Evidence map›Paper›PMID 32039406›Full record

ArticleJHEP reports : innovation in hepatology2020

Efficient

Samantha L Ginn, Anais K Amaya, Sophia H Y Liao, Erhua Zhu, Sharon C Cunningham, Michael Lee, Claus V Hallwirth, Grant J Logan, Szun S Tay, Anthony J Cesare and 5 more

Open access · goldAbstract read
PubMed Full text at PMC Publisher
In one paragraph

Article in JHEP reports : innovation in hepatology, 2020. 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
1.2field-weighted citation impact, top 21% 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, 31 citations in OpenAlex.

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  18. Modelling urea cycle disorders using iPSCs.NPJ Regenerative medicine · 2022
    Review
  19. Review
  20. Novel vectors and approaches for gene therapy in liver diseases.JHEP reports : innovation in hepatology · 2021
    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

15 authors at 2 institutions in 3 countries.

Samantha L GinnGene Therapy Research Unit, Children's Medical Research Institute, Faculty of Medicine and Health, The University of Sydney and Sydney Children's Hospitals Network, Westmead, Australia.
Anais K AmayaGene Therapy Research Unit, Children's Medical Research Institute, Faculty of Medicine and Health, The University of Sydney and Sydney Children's Hospitals Network, Westmead, Australia.
Sophia H Y LiaoGene Therapy Research Unit, Children's Medical Research Institute, Faculty of Medicine and Health, The University of Sydney and Sydney Children's Hospitals Network, Westmead, Australia.
Erhua ZhuGene Therapy Research Unit, Children's Medical Research Institute, Faculty of Medicine and Health, The University of Sydney and Sydney Children's Hospitals Network, Westmead, Australia.
Sharon C CunninghamGene Therapy Research Unit, Children's Medical Research Institute, Faculty of Medicine and Health, The University of Sydney and Sydney Children's Hospitals Network, Westmead, Australia.
Michael LeeTelomere Length Regulation Group, Children's Medical Research Institute, Faculty of Medicine and Health, The University of Sydney, Westmead, Australia.
Claus V HallwirthGene Therapy Research Unit, Children's Medical Research Institute, Faculty of Medicine and Health, The University of Sydney and Sydney Children's Hospitals Network, Westmead, Australia.
Grant J LoganGene Therapy Research Unit, Children's Medical Research Institute, Faculty of Medicine and Health, The University of Sydney and Sydney Children's Hospitals Network, Westmead, Australia.
Szun S TayGene Therapy Research Unit, Children's Medical Research Institute, Faculty of Medicine and Health, The University of Sydney and Sydney Children's Hospitals Network, Westmead, Australia.
Anthony J CesareGenome Integrity Unit, Children's Medical Research Institute, Faculty of Medicine and Health, The University of Sydney, Westmead, Australia.
Hilda A PickettTelomere Length Regulation Group, Children's Medical Research Institute, Faculty of Medicine and Health, The University of Sydney, Westmead, Australia.
Markus GrompeSchool of Medicine, Oregon Health & Science University, Portland, Oregon.
Kimberley DilworthTranslational Vectorology Group and Vector & Genome Engineering Facility, Children's Medical Research Institute, Faculty of Medicine and Health, The University of Sydney, Westmead, Australia.
Leszek LisowskiTranslational Vectorology Group and Vector & Genome Engineering Facility, Children's Medical Research Institute, Faculty of Medicine and Health, The University of Sydney, Westmead, Australia.
Ian E AlexanderGene Therapy Research Unit, Children's Medical Research Institute, Faculty of Medicine and Health, The University of Sydney and Sydney Children's Hospitals Network, Westmead, Australia.
The University of Sydney · AUOregon Health & Science University · US

Funding

No grant is acknowledged in the PubMed record.

6 · The paper itself

Abstract

BACKGROUND &

aimsGenome editing technology has immense therapeutic potential and is likely to rapidly supplant contemporary gene addition approaches. Key advantages include the capacity to directly repair mutant loci with resultant recovery of physiological gene expression and maintenance of durable therapeutic effects in replicating cells. In this study, we aimed to repair a disease-causing point mutation in the ornithine transcarbamylase (

methodsEditing reagents for precise CRISPR/SaCas9-mediated cleavage and homology-directed repair (HDR) of the human

resultsFollowing AAV-mediated delivery of editing reagents to patient-derived primary human hepatocytes

conclusionsWe report efficient single-nucleotide correction of a disease-causing mutation in the LAY SUMMARY: The ability to efficiently and safely correct disease-causing mutations remains the holy grail of gene therapy. Herein, we demonstrate, for the first time, efficient

Indexed as

7 NGS, next-generation sequencingAAV, adeno-associated virusBrdU, bromodeoxyuridineCRISPR-Cas9FRG, Fah-/-Rag2-/-Il2rg-/-genome editingHDR, homology-directed repairhomology-directed repairhumanised FRG miceInDels, insertions and deletionsITR, inverted terminal repeatsLSP1, liver-specific promoterNHEJ, non-homologous end joiningNP59 capsidOTC deficiencypA, bovine growth hormone polyadenylation signal sequencePAM, protospacer adjacent motifPRE, mutant form of the Woodchuck hepatitis virus posttranscriptional regulatory elementprimary human hepatocytesrAAV, recombinant adeno-associated virusrecombinant AAVRTA, Real Time AnalysisSaCas9, Staphylococcus aureus Cas9 nucleasesgRNA, single guide RNASV40 pA, SV40 polyadenylation signal sequencesynthetic capsidTBG, human thyroxine binding globulin promoterU6, RNA polymerase III promoter for human U6 snRNAWT, wild-type

Identifiers

PMID32039406
PMCPMC7005564
OpenAlexW2998525228

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.