Evidence map›Paper›PMID 34475560›Full record

ArticleNature biotechnology2022

Efficient CRISPR editing with a hypercompact Cas12f1 and engineered guide RNAs delivered by adeno-associated virus.

Do Yon Kim, Jeong Mi Lee, Su Bin Moon, Hyun Jung Chin, Seyeon Park, Youjung Lim, Daesik Kim, Taeyoung Koo, Jeong-Heon Ko, Yong-Sam Kim

Open access · hybridAbstract read
In one paragraph

Article in Nature biotechnology, 2022. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Cited by 171 papers.

0numbers the graph read from it
0cells of the map it votes in
171citing papers in PubMed
16.5field-weighted citation impact, top 1% 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

171 citing papers in PubMed, 264 citations in OpenAlex.

  1. Article
  2. RNA therapeutics: current status and future directions.Signal transduction and targeted therapy · 2026
    Review
  3. Review
  4. Article
  5. Review
  6. Article
  7. Review
  8. Rapid and efficient detection of Mycoplasma pneumoniae via an ERA/Cas12f1_ge4.1-coupled colloidal gold lateral flow assay.European journal of clinical microbiology & infectious diseases : official publication of the European Society of Clinical Microbiology · 2026
    Article
  9. Article
  10. Review
  11. Clinical translation of epigenome editing technologies.Current opinion in biomedical engineering · 2026
    Article
  12. Article
  13. Article
  14. Review
  15. Review
  16. Review
  17. Article
  18. Article
  19. Article
  20. Review

111 more citing papers are in PubMed but not listed here.

4 · The record

Corrections and comments

5 · Who and what money

Authors and funding

10 authors at 3 institutions in 1 country.

Do Yon Kim *Genome Editing Research Center, Korea Research Institute of Bioscience & BioTechnology, Daejeon, Republic of Korea.
Jeong Mi Lee *Genome Editing Research Center, Korea Research Institute of Bioscience & BioTechnology, Daejeon, Republic of Korea.ORCID http://orcid.org/0000-0001-6829-2802
Su Bin MoonGenome Editing Research Center, Korea Research Institute of Bioscience & BioTechnology, Daejeon, Republic of Korea.
Hyun Jung ChinGenome Editing Research Center, Korea Research Institute of Bioscience & BioTechnology, Daejeon, Republic of Korea.
Seyeon ParkGenKOre, Daejeon, Republic of Korea.
Youjung LimGenKOre, Daejeon, Republic of Korea.
Daesik KimGenome Editing Research Center, Korea Research Institute of Bioscience & BioTechnology, Daejeon, Republic of Korea.ORCID http://orcid.org/0000-0003-0806-5979
Taeyoung KooDepartment of Biomedical and Pharmaceutical Sciences, Graduate School, Kyung Hee University, Seoul, Republic of Korea.
Jeong-Heon KoGenome Editing Research Center, Korea Research Institute of Bioscience & BioTechnology, Daejeon, Republic of Korea.
Yong-Sam KimGenome Editing Research Center, Korea Research Institute of Bioscience & BioTechnology, Daejeon, Republic of Korea. omsys1@kribb.re.kr.ORCID http://orcid.org/0000-0002-3133-3954
Korea Research Institute of Bioscience and Biotechnology · KRKorea Research Institute for Human Settlements · KRKyung Hee University · KR

Funding

No grant is acknowledged in the PubMed record.

6 · The paper itself

Abstract

Gene therapy would benefit from a miniature CRISPR system that fits into the small adeno-associated virus (AAV) genome and has high cleavage activity and specificity in eukaryotic cells. One of the most compact CRISPR-associated nucleases yet discovered is the archaeal Un1Cas12f1. However, Un1Cas12f1 and its variants have very low activity in eukaryotic cells. In the present study, we redesigned the natural guide RNA of Un1Cas12f1 at five sites: the 5' terminus of the trans-activating CRISPR RNA (tracrRNA), the tracrRNA-crRNA complementary region, a penta(uridinylate) sequence, the 3' terminus of the crRNA and a disordered stem 2 region in the tracrRNA. These optimizations synergistically increased the average indel frequency by 867-fold. The optimized Un1Cas12f1 system enabled efficient, specific genome editing in human cells when delivered by plasmid vectors, PCR amplicons and AAV. As Un1Cas12f1 cleaves outside the protospacer, it can be used to create large deletions efficiently. The engineered Un1Cas12f1 system showed efficiency comparable to that of SpCas9 and specificity similar to that of AsCas12a.

Indexed as

DependovirusRNA, Guide, CRISPR-Cas SystemsCRISPR-Cas SystemsEndonucleasesGene EditingHumansRNAEndonucleasesRNARNA, Guide, CRISPR-Cas Systems

Identifiers

PMID34475560
PMCPMC8763643
OpenAlexW3197248033

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.