Gene therapyJan 14, 2026

Optimizing gold nanoparticles for affordable and flexible gene editing in blood stem cells

CRISPR Gene Editing Weekly Brief ↗PubMed ↗DOI ↗OA ↗

Updated Jan 16, 2026

Abstract

Efficient gene editing was achieved in primary CD34+ hematopoietic stem and progenitor cells using a novel gold-polymer hybrid nanoparticle platform for less than $70 per million cells treated.

The gold-polymer hybrid nanoparticle system successfully delivered multiple CRISPR systems, including Cas9 and Cas12a, into HSPCs.
Editing efficiency was maintained without compromising cell viability.
The nanoparticle formulation can be assembled in under 2 hours.
This approach offers a scalable and cost-effective solution for gene editing in HSPC research and therapy.

Simplified

Efficient delivery of CRISPR ribonucleoproteins into primary hematopoietic stem and progenitor cells (HSPCs) is essential for durable gene editing therapies but remains challenging. Here, we advance a modular, benchtop-assembled gold-polymer hybrid nanoparticle (CRISPR-AuNP) platform that enables non-viral delivery of multiple CRISPR systems into HSPCs. Guided by a mechanistic understanding of Cas9's interaction with gold surfaces, we engineered the formulation by conjugating pre-formed RNP-polymer complexes, assembled using thiolated polyethyleneimine-polyethylene glycol, to gold nanoparticles. This system achieved efficient editing in primary CD34+ HSPCs for Cas9, Cas12a, and Cas12a-M29-1 without compromising cell viability. Notably, the nanoformulation can be assembled in under 2 h in a PCR tube for less than $70/million HSPCs treated. This work establishes a scalable, cost-effective, and accessible gene editing system with the potential to democratize CRISPR applications in HSPC research and therapy.

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Funding

Competing interests

Competing interests: JEA is a co-inventor on U.S. Patent WO2018226762A1 entitled “Genomic Safe Harbors for Genetic Therapies in Human Stem Cells and Engineered Nanoparticles to Provide Targeted Genetic Therapies”. All other authors declare no competing interests. Ethical approval: Healthy adults donated cells for this study through the Core Center of Excellence in Hematology at Fred Hutchinson Cancer Center under an approved protocol (no. 985.03), which was compliant with the Declaration of Helsinki and Belmont Report.

PubMed

Optimizing gold nanoparticles for affordable and flexible gene editing in blood stem cells

Abstract

Full Text

Funding

Competing interests

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