# Elucidating a physiological role for ARRDC-1 Mediated Microvesicles (ARMMs): A versatile platform for the delivery of biological therapeutics

> **NIH NIH F31** · HARVARD UNIVERSITY D/B/A HARVARD SCHOOL OF PUBLIC HEALTH · 2020 · $31,300

## Abstract

PROJECT SUMMARY
The delivery of biological therapeutics to cells or tissues is limited by a number of factors,
including poor permeability, target specificity, and immunogenicity. Therefore, there is an urgent
need to develop new technologies to deliver therapeutics more efficiently, safely, and
specifically. We have recently discovered novel small vesicles known as ARMMs (ARRDC1-
Mediated Microvesicles) that are naturally secreted by mammalian cells. We have found that
ARMMs exhibit several important features that make them suitable for delivery of biological
therapeutics: 1) ARMMs can be readily produced by overexpression of the ARRDC1 protein and
are relatively uniform in size (average ~100 nm), 2) specific protein molecules are actively
recruited into ARMMs and are protected by the lipid bilayer from degradation, and 3) ARMMs
can deliver bioactive cargos into recipient cells. Specifically, I have shown that ARMMs can
efficiently package and functionally deliver diverse payloads (tumor suppressor protein
p53, GFP and p53 RNAs, and the CRISPR-Cas9/guide RNA complex) into cultured
recipient cells. Additionally, ARMMs likely elicit little immunogenic response as they are
produced by human cells endogenously and can be found in circulating blood. However,
although ARMMs have shown great promise as ideal vehicles for the delivery of therapeutic
cargos, our studies have been done primarily in cell culture. Thus, key questions remain: does
ARMMs-mediated delivery work in vivo (and what is the tissue distribution of injected ARMMs)?
Therefore, my overarching goal in the proposed study is to investigate the in vivo trafficking
and tissue biodistribution of ARMMS. I plan to characterize ARMMs translocation in vivo
using both C. elegans and mouse models by 1) Defining ARMMs trafficking potential in C.
elegans and by 2) Elucidating ARMMs translocation and tissue bioavailability in mice. This
proof-of-principle study will provide evidence to support further development of ARMMs into a
potentially superior vehicle for delivery of a myriad of biological therapeutics.

## Key facts

- **NIH application ID:** 9831559
- **Project number:** 5F31GM131567-02
- **Recipient organization:** HARVARD UNIVERSITY D/B/A HARVARD SCHOOL OF PUBLIC HEALTH
- **Principal Investigator:** Joseph Beyene
- **Activity code:** F31 (R01, R21, SBIR, etc.)
- **Funding institute:** NIH
- **Fiscal year:** 2020
- **Award amount:** $31,300
- **Award type:** 5
- **Project period:** 2018-11-16 → 2020-11-15

## Primary source

NIH RePORTER: https://reporter.nih.gov/project-details/9831559

## Citation

> US National Institutes of Health, RePORTER application 9831559, Elucidating a physiological role for ARRDC-1 Mediated Microvesicles (ARMMs): A versatile platform for the delivery of biological therapeutics (5F31GM131567-02). Retrieved via AI Analytics 2026-05-23 from https://api.ai-analytics.org/grant/nih/9831559. Licensed CC0.

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