Evidence map›Paper›PMID 35595757›Full record

ArticleNature communications2022

Engineered Cas12i2 is a versatile high-efficiency platform for therapeutic genome editing.

Colin McGaw, Anthony J Garrity, Gabrielle Z Munoz, Jeffrey R Haswell, Sejuti Sengupta, Elise Keston-Smith, Pratyusha Hunnewell, Alexa Ornstein, Mishti Bose, Quinton Wessells and 15 more

Open access · goldAbstract read
In one paragraph

Article in Nature communications, 2022. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Cited by 24 papers.

0numbers the graph read from it
0cells of the map it votes in
24citing papers in PubMed
3.9field-weighted citation impact, top 5% of its field
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

24 citing papers in PubMed, 48 citations in OpenAlex.

  1. Article
  2. Article
  3. Article
  4. Messenger RNA and guide RNA distributions in lipid nanoparticles impact gene-editing efficiency in vivo.Molecular therapy : the journal of the American Society of Gene Therapy · 2026
    Article
  5. Article
  6. Article
  7. Article
  8. Article
  9. Article
  10. Genome Editing in Mouse Embryo Using the CRISPR/Cas12i3 System.International journal of molecular sciences · 2025
    Article
  11. Current Non-Viral-Based Strategies to Manufacture CAR-T Cells.International journal of molecular sciences · 2024
    Review
  12. Article
  13. Review
  14. Article
  15. Rational Design of Enhanced Nme2Cas9 and Nme2bioRxiv : the preprint server for biology · 2024
    Article
  16. Review
  17. Article
  18. CRISPR/Cpf1-FOKI-induced gene editing inSynthetic and systems biotechnology · 2024
    Article
  19. Genome editing in rice using CRISPR/Cas12i3.Plant biotechnology journal · 2024
    Article
  20. 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

25 authors at 1 institution in 1 country.

Colin McGaw *Arbor Biotechnologies, 20 Acorn Park Drive, Tower 500, Cambridge, MA, USA.
Anthony J Garrity *Arbor Biotechnologies, 20 Acorn Park Drive, Tower 500, Cambridge, MA, USA.
Gabrielle Z MunozArbor Biotechnologies, 20 Acorn Park Drive, Tower 500, Cambridge, MA, USA.
Jeffrey R HaswellArbor Biotechnologies, 20 Acorn Park Drive, Tower 500, Cambridge, MA, USA.
Sejuti SenguptaArbor Biotechnologies, 20 Acorn Park Drive, Tower 500, Cambridge, MA, USA.
Elise Keston-SmithArbor Biotechnologies, 20 Acorn Park Drive, Tower 500, Cambridge, MA, USA.
Pratyusha HunnewellArbor Biotechnologies, 20 Acorn Park Drive, Tower 500, Cambridge, MA, USA.
Alexa OrnsteinArbor Biotechnologies, 20 Acorn Park Drive, Tower 500, Cambridge, MA, USA.
Mishti BoseArbor Biotechnologies, 20 Acorn Park Drive, Tower 500, Cambridge, MA, USA.
Quinton WessellsArbor Biotechnologies, 20 Acorn Park Drive, Tower 500, Cambridge, MA, USA.ORCID 0000-0002-8979-4454
Noah JakimoArbor Biotechnologies, 20 Acorn Park Drive, Tower 500, Cambridge, MA, USA.
Paul YanArbor Biotechnologies, 20 Acorn Park Drive, Tower 500, Cambridge, MA, USA.
Huaibin ZhangArbor Biotechnologies, 20 Acorn Park Drive, Tower 500, Cambridge, MA, USA.
Lauren E AlfonseArbor Biotechnologies, 20 Acorn Park Drive, Tower 500, Cambridge, MA, USA.
Roy ZiblatArbor Biotechnologies, 20 Acorn Park Drive, Tower 500, Cambridge, MA, USA.
Jason M CarteArbor Biotechnologies, 20 Acorn Park Drive, Tower 500, Cambridge, MA, USA.
Wei-Cheng LuArbor Biotechnologies, 20 Acorn Park Drive, Tower 500, Cambridge, MA, USA.
Derek CerchioneArbor Biotechnologies, 20 Acorn Park Drive, Tower 500, Cambridge, MA, USA.
Brendan HilbertArbor Biotechnologies, 20 Acorn Park Drive, Tower 500, Cambridge, MA, USA.
Shanmugapriya SothiselvamArbor Biotechnologies, 20 Acorn Park Drive, Tower 500, Cambridge, MA, USA.
Winston X YanArbor Biotechnologies, 20 Acorn Park Drive, Tower 500, Cambridge, MA, USA.
David R ChengArbor Biotechnologies, 20 Acorn Park Drive, Tower 500, Cambridge, MA, USA.
David A ScottArbor Biotechnologies, 20 Acorn Park Drive, Tower 500, Cambridge, MA, USA.
Tia DiTommasoArbor Biotechnologies, 20 Acorn Park Drive, Tower 500, Cambridge, MA, USA. tditommaso@arbor.bio.ORCID 0000-0001-5309-300X
Shaorong ChongArbor Biotechnologies, 20 Acorn Park Drive, Tower 500, Cambridge, MA, USA.
Arbor Research Collaborative for Health · US

Funding

No grant is acknowledged in the PubMed record.

6 · The paper itself

Abstract

The CRISPR-Cas type V-I is a family of Cas12i-containing programmable nuclease systems guided by a short crRNA without requirement for a tracrRNA. Here we present an engineered Type V-I CRISPR system (Cas12i), ABR-001, which utilizes a tracr-less guide RNA. The compact Cas12i effector is capable of self-processing pre-crRNA and cleaving dsDNA targets, which facilitates versatile delivery options and multiplexing, respectively. We apply an unbiased mutational scanning approach to enhance initially low editing activity of Cas12i2. The engineered variant, ABR-001, exhibits broad genome editing capability in human cell lines, primary T cells, and CD34+ hematopoietic stem and progenitor cells, with both robust efficiency and high specificity. In addition, ABR-001 achieves a high level of genome editing when delivered via AAV vector to HEK293T cells. This work establishes ABR-001 as a versatile, specific, and high-performance platform for ex vivo and in vivo gene therapy.

Indexed as

CRISPR-Cas SystemsGene EditingEndonucleasesHEK293 CellsHumansRNARNA, Guide, CRISPR-Cas SystemsEndonucleasesRNARNA, Guide, CRISPR-Cas Systems

Identifiers

PMID35595757
PMCPMC9122993
OpenAlexW4280544078

What OpenQuestion holds

Textmetadata
LicenceCC BY
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.