Evidence map›Paper›PMID 39524974›Full record

ArticleMolecular therapy. Methods & clinical development2024

Single cell and TCR analysis of immune cells from AAV gene therapy-dosed Duchenne muscular dystrophy patients.

Michael R Emami, Mark A Brimble, Alejandro Espinoza, Jane Owens, Laurence O Whiteley, Sandra Casinghino, Thomas A Lanz, Philip K Farahat, Matteo Pellegrini, Courtney S Young and 5 more

Abstract read
PubMed Full text at PMC Publisher
In one paragraph

Article in Molecular therapy. Methods & clinical development, 2024. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Cited by 5 papers.

0numbers the graph read from it
0cells of the map it votes in
5citing papers in PubMed
–field-weighted citation impact
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

5 citing papers in PubMed.

  1. Review
  2. Review
  3. Article
  4. Review
  5. 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.

Michael R EmamiDepartment of Neurology, David Geffen School of Medicine, University of California, Los Angeles, Los Angeles, CA 90095, USA.
Mark A BrimbleDepartment of Host-Microbe Interactions, St Jude Children's Research Hospital, Memphis, TN 38105, USA.
Alejandro EspinozaDepartment of Human Genetics, University of California, Los Angeles, Los Angeles, CA 90095, USA.
Jane OwensPfizer Inc., Rare Disease Research Unit, Cambridge, MA 02139, USA.
Laurence O WhiteleyPfizer Inc., Drug Safety Research & Development, Cambridge, MA 02139, USA.
Sandra CasinghinoPfizer Inc., Drug Safety Research & Development, Groton, CT 06340, USA.
Thomas A LanzPfizer Inc., Drug Safety Research & Development, Groton, CT 06340, USA.
Philip K FarahatDepartment of Physiology and Biophysics, University of California, Irvine, Irvine, CA 92697, USA.
Matteo PellegriniDepartment of Molecular, Cell and Developmental Biology, and the Institute for Quantitative and Computational Biosciences - The Collaboratory, University of California, Los Angeles, Los Angeles, CA 90095, USA.
Courtney S YoungMyoGene Bio, San Diego, CA 92121, USA.
Paul G ThomasDepartment of Host-Microbe Interactions, St Jude Children's Research Hospital, Memphis, TN 38105, USA.
Elizabeth M McNallyCenter for Genetic Medicine, Northwestern University Feinberg School of Medicine, Chicago, IL 60611, USA.
S Armando VillaltaDepartment of Physiology and Biophysics, University of California, Irvine, Irvine, CA 92697, USA.
Stefan A SchattgenDepartment of Host-Microbe Interactions, St Jude Children's Research Hospital, Memphis, TN 38105, USA.
Melissa J SpencerDepartment of Neurology, David Geffen School of Medicine, University of California, Los Angeles, Los Angeles, CA 90095, USA.

Funding

SARCOGLYCAN IN MYOPATHY AND MUSCLE MEMBRANE STABILITYR01HL061322 · NHLBI · UNIVERSITY OF CHICAGO · PI MCNALLY, ELIZABETH M · 1999 to 2022
$7.5M
Myoferlin in Muscle Membrane Fusion and RepairR01NS047726 · NINDS · UNIVERSITY OF CHICAGO · PI ALEXIS R. DEMONBREUN, Elizabeth M McNally · 2003 to 2026
$7.2M
DECODING THE INTERACTIONS BETWEEN T CELL RECEPTORS AND PEPTIDE-MHCR01AI136514 · NIAID · ST. JUDE CHILDREN'S RESEARCH HOSPITAL · PI Paul G. Thomas · 2018 to 2026
$6.9M
Understanding and Improving Therapies for the Muscular Dystrophies through Noninvasive BiomarkersP50AR052646 · NIAMS · UNIVERSITY OF FLORIDA · PI JUDGE, ANDREW ROBERT · 2021 to 2024
$6.2M
DYNAMICS AND EVOLUTION OF IMMUNE RESPONSES TO INFLUENZA VIRUSESU01AI150747 · NIAID · EMORY UNIVERSITY · PI AHMED, RAFI, ANTIA, RUSTOM NOSHIR · 2020 to 2024
$5.9M
Optimizing and validation of gene therapy vectors to treat limb girdle muscular dystophyR01NS117912 · NINDS · UNIVERSITY OF CALIFORNIA LOS ANGELES · PI CHAMBERLAIN, JEFFREY S, SPENCER, MELISSA JAN · 2020 to 2024
$2.6M
Novel macrophage and regulatory T cell interactions that promote the pathogenesis of Duchenne muscular dystrophyR01NS120060 · NINDS · UNIVERSITY OF CALIFORNIA-IRVINE · PI VILLALTA, SERGIO ARMANDO · 2020 to 2024
$2.5M
Immunology Research Training GrantT32AI177324 · NIAID · UNIVERSITY OF CALIFORNIA-IRVINE · PI Eric Pearlman · 2023 to 2026
$735k
Integrating T cell receptor features with gene expression profiles to define T cell specificity and differentiationR21AI169085 · NIAID · FRED HUTCHINSON CANCER RESEARCH CENTER · PI BRADLEY, PHILIP · 2022 to 2023
$511k
Tolerization of dystrophin and adeno-associated virus immunityR21NS114918 · NINDS · UNIVERSITY OF CALIFORNIA-IRVINE · PI VILLALTA, SERGIO ARMANDO · 2020 to 2020
$431k
NHLBI NIH HHS R01 HL061322NIAID NIH HHS R01 AI136514NIAID NIH HHS R21 AI169085NIAID NIH HHS T32 AI177324NIAID NIH HHS U01 AI150747NIAMS NIH HHS P50 AR052646NINDS NIH HHS R01 NS047726NINDS NIH HHS R01 NS117912NINDS NIH HHS R01 NS120060NINDS NIH HHS R21 NS114918
6 · The paper itself

Abstract

Clinical trials for Duchenne muscular dystrophy (DMD) are assessing the therapeutic efficacy of systemically delivered adeno-associated virus (AAV) carrying a modified

Indexed as

AAVadeno-associated virusDuchennegene therapyimmune responsemuscular dystrophyneuromuscular disorderssingle-cell sequencingT cell receptor sequencing

Identifiers

PMID39524974
PMCPMC11546459

What OpenQuestion holds

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Registered trials

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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.