Evidence map›Paper›PMID 41535609›Full record

ArticleGene therapy2026

CRISPR-AuNP: physicochemical optimization of a gold nanoparticle platform for cost-effective and modular non-viral gene editing in HSPCs.

Karthikeya S V Gottimukkala, Daniel D Lane, Rachel Cunningham, Haleema S Malik, Youngseo Jwa, Molly E Cassidy, Jack M P Castelli, Mark R Enstrom, Katrina Poljakov, Grady Gastelum and 3 more

Abstract read
In one paragraph

Article in Gene therapy, 2026. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Cited by 2 papers.

0numbers the graph read from it
0cells of the map it votes in
2citing papers in PubMed
–field-weighted citation impact
1 · What the graph read from it

What it found

Each row is one number read from the abstract, on the scale the paper reported it, with its interval. Left of the dashed line favours the treatment, right favours the comparator. Under each row is the sentence it came from. New to these charts? A ten-minute tutorial.

The abstract states no effect estimate the extractor could read, or names no intervention and outcome on the map, so this paper lights no cell and moves no belief. It is still indexed, cited and linked below.

2 · The registry

The trial behind it

Trials whose registry record cites this paper, or whose number appears in the abstract. A trial that started after this paper was published is citing it as background, not reporting it.

Neither the registry nor the abstract names a trial number. If this is a trial report, that itself is worth knowing.

3 · Its place in the literature

Who cites it

2 citing papers in PubMed.

  1. Article
  2. Review
4 · The record

Corrections and comments

PubMed lists nothing against this paper. Absence here is not a guarantee, only a check that was made.

5 · Who and what money

Authors and funding

13 authors.

Karthikeya S V GottimukkalaTranslational Science and Therapeutics Division, Fred Hutchinson Cancer Center, Seattle, WA, USA.
Daniel D LaneTranslational Science and Therapeutics Division, Fred Hutchinson Cancer Center, Seattle, WA, USA.
Rachel CunninghamTranslational Science and Therapeutics Division, Fred Hutchinson Cancer Center, Seattle, WA, USA.
Haleema S MalikTranslational Science and Therapeutics Division, Fred Hutchinson Cancer Center, Seattle, WA, USA.
Youngseo JwaTranslational Science and Therapeutics Division, Fred Hutchinson Cancer Center, Seattle, WA, USA.
Molly E CassidyTranslational Science and Therapeutics Division, Fred Hutchinson Cancer Center, Seattle, WA, USA.
Jack M P CastelliTranslational Science and Therapeutics Division, Fred Hutchinson Cancer Center, Seattle, WA, USA.ORCID 0000-0001-5834-0620
Mark R EnstromTranslational Science and Therapeutics Division, Fred Hutchinson Cancer Center, Seattle, WA, USA.
Katrina PoljakovTranslational Science and Therapeutics Division, Fred Hutchinson Cancer Center, Seattle, WA, USA.
Grady GastelumTranslational Science and Therapeutics Division, Fred Hutchinson Cancer Center, Seattle, WA, USA.
Samuel H HoMetagenomi, Inc., Emeryville, CA, USA.ORCID 0000-0001-7647-0752
Carlos TassaMetagenomi, Inc., Emeryville, CA, USA.
Jennifer E AdairDepartment of Medical Oncology, University of Washington, Seattle, WA, USA. jennifer.adair@umassmed.edu.ORCID 0000-0003-4599-016X

Funding

Translational Bioimaging Core Shared ResourceP30CA015704 · NCI · FRED HUTCHINSON CANCER RESEARCH CENTER · PI Eric Collisson · 1985 to 2026
$296.4M
Stem Cell and Transplantation BiologyU54DK106829 · NIDDK · FRED HUTCHINSON CANCER RESEARCH CENTER · PI DEREK L STIREWALT · 2015 to 2026
$9.1M
In vivo CRISPR engineering of B cells to produce anti-HIV broadly neutralizing antibodies using novel nanoparticlesR01AI167009 · NIAID · UNIV OF MASSACHUSETTS MED SCH WORCESTER · PI Jennifer Eileen Adair, Justin J Taylor · 2022 to 2026
$4.2M
Targeted genetic engineering of B cells to induce protective antibody responses to viral pathogensR01AI158728 · NIAID · UNIV OF MASSACHUSETTS MED SCH WORCESTER · PI Jennifer Eileen Adair, Justin J Taylor · 2022 to 2026
$3.8M
NCI NIH HHS P30 CA015704NIAID NIH HHS R01 AI158728NIAID NIH HHS R01 AI167009NIDDK NIH HHS U54 DK106829
6 · The paper itself

Abstract

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.

Indexed as

CRISPR-Cas SystemsGene EditingGoldHematopoietic Stem CellsMetal NanoparticlesCost-Benefit AnalysisHumansPolyethylene GlycolsPolyethyleneimineGoldPolyethylene GlycolsPolyethyleneimine

Identifiers

PMID41535609
PMCPMC13056566

What OpenQuestion holds

Textmetadata
LicenceCC BY-NC-ND
Read underepoch 390

Registered trials

None linked

Read under generation 80e0d062 · epoch 390. Bibliography from PubMed, PubMed Central and OpenAlex; grants from NIH RePORTER; trial links from ClinicalTrials.gov; estimates, votes and beliefs from the OpenQuestion graph.