ORCID: https://orcid.org/0000-0002-9837-7345)
- Ramón María Alvargonzález Rodríguez (ORCID: https://orcid.org/0000-0002-6770-5123)
- Zunwei Chen (ORCID: https://orcid.org/0000-0001-7773-1635)
- Quan Lu (ORCID: https://orcid.org/0000-0003-3915-0762)
- Qiyu Wang (ORCID: https://orcid.org/0000-0003-4959-7437)
- Zhi Qiao
Institutions
- Boston Children's Hospital (US)
- Harvard University (US)
- NewGen Therapeutics (United States) (US)
- Paul Ehrlich Institut (DE)
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