journal article·2026-06-15Open access

Ultrasound‐Actuated Gene Editing in Human Kidney Organoids

Michael A. Miller, Nicole Vo, Utkarsh, Ivan Sokirniy, Justin Pritchard, Benjamin S. Freedman, Scott H. Medina

Abstract

ABSTRACT Efficient delivery of gene editing ribonucleoproteins (RNPs) into the interior of solid tissues remains a key hurdle to the clinical translation of non‐viral CRISPR‐Cas9 technologies. Here, we report acoustically‐actuated peptide nanoemulsions (NPeps) that can be spatiotemporally guided and activated by ultrasound to ballistically deliver RNPs into cells within the bulk of dense 3D cellular structures. Using human kidney organoids as a model, we demonstrate NPep vectors improve the spatial profile of gene editing in the organoid mass relative to commercial lipofection reagents, without disruption of tissue structure or qualitative viability features. This technologic paradigm is poised to advance imaging‐guided, deep tissue RNP delivery modalities to expand the clinical diagnostic and therapeutic potential of CRISPR‐Cas9 editing strategies.
Source: crossref · Quality: baseline

Research context

OpenAlex topics: CRISPR and Genetic Engineering · RNA Interference and Gene Delivery · Ultrasound and Hyperthermia Applications

SciVX discovery tags are terminology-based. OpenAlex topics are source-provided classifications.

Affiliations

Department of Biomedical Engineering Pennsylvania State University University Park Pennsylvania USA

Division of Nephrology University of Washington School of Medicine Seattle Washington USA

Kidney Research Institute University of Washington School of Medicine Seattle Washington USA

Institute for Stem Cell and Regenerative Medicine University of Washington School of Medicine Seattle Washington USA

Department of Medicine University of Washington School of Medicine Seattle Washington USA

Molecular, Cellular, and Integrative Biosciences Program Pennsylvania State University, University Park Pennsylvania USA

Huck Institutes of the Life Sciences Pennsylvania State University University Park Pennsylvania USA

Department of Laboratory Medicine & Pathology University of Washington School of Medicine Seattle Washington USA

Center for Biodevices Pennsylvania State University University Park Pennsylvania USA

Funding

National Science Foundation

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