Evidence map›Paper›PMID 41350944›Full record

ArticleNature biotechnology2025

In vivo gene editing of human hematopoietic stem and progenitor cells using envelope-engineered virus-like particles.

Vladimir V Botchkarev, Sean Harrington, Matteo Stoppato, Alexander Justen, Casey Kimber, Anjali Kapuria, Keylie M Gibson, Chi-Shuen Chu, Yuanxin Xu, Kelsey Haugh and 26 more

Abstract read
PubMed Publisher
In one paragraph

Article in Nature biotechnology, 2025. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Cited by 14 papers.

0numbers the graph read from it
0cells of the map it votes in
14citing 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

14 citing papers in PubMed.

  1. Article
  2. Article
  3. Article
  4. Article
  5. Article
  6. Genetic influences on haematopoiesis.Nature reviews. Genetics · 2026
    Review
  7. Restoration of the immune system with base editing and non-genotoxic conditioning in a Rag2 point-mutant mouse model.Molecular therapy : the journal of the American Society of Gene Therapy · 2026
    Article
  8. Review
  9. Article
  10. Review
  11. Article
  12. Review
  13. Review
  14. 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

36 authors.

Vladimir V Botchkarev *Sana Biotechnology, Inc., Cambridge, MA, USA.
Sean Harrington *Sana Biotechnology, Inc., Cambridge, MA, USA.
Matteo StoppatoSana Biotechnology, Inc., Cambridge, MA, USA.
Alexander JustenSana Biotechnology, Inc., Cambridge, MA, USA.
Casey KimberSana Biotechnology, Inc., Cambridge, MA, USA.
Anjali KapuriaSana Biotechnology, Inc., Cambridge, MA, USA.
Keylie M GibsonSana Biotechnology, Inc., Cambridge, MA, USA.
Chi-Shuen ChuSana Biotechnology, Inc., Cambridge, MA, USA.
Yuanxin XuSana Biotechnology, Inc., Cambridge, MA, USA.
Kelsey HaughSana Biotechnology, Inc., Cambridge, MA, USA.ORCID http://orcid.org/0000-0002-4277-2493
Ramya AnkalaSana Biotechnology, Inc., Cambridge, MA, USA.
Nathan KipnissSana Biotechnology, Inc., Cambridge, MA, USA.ORCID http://orcid.org/0000-0001-9864-942X
Andre DeGrootSana Biotechnology, Inc., Cambridge, MA, USA.ORCID http://orcid.org/0000-0002-7447-938X
Emerson MooreSana Biotechnology, Inc., Cambridge, MA, USA.
Rowena de JesusSana Biotechnology, Inc., Cambridge, MA, USA.
Funmi AdewaleSana Biotechnology, Inc., Cambridge, MA, USA.ORCID http://orcid.org/0000-0002-8808-5277
Kathy DanielsSana Biotechnology, Inc., Cambridge, MA, USA.
Samantha CrockerSana Biotechnology, Inc., Cambridge, MA, USA.
Anna LiangSana Biotechnology, Inc., Cambridge, MA, USA.
Shannon JoyceSana Biotechnology, Inc., Cambridge, MA, USA.
Nicole RobertoSana Biotechnology, Inc., Cambridge, MA, USA.ORCID http://orcid.org/0009-0001-9577-1371
Pamela AngelSana Biotechnology, Inc., Cambridge, MA, USA.
Derek SmithSana Biotechnology, Inc., Cambridge, MA, USA.
Athena WongSana Biotechnology, Inc., Cambridge, MA, USA.
Nick AdlerSana Biotechnology, Inc., Cambridge, MA, USA.
Valeria BerlfeinSana Biotechnology, Inc., Cambridge, MA, USA.
Shirisha AmatyaSana Biotechnology, Inc., Cambridge, MA, USA.
Patricia CruiteSana Biotechnology, Inc., Cambridge, MA, USA.
Shariq UsmaniSana Biotechnology, Inc., Cambridge, MA, USA.
Albert RuzoSana Biotechnology, Inc., Cambridge, MA, USA.
Benjamin FerlandSana Biotechnology, Inc., Cambridge, MA, USA.
Sundeep ChandraSana Biotechnology, Inc., Cambridge, MA, USA.
Edward J RebarSana Biotechnology, Inc., Cambridge, MA, USA.
Jagesh V ShahSana Biotechnology, Inc., Cambridge, MA, USA.
Kyle TrudeauSana Biotechnology, Inc., Cambridge, MA, USA.
Luca BiascoSana Biotechnology, Inc., Cambridge, MA, USA. l.biasco@ucl.ac.uk.ORCID http://orcid.org/0000-0003-4022-7640

Funding

No grant is acknowledged in the PubMed record.

6 · The paper itself

Abstract

Engineered virus-like particles (VLPs) are a promising technology for in vivo gene editing of human hematopoietic stem and progenitor cells (HSPCs). Here we design and test two different VLP envelopes for human HSPC editing in vitro and in vivo. The first is an optimized version of the baboon envelope BaEVTR, which efficiently transduces human HSPCs in vitro. We show that the optimized BaEVTR VLP enables in vivo editing of β2 microglobulin in long-term human HSPCs (31% at 8 weeks after dosing) and editing of two hemoglobinopathy-relevant loci, BCL11A and HBG1/2 (26% and 7.5%, respectively, at 5 days after dosing), inducing fetal hemoglobin. Our second VLP design uses a CD133-targeted envelope designed to reduce the transduction of mature blood cells and achieves higher in vivo specificity for HSPCs compared to the optimized BaEVTR VLP. As avoiding delivery in filter organs such as the liver would enhance efficiency and safety, we also demonstrate that both VLPs avoid human hepatocytes in a humanized liver model.

Identifiers

PMID41350944

What OpenQuestion holds

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Registered trials

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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.