Evidence map›Paper›PMID 29499946›Full record

ArticleMolecular therapy. Nucleic acids2018

Transcriptome Profiling of Neovascularized Corneas Reveals miR-204 as a Multi-target Biotherapy Deliverable by rAAVs.

Yi Lu, Phillip W L Tai, Jianzhong Ai, Dominic J Gessler, Qin Su, Xieyi Yao, Qiang Zheng, Phillip D Zamore, Xun Xu, Guangping Gao

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

Article in Molecular therapy. Nucleic acids, 2018. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Cited by 22 papers.

0numbers the graph read from it
0cells of the map it votes in
22citing papers in PubMed
1.5field-weighted citation impact, top 17% 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

22 citing papers in PubMed, 38 citations in OpenAlex.

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  16. Gene Therapy in the Anterior Eye Segment.Current gene therapy · 2022
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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 4 institutions in 2 countries.

Yi LuDepartment of Ophthalmology, Shanghai General Hospital, Shanghai Jiaotong University, Shanghai 200080, China; Shanghai Key Laboratory of Ocular Fundus Diseases, Shanghai 200080, China; Shanghai Engineering Center for Visual Science and Photomedicine, Shanghai 200080, China; Horae Gene Therapy Center, UMass Medical School, Worcester, MA 01605, USA.
Phillip W L TaiHorae Gene Therapy Center, UMass Medical School, Worcester, MA 01605, USA; Li Weibo Institute for Rare Diseases Research, UMass Medical School, Worcester, MA 01605, USA; Department of Microbiology and Physiological Systems, University of Massachusetts Medical School, Worcester, MA 01605, USA.
Jianzhong AiHorae Gene Therapy Center, UMass Medical School, Worcester, MA 01605, USA; Li Weibo Institute for Rare Diseases Research, UMass Medical School, Worcester, MA 01605, USA; Department of Microbiology and Physiological Systems, University of Massachusetts Medical School, Worcester, MA 01605, USA; Department of Urology, Institute for Urology, West China Hospital, Sichuan University, Chengdu, Sichuan 610041, China.
Dominic J GesslerHorae Gene Therapy Center, UMass Medical School, Worcester, MA 01605, USA; Li Weibo Institute for Rare Diseases Research, UMass Medical School, Worcester, MA 01605, USA; Department of Microbiology and Physiological Systems, University of Massachusetts Medical School, Worcester, MA 01605, USA.
Qin SuHorae Gene Therapy Center, UMass Medical School, Worcester, MA 01605, USA; Li Weibo Institute for Rare Diseases Research, UMass Medical School, Worcester, MA 01605, USA.
Xieyi YaoDepartment of Ophthalmology, Shanghai General Hospital, Shanghai Jiaotong University, Shanghai 200080, China; Shanghai Key Laboratory of Ocular Fundus Diseases, Shanghai 200080, China; Shanghai Engineering Center for Visual Science and Photomedicine, Shanghai 200080, China.
Qiang ZhengResearch and Development Center, Chengdu Kanghong Pharmaceuticals Group Co., Chengdu, Sichuan 610036, China.
Phillip D ZamoreRNA Therapeutics Institute, UMass Medical School, Worcester, MA 01605, USA.
Xun XuDepartment of Ophthalmology, Shanghai General Hospital, Shanghai Jiaotong University, Shanghai 200080, China; Shanghai Key Laboratory of Ocular Fundus Diseases, Shanghai 200080, China; Shanghai Engineering Center for Visual Science and Photomedicine, Shanghai 200080, China. Electronic address: drxuxun@sjtu.edu.cn.
Guangping GaoHorae Gene Therapy Center, UMass Medical School, Worcester, MA 01605, USA; Li Weibo Institute for Rare Diseases Research, UMass Medical School, Worcester, MA 01605, USA; Department of Microbiology and Physiological Systems, University of Massachusetts Medical School, Worcester, MA 01605, USA; Department of Urology, Institute for Urology, West China Hospital, Sichuan University, Chengdu, Sichuan 610041, China. Electronic address: guangping.gao@umassmed.edu.
University of Massachusetts Chan Medical School · USUMass Memorial Health Care · USShanghai First People's Hospital · CNShanghai Jiao Tong University · CN

Funding

Viral escape from AAV expressed transgenesP01AI100263 · NIAID · SCRIPPS FLORIDA · PI GAO, GUANGPING · 2012 to 2016
$11.9M
Viral Vector CoreP01HL131471 · NHLBI · UNIV OF MASSACHUSETTS MED SCH WORCESTER · PI XUE, WEN · 2016 to 2020
$11.3M
Oligodendrocyte-focused rAAV gene therapy strategies for Canavan disease and LeukodystrophiesR01NS076991 · NINDS · UNIV OF MASSACHUSETTS MED SCH WORCESTER · PI Guangping Gao · 2012 to 2026
$5.7M
Next Generation of Recombinant AAV Serotype Vectors for Gene TherapyR01HL097088 · NHLBI · UNIVERSITY OF FLORIDA · PI GAO, GUANGPING, HERZOG, ROLAND W. · 2010 to 2018
$5.4M
NHLBI NIH HHS P01 HL131471NHLBI NIH HHS R01 HL097088NIAID NIH HHS P01 AI100263NINDS NIH HHS R01 NS076991
6 · The paper itself

Abstract

Corneal neovascularization (NV) is the major sight-threatening pathology caused by angiogenic stimuli. Current drugs that directly target pro-angiogenic factors to inhibit or reverse the disease require multiple rounds of administration and have limited efficacies. Here, we identify potential anti-angiogenic corneal microRNAs (miRNAs) and demonstrate a framework that employs discovered miRNAs as biotherapies deliverable by recombinant adeno-associated viruses (rAAVs). By querying differentially expressed miRNAs in neovascularized mouse corneas induced by alkali burn, we have revealed 39 miRNAs that are predicted to target more than 5,500 differentially expressed corneal mRNAs. Among these, we selected miR-204 and assessed its efficacy and therapeutic benefit for treating injured corneas. Our results show that delivery of miR-204 by rAAV normalizes multiple novel target genes and biological pathways to attenuate vascularization of injured mouse cornea. Importantly, this gene therapy treatment alternative is efficacious and safe for mitigating corneal NV. Overall, our work demonstrates the discovery of potential therapeutic miRNAs in corneal disorders and their translation into viable treatment alternatives.

Indexed as

adeno-associated viruscorneal neovascularizationgene therapymicroRNAmiR-204

Identifiers

PMID29499946
PMCPMC5862543
OpenAlexW2777288088

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.