Evidence map›Paper›PMID 37658603›Full record

Trial reportMolecular therapy : the journal of the American Society of Gene Therapy2023

In vivo CRISPR gene editing in patients with herpetic stromal keratitis.

Anji Wei, Di Yin, Zimeng Zhai, Sikai Ling, Huangying Le, Lijia Tian, Jianjiang Xu, Soren R Paludan, Yujia Cai, Jiaxu Hong

Registry-linked trialOpen access · greenAbstract readClinical Trial
PubMed Full text at PMC Publisher
In one paragraph

Trial report in Molecular therapy : the journal of the American Society of Gene Therapy, 2023. The graph could read no effect estimate from its abstract, so it casts no vote on the map. It is linked to trial NCT04560790 (CRISPR/Cas9 mRNA Instantaneous Gene Editing Therapy Assisted Corneal Transplantation in the Treatment of Refractory Viral Keratitis), which is not on this map. Cited by 40 papers.

0numbers the graph read from it
0cells of the map it votes in
40citing papers in PubMed
9.9field-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.

NCT04560790 nacompletednot on this map

CRISPR/Cas9 mRNA Instantaneous Gene Editing Therapy Assisted Corneal Transplantation in the Treatment of Refractory Viral Keratitis

TypeinterventionalSponsorShanghai BDgene Co., Ltd.Ran2020 to 2022Enrolled3ConditionsViral Keratitis, Blindness Eye, Herpes Simplex Virus Infection, CorneaArmsBD111 Adult single group Dose
3 · Its place in the literature

Who cites it

40 citing papers in PubMed, 46 citations in OpenAlex.

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  9. Developments in genital herpes: progress in prevention and treatment.Current opinion in infectious diseases · 2026
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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

10 authors at 3 institutions in 2 countries.

Anji WeiDepartment of Ophthalmology and Visual Science, Eye, and ENT Hospital, Shanghai Medical College, Fudan University, Shanghai, China.
Di YinKey Laboratory of Systems Biomedicine (Ministry of Education), Shanghai Center for Systems Biomedicine, Shanghai Jiao Tong University, Shanghai, China.
Zimeng ZhaiDepartment of Ophthalmology and Visual Science, Eye, and ENT Hospital, Shanghai Medical College, Fudan University, Shanghai, China.
Sikai LingBDgene Therapeutics, Shanghai, China.
Huangying LeKey Laboratory of Systems Biomedicine (Ministry of Education), Shanghai Center for Systems Biomedicine, Shanghai Jiao Tong University, Shanghai, China.
Lijia TianDepartment of Ophthalmology and Visual Science, Eye, and ENT Hospital, Shanghai Medical College, Fudan University, Shanghai, China.
Jianjiang XuDepartment of Ophthalmology and Visual Science, Eye, and ENT Hospital, Shanghai Medical College, Fudan University, Shanghai, China.
Soren R PaludanDepartment of Biomedicine, Aarhus University, Aarhus, Denmark.
Yujia CaiKey Laboratory of Systems Biomedicine (Ministry of Education), Shanghai Center for Systems Biomedicine, Shanghai Jiao Tong University, Shanghai, China. Electronic address: yujia.cai@sjtu.edu.cn.
Jiaxu HongDepartment of Ophthalmology and Visual Science, Eye, and ENT Hospital, Shanghai Medical College, Fudan University, Shanghai, China; Shanghai Key Laboratory of Visual Impairment and Restoration, Science and Technology Commission of Shanghai Municipality, Shanghai, China; Shanghai Engineering Research Center of Synthetic Immunology, Shanghai, China. Electronic address: Jiaxu.hong@fdeent.org.
Eye & ENT Hospital of Fudan University · CNShanghai Jiao Tong University · CNAarhus University · DK

Funding

No grant is acknowledged in the PubMed record.

6 · The paper itself

Abstract

In vivo CRISPR gene therapy holds large clinical potential, but the safety and efficacy remain largely unknown. Here, we injected a single dose of herpes simplex virus 1 (HSV-1)-targeting CRISPR formulation in the cornea of three patients with severe refractory herpetic stromal keratitis (HSK) during corneal transplantation. Our study is an investigator-initiated, open-label, single-arm, non-randomized interventional trial at a single center (NCT04560790). We found neither detectable CRISPR-induced off-target cleavages by GUIDE-seq nor systemic adverse events for 18 months on average in all three patients. The HSV-1 remained undetectable during the study. Our preliminary clinical results suggest that in vivo gene editing targeting the HSV-1 genome holds acceptable safety as a potential therapy for HSK.

Indexed as

Herpesvirus 1, HumanKeratitis, HerpeticClustered Regularly Interspaced Short Palindromic RepeatsCorneaGene EditingHumansCRISPR-Cas9herpes simplex virusherpetic stromal keratitisin vivo gene editinglentiviral particle

Identifiers

PMID37658603
PMCPMC10638052
OpenAlexW4386319765

What OpenQuestion holds

Textmetadata
Read underepoch 390

Registered trials

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.