Evidence map›Paper›PMID 37075755›Full record

ArticleCell2023

Enveloped viruses pseudotyped with mammalian myogenic cell fusogens target skeletal muscle for gene delivery.

Sajedah M Hindi, Michael J Petrany, Elena Greenfeld, Leah C Focke, Alyssa A W Cramer, Michael A Whitt, Ramzi J Khairallah, Christopher W Ward, Jeffrey S Chamberlain, Benjamin Podbilewicz and 2 more

Erratum issuedOpen access · greenAbstract read
In one paragraph

Article in Cell, 2023. The graph could read no effect estimate from its abstract, so it casts no vote on the map. An erratum has been issued. Cited by 26 papers.

0numbers the graph read from it
0cells of the map it votes in
26citing papers in PubMed
10.0field-weighted citation impact, top 2% 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

26 citing papers in PubMed, 32 citations in OpenAlex.

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  17. The road toward AAV-mediated gene therapy of Duchenne muscular dystrophy.Molecular therapy : the journal of the American Society of Gene Therapy · 2025
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4 · The record

Corrections and comments

5 · Who and what money

Authors and funding

12 authors at 5 institutions in 2 countries.

Sajedah M HindiDivision of Molecular Cardiovascular Biology, Cincinnati Children's Hospital Medical Center, Cincinnati, OH, USA.
Michael J PetranyDivision of Molecular Cardiovascular Biology, Cincinnati Children's Hospital Medical Center, Cincinnati, OH, USA.
Elena GreenfeldDepartment of Biology, Technion-Israel Institute of Technology, Haifa, Israel.
Leah C FockeDivision of Molecular Cardiovascular Biology, Cincinnati Children's Hospital Medical Center, Cincinnati, OH, USA.
Alyssa A W CramerDivision of Molecular Cardiovascular Biology, Cincinnati Children's Hospital Medical Center, Cincinnati, OH, USA.
Michael A WhittDepartment of Microbiology, Immunology, and Biochemistry, University of Tennessee Health Science Center, Memphis, TN, USA.
Ramzi J KhairallahMyologica, LLC, New Market, MD, USA.
Christopher W WardDepartment of Orthopedics and Center for Biomedical Engineering and Technology (BioMET), University of Maryland School of Medicine, Baltimore, MD, USA.
Jeffrey S ChamberlainDepartments of Neurology, Medicine and Biochemistry, Wellstone Muscular Dystrophy Specialized Research Center, University of Washington School of Medicine, Seattle, WA, USA.
Benjamin PodbilewiczDepartment of Biology, Technion-Israel Institute of Technology, Haifa, Israel.
Vikram PrasadDivision of Molecular Cardiovascular Biology, Cincinnati Children's Hospital Medical Center, Cincinnati, OH, USA.
Douglas P MillayDivision of Molecular Cardiovascular Biology, Cincinnati Children's Hospital Medical Center, Cincinnati, OH, USA; Department of Pediatrics, University of Cincinnati College of Medicine, Cincinnati, OH, USA. Electronic address: douglas.millay@cchmc.org.
Cincinnati Children's Hospital Medical Center · USTechnion – Israel Institute of Technology · ILUniversity of Maryland, Baltimore · USUniversity of Tennessee Health Science Center · USUniversity of Washington · US

Funding

Wellstone Muscular Dystrophy Specialized Research Center (Seattle)P50AR065139 · NIAMS · UNIVERSITY OF WASHINGTON · PI JEFFREY S CHAMBERLAIN · 2018 to 2026
$15.9M
DYSTROPHIN REPLACEMENT IN MDX MICER01AR040864 · NIAMS · UNIVERSITY OF WASHINGTON · PI JEFFREY S CHAMBERLAIN · 1991 to 2026
$8.8M
Deciphering mechanisms of myoblast fusionR01AR068286 · NIAMS · CINCINNATI CHILDRENS HOSP MED CTR · PI MILLAY, DOUGLAS PAUL · 2015 to 2024
$4.2M
Role of skeletal muscle stem cell fusion and fibrosis during agingR01AG059605 · NIA · CINCINNATI CHILDRENS HOSP MED CTR · PI MILLAY, DOUGLAS PAUL · 2018 to 2022
$1.7M
Improving delivery of therapeutic material to skeletal muscleR61AR076771 · NIAMS · CINCINNATI CHILDRENS HOSP MED CTR · PI MILLAY, DOUGLAS PAUL · 2019 to 2020
$795k
Improving delivery of therapeutic material to skeletal muscleR33AR076771 · NIAMS · CINCINNATI CHILDRENS HOSP MED CTR · PI MILLAY, DOUGLAS PAUL · 2022 to 2022
$397k
NIAMS NIH HHS P50 AR065139NIAMS NIH HHS R01 AR040864NIAMS NIH HHS R01 AR068286NIAMS NIH HHS R33 AR076771NIAMS NIH HHS R61 AR076771NIA NIH HHS R01 AG059605
6 · The paper itself

Abstract

Entry of enveloped viruses into cells is mediated by viral fusogenic proteins that drive membrane rearrangements needed for fusion between viral and target membranes. Skeletal muscle development also requires membrane fusion events between progenitor cells to form multinucleated myofibers. Myomaker and Myomerger are muscle-specific cell fusogens but do not structurally or functionally resemble classical viral fusogens. We asked whether the muscle fusogens could functionally substitute for viral fusogens, despite their structural distinctiveness, and fuse viruses to cells. We report that engineering of Myomaker and Myomerger on the membrane of enveloped viruses leads to specific transduction of skeletal muscle. We also demonstrate that locally and systemically injected virions pseudotyped with the muscle fusogens can deliver μDystrophin to skeletal muscle of a mouse model of Duchenne muscular dystrophy and alleviate pathology. Through harnessing the intrinsic properties of myogenic membranes, we establish a platform for delivery of therapeutic material to skeletal muscle.

Indexed as

BioengineeringLentivirusMembrane ProteinsMuscle, SkeletalMuscular Dystrophy, DuchenneAnimalsCell FusionDisease Models, AnimalMembrane FusionMiceMuscle DevelopmentViral TropismMembrane Proteinscell fusiongene therapymuscular dystrophymyomakermyomerger/myomixerviral pseudotyping

Identifiers

PMID37075755
PMCPMC11181154
OpenAlexW4366347600

What OpenQuestion holds

Textmetadata
LicenceTDM
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.