Evidence map›Paper›PMID 32729022›Full record

ArticleProtein & cell2020

Generation of a Hutchinson-Gilford progeria syndrome monkey model by base editing.

Fang Wang, Weiqi Zhang, Qiaoyan Yang, Yu Kang, Yanling Fan, Jingkuan Wei, Zunpeng Liu, Shaoxing Dai, Hao Li, Zifan Li and 8 more

Open access · diamondAbstract read
In one paragraph

Article in Protein & cell, 2020. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Cited by 52 papers.

0numbers the graph read from it
0cells of the map it votes in
52citing papers in PubMed
5.3field-weighted citation impact, top 3% 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

52 citing papers in PubMed, 92 citations in OpenAlex.

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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 6 institutions in 2 countries.

Fang WangYunnan Key Laboratory of Primate Biomedical Research, Institute of Primate Translational Medicine, Kunming University of Science and Technology, Kunming, 650500, China.
Weiqi ZhangInstitute for Stem Cell and Regeneration, Chinese Academy of Science, Beijing, 100101, China.
Qiaoyan YangThe Leon H Charney Division of Cardiology, New York University School of Medicine, New York, NY, 10016, USA.
Yu KangYunnan Key Laboratory of Primate Biomedical Research, Institute of Primate Translational Medicine, Kunming University of Science and Technology, Kunming, 650500, China.
Yanling FanCAS Key Laboratory of Genomic and Precision Medicine, Beijing Institute of Genomics, Chinese Academy of Sciences, Beijing, 100101, China.
Jingkuan WeiYunnan Key Laboratory of Primate Biomedical Research, Institute of Primate Translational Medicine, Kunming University of Science and Technology, Kunming, 650500, China.
Zunpeng LiuUniversity of Chinese Academy of Sciences, Beijing, 100049, China.
Shaoxing DaiYunnan Key Laboratory of Primate Biomedical Research, Institute of Primate Translational Medicine, Kunming University of Science and Technology, Kunming, 650500, China.
Hao LiCAS Key Laboratory of Genomic and Precision Medicine, Beijing Institute of Genomics, Chinese Academy of Sciences, Beijing, 100101, China.
Zifan LiYunnan Key Laboratory of Primate Biomedical Research, Institute of Primate Translational Medicine, Kunming University of Science and Technology, Kunming, 650500, China.
Lizhu XuYunnan Key Laboratory of Primate Biomedical Research, Institute of Primate Translational Medicine, Kunming University of Science and Technology, Kunming, 650500, China.
Chu ChuYunnan Key Laboratory of Primate Biomedical Research, Institute of Primate Translational Medicine, Kunming University of Science and Technology, Kunming, 650500, China.
Jing QuInstitute for Stem Cell and Regeneration, Chinese Academy of Science, Beijing, 100101, China.
Chenyang SiYunnan Key Laboratory of Primate Biomedical Research, Institute of Primate Translational Medicine, Kunming University of Science and Technology, Kunming, 650500, China.
Weizhi JiYunnan Key Laboratory of Primate Biomedical Research, Institute of Primate Translational Medicine, Kunming University of Science and Technology, Kunming, 650500, China. wji@lpbr.cn.
Guang-Hui LiuInstitute for Stem Cell and Regeneration, Chinese Academy of Science, Beijing, 100101, China. ghliu@ioz.ac.cn.
Chengzu LongThe Leon H Charney Division of Cardiology, New York University School of Medicine, New York, NY, 10016, USA. Chengzu.long@nyulangone.org.
Yuyu NiuYunnan Key Laboratory of Primate Biomedical Research, Institute of Primate Translational Medicine, Kunming University of Science and Technology, Kunming, 650500, China. niuyy@lpbr.cn.
Kunming University of Science and Technology · CNNew York University · USShanghai Center For Bioinformation Technology · CNBeijing Institute of Genomics · CNChinese Academy of Sciences · CNInstitute of Zoology · CN

Funding

No grant is acknowledged in the PubMed record.

6 · The paper itself

Abstract

Many human genetic diseases, including Hutchinson-Gilford progeria syndrome (HGPS), are caused by single point mutations. HGPS is a rare disorder that causes premature aging and is usually caused by a de novo point mutation in the LMNA gene. Base editors (BEs) composed of a cytidine deaminase fused to CRISPR/Cas9 nickase are highly efficient at inducing C to T base conversions in a programmable manner and can be used to generate animal disease models with single amino-acid substitutions. Here, we generated the first HGPS monkey model by delivering a BE mRNA and guide RNA (gRNA) targeting the LMNA gene via microinjection into monkey zygotes. Five out of six newborn monkeys carried the mutation specifically at the target site. HGPS monkeys expressed the toxic form of lamin A, progerin, and recapitulated the typical HGPS phenotypes including growth retardation, bone alterations, and vascular abnormalities. Thus, this monkey model genetically and clinically mimics HGPS in humans, demonstrating that the BE system can efficiently and accurately generate patient-specific disease models in non-human primates.

Indexed as

Disease Models, AnimalGene EditingLamin Type AProgeriaAnimalsFemaleHumansMacaca fascicularisLamin Type Abase editingHGPSnon-human primate

Identifiers

PMID32729022
PMCPMC7647984
OpenAlexW3045582016

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.