Evidence map›Paper›PMID 38256061›Full record

ArticleInternational journal of molecular sciences2024

CRISPR-Cas12a for Highly Efficient and Marker-Free Targeted Integration in Human Pluripotent Stem Cells.

Ruba Hammad, Jamal Alzubi, Manuel Rhiel, Kay O Chmielewski, Laura Mosti, Julia Rositzka, Marcel Heugel, Jan Lawrenz, Valentina Pennucci, Birgitta Gläser and 8 more

Open access · goldAbstract read
In one paragraph

Article in International journal of molecular sciences, 2024. 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
0.9field-weighted citation impact, top 28% 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

2 citing papers in PubMed, 4 citations in OpenAlex.

  1. Article
  2. Article
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

18 authors at 2 institutions in 1 country.

Ruba HammadInstitute for Transfusion Medicine and Gene Therapy, Medical Center-University of Freiburg, 79106 Freiburg, Germany.
Jamal AlzubiInstitute for Transfusion Medicine and Gene Therapy, Medical Center-University of Freiburg, 79106 Freiburg, Germany.ORCID 0000-0003-4284-2006
Manuel RhielInstitute for Transfusion Medicine and Gene Therapy, Medical Center-University of Freiburg, 79106 Freiburg, Germany.ORCID 0000-0003-0741-2780
Kay O ChmielewskiInstitute for Transfusion Medicine and Gene Therapy, Medical Center-University of Freiburg, 79106 Freiburg, Germany.
Laura MostiInstitute for Transfusion Medicine and Gene Therapy, Medical Center-University of Freiburg, 79106 Freiburg, Germany.ORCID 0000-0001-8877-8359
Julia RositzkaInstitute for Transfusion Medicine and Gene Therapy, Medical Center-University of Freiburg, 79106 Freiburg, Germany.
Marcel HeugelInstitute for Transfusion Medicine and Gene Therapy, Medical Center-University of Freiburg, 79106 Freiburg, Germany.
Jan LawrenzInstitute for Transfusion Medicine and Gene Therapy, Medical Center-University of Freiburg, 79106 Freiburg, Germany.ORCID 0000-0003-3767-7130
Valentina PennucciInstitute for Transfusion Medicine and Gene Therapy, Medical Center-University of Freiburg, 79106 Freiburg, Germany.
Birgitta GläserInstitute of Human Genetics, Medical Center-University of Freiburg, 79106 Freiburg, Germany.ORCID 0000-0002-5792-7910
Judith FischerInstitute of Human Genetics, Medical Center-University of Freiburg, 79106 Freiburg, Germany.ORCID 0000-0002-8580-8118
Axel SchambachInstitute for Experimental Hematology, Hannover Medical School, 30625 Hannover, Germany.
Thomas MoritzInstitute for Experimental Hematology, Hannover Medical School, 30625 Hannover, Germany.
Nico LachmannREBIRTH Center for Regenerative and Translational Medicine, Hannover Medical School, 30625 Hannover, Germany.ORCID 0000-0002-4245-1497
Tatjana I CornuInstitute for Transfusion Medicine and Gene Therapy, Medical Center-University of Freiburg, 79106 Freiburg, Germany.ORCID 0000-0002-7206-7541
Claudio MussolinoInstitute for Transfusion Medicine and Gene Therapy, Medical Center-University of Freiburg, 79106 Freiburg, Germany.
Richard SchäferInstitute for Transfusion Medicine and Gene Therapy, Medical Center-University of Freiburg, 79106 Freiburg, Germany.
Toni CathomenInstitute for Transfusion Medicine and Gene Therapy, Medical Center-University of Freiburg, 79106 Freiburg, Germany.ORCID 0000-0002-7757-4630
University of Freiburg · DEMedizinische Hochschule Hannover · DE

Funding

Federal Ministry of Education and Research 01EK1602 (iMACnet)freiburg Open Access Publication Fund
6 · The paper itself

Abstract

The CRISPR-Cas12a platform has attracted interest in the genome editing community because the prototypical Acidaminococcus Cas12a generates a staggered DNA double-strand break upon binding to an AT-rich protospacer-adjacent motif (PAM, 5'-TTTV). The broad application of the platform in primary human cells was enabled by the development of an engineered version of the natural Cas12a protein, called Cas12a Ultra. In this study, we confirmed that CRISPR-Cas12a Ultra ribonucleoprotein complexes enabled allelic gene disruption frequencies of over 90% at multiple target sites in human T cells, hematopoietic stem and progenitor cells (HSPCs), and induced pluripotent stem cells (iPSCs). In addition, we demonstrated, for the first time, the efficient knock-in potential of the platform in human iPSCs and achieved targeted integration of a

Indexed as

Induced Pluripotent Stem CellsPluripotent Stem CellsAllelesCRISPR-Cas SystemsHematopoietic Stem CellsHumansAAVCas12aCpf1Cpf1 UltraCRISPRgenome editingHSCHSPCiPSCT cell

Identifiers

PMID38256061
PMCPMC10816062
OpenAlexW4390788591

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.