# Gene Delivery Nanoparticles to Treat Glioblastoma

> **NIH NIH R01** · JOHNS HOPKINS UNIVERSITY · 2020 · $364,921

## Abstract

There is a need to create new biotechnology to safely and effectively deliver miRNA to target cells 
of interest in the brain. We propose to develop enhanced polymeric nanoparticles designed to be 
stable extracellularly and bioreducible intracellularly, to enable safe and effective delivery of 
miRNA.  We will validate these particles in models of brain cancer as it is a deadly disease that 
is in critical need of new therapies.  In particular, we will evaluate treatment of human 
glioblastoma in orthotopic models compared to the standard of care.  Polymer structures will be 
synthesized and utilized to fabricate non-viral nanoparticles for delivery to multiple brain cell 
types including healthy cells and cancer cells. Nanoparticle formulations will be developed that 
remain stable in cerebrospinal fluid (CSF) and that can spread throughout the brain following 
administration by convection-enhanced delivery (CED).  We will investigate novel miRNA drugs 
capable of overcoming known drug and radiation resistances by targeting tumor-propagating brain 
cancer stem cells (BCSCs).  Novel miRNA combinations will be evaluated in vitro and in vivo. The 
nanoparticles will be tracked to evaluate their intracellular and extracellular delivery and cell 
specificity.  Transport and safety through normal and tumor- bearing rodent brains following CED 
will be analyzed.  Leading nanoparticles will be evaluated in an orthotopic model for human 
glioblastoma using molecular and cell biological endpoints and anti-tumor efficacy will be compared 
to the standard of care as well as in combination with the standard of care. We will create an 
innovative technology for miRNA delivery and validate it in models of brain cancer.

## Key facts

- **NIH application ID:** 9838740
- **Project number:** 5R01CA228133-07
- **Recipient organization:** JOHNS HOPKINS UNIVERSITY
- **Principal Investigator:** Jordan Green
- **Activity code:** R01 (R01, R21, SBIR, etc.)
- **Funding institute:** NIH
- **Fiscal year:** 2020
- **Award amount:** $364,921
- **Award type:** 5
- **Project period:** 2018-12-16 → 2023-11-30

## Primary source

NIH RePORTER: https://reporter.nih.gov/project-details/9838740

## Citation

> US National Institutes of Health, RePORTER application 9838740, Gene Delivery Nanoparticles to Treat Glioblastoma (5R01CA228133-07). Retrieved via AI Analytics 2026-05-22 from https://api.ai-analytics.org/grant/nih/9838740. Licensed CC0.

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