# Molecular MRI for in vivo tracking of gene editing and gene edited cells

> **NIH NIH UH3** · UNIVERSITY OF CALIFORNIA BERKELEY · 2021 · $481,500

## Abstract

Project Summary
 Gene therapy represents a potential mechanism to restore gene expression patterns in failing organs,
particularly in heart failure where downregulated expression of multiple proteins contributes to diastolic failure.
Methods for in vivo gene editing have undergone significant development, particularly with novel engineered
variants of adeno-associated viral (AAV) vectors for delivery of transgenes or as vehicles for CRISPR/Cas9.
However, verification of successful gene editing and longitudinal monitoring of spatiotemporal expression
patterns still require invasive biopsies. Biopsies suffer from spatial sampling error, inflict pain, and in patients
with heart failure increase the risk of sudden death. Serial non-invasive and multi-organ quantification of the
delivery and transduction of gene editing cargo and subsequent transgene expression would provide critical
data for further development of gene therapies, and potentially for comprehensive patient monitoring. Magnetic
resonance imaging (MRI), which has a large install base throughout the United States, is used as part of
routine clinical assessment of cardiac structure and function. An emerging MRI approach termed chemical
exchange saturation transfer (CEST) utilizes the endogenous exchange of magnetization between
macromolecules and water for in vivo molecular imaging. We have previously developed CEST-MRI methods
for non-invasive cardiac imaging of tissue fibrosis, metabolic dysfunction, cell tracking, and most recently to
quantify the expression of a genetically encoded 50-Lysine reporter peptide. In this proposal, we seek to
develop CEST-MRI methods that exploit the surface Lysine residues of the AAV2 viral capsid protein 3 (VP3)
for endogenous CEST-MRI of cellular AAV2 transduction and endosomal escape. Next, we seek to combine
such assessment with CEST-MRI of spatiotemporal patterns of transgene expression in the heart and liver
alongside corresponding changes in cardiac structure/function. If successful, these methods can be easily
implemented on existing clinical MRI scanners, and provide an endogenous mechanism for tracking of gene
editing cargo and subsequent multi-scale outcomes without the need for biopsy.

## Key facts

- **NIH application ID:** 10450896
- **Project number:** 4UH3EB028908-03
- **Recipient organization:** UNIVERSITY OF CALIFORNIA BERKELEY
- **Principal Investigator:** MORIEL VANDSBURGER
- **Activity code:** UH3 (R01, R21, SBIR, etc.)
- **Funding institute:** NIH
- **Fiscal year:** 2021
- **Award amount:** $481,500
- **Award type:** 4N
- **Project period:** 2019-09-03 → 2023-07-31

## Primary source

NIH RePORTER: https://reporter.nih.gov/project-details/10450896

## Citation

> US National Institutes of Health, RePORTER application 10450896, Molecular MRI for in vivo tracking of gene editing and gene edited cells (4UH3EB028908-03). Retrieved via AI Analytics 2026-05-22 from https://api.ai-analytics.org/grant/nih/10450896. Licensed CC0.

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