# Engineering novel delivery systems with synthetic biology

> **NIH NIH R21** · UTAH STATE HIGHER EDUCATION SYSTEM--UNIVERSITY OF UTAH · 2020 · $228,750

## Abstract

Project Summary
The goal of this proposal is to increase our understanding of the therapeutic benefits of using platelets as
delivery systems for disease treatments, with the long-term goal of developing platelet-based therapeutic cell
treatments for metabolic diseases, specifically, lysosomal storage diseases (LSDs). LSDs are inherited
metabolic diseases that are characterized by an abnormal buildup of various toxic materials in the body’s cells
as a result of lysosomal enzyme deficiencies. These malfunctioning enzymes represent a group of about 50
different genetic diseases and, though individually rare, their combined prevalence is estimated to be 1 in
every 8,000 births. LSDs affect different parts of the body including the skeleton, brain, skin, heart, and central
nervous system. All patients with LSD have a limited life expectancy.
Platelets are anucleate blood cells that circulate throughout the body and play an important role in
homeostasis, wound healing, angiogenesis, inflammation, and clot formation. Platelets are naturally filled with
secretory granules that store large amounts of proteins, which are formed from the cytoplasm of
megakaryocytes (MKs), their precursor cells. When platelets are activated, a large number of bioactive
proteins are released from their granules to participate in a myriad of physiological processes. We propose to
take advantage of platelets’ innate storage, trafficking, and release capacities, to engineer them as delivery
vehicles for the development of next generation delivery methods for lysosomal enzymes to treat patients with
LSDs. My central vision is to engineer blood platelets to control the secretion of enzymes required for proper
lysosomal function as a therapeutic treatment for patients with LSDs. I will build upon my previous work where
I have developed novel genetic tools and circuits to re-engineer cells to perform specific tasks to systematically
design, build, and characterize new genetic tools for packaging lysosomal enzymes into platelets. Additionally,
I will rationally design receptors that are capable of activating platelets to trigger the release of enzymes upon
binding to specific drugs and/or binding to tissue specific peptides. Success from this proposal will result in
paradigm-shifting therapies with unprecedented levels of flexibility, precision, and personalization. The
resulting engineered platelets will be the most sophisticated therapeutic agents ever developed for treating
metabolic disorders. Furthermore, the genetic tools and design principles developed here will serve as a
general platform that can be combined with any other treatments to complement existing therapies, thus this
work will have an immediate and broad impact on many metabolic disorders.

## Key facts

- **NIH application ID:** 9821206
- **Project number:** 5R21EB025413-03
- **Recipient organization:** UTAH STATE HIGHER EDUCATION SYSTEM--UNIVERSITY OF UTAH
- **Principal Investigator:** Tara Lynn Deans
- **Activity code:** R21 (R01, R21, SBIR, etc.)
- **Funding institute:** NIH
- **Fiscal year:** 2020
- **Award amount:** $228,750
- **Award type:** 5
- **Project period:** 2018-01-01 → 2020-11-30

## Primary source

NIH RePORTER: https://reporter.nih.gov/project-details/9821206

## Citation

> US National Institutes of Health, RePORTER application 9821206, Engineering novel delivery systems with synthetic biology (5R21EB025413-03). Retrieved via AI Analytics 2026-05-23 from https://api.ai-analytics.org/grant/nih/9821206. Licensed CC0.

---

*[NIH grants dataset](/datasets/nih-grants) · CC0 1.0*