Evidence map›Paper›PMID 36401038›Full record

ArticleMethods in molecular biology (Clifton, N.J.)2023

Assessment of the Gene Therapy Immune Response in the Canine Muscular Dystrophy Model.

Chady H Hakim, Sandeep R P Kumar, Dennis Pérez-López, James Teixeira, Roland W Herzog, Dongsheng Duan

Abstract read
PubMed Publisher
In one paragraph

Article in Methods in molecular biology (Clifton, N.J.), 2023. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Cited by 4 papers.

0numbers the graph read from it
0cells of the map it votes in
4citing papers in PubMed
6.5field-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

4 citing papers in PubMed, 6 citations in OpenAlex.

  1. Canine Model of Duchenne Muscular Dystrophy.Methods in molecular biology (Clifton, N.J.) · 2026
    Article
  2. Article
  3. Article
  4. 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

6 authors at 2 institutions in 1 country.

Chady H HakimDepartment of Molecular Microbiology and Immunology, School of Medicine, The University of Missouri, Columbia, MO, USA.
Sandeep R P KumarDepartment of Pediatrics, Indiana University, Indianapolis, IN, USA.
Dennis Pérez-LópezDepartment of Molecular Microbiology and Immunology, School of Medicine, The University of Missouri, Columbia, MO, USA.
James TeixeiraDepartment of Molecular Microbiology and Immunology, School of Medicine, The University of Missouri, Columbia, MO, USA.
Roland W HerzogDepartment of Pediatrics, Indiana University, Indianapolis, IN, USA.
Dongsheng DuanDepartment of Molecular Microbiology and Immunology, School of Medicine, The University of Missouri, Columbia, MO, USA. duand@missouri.edu.
University of Missouri · USIndiana University – Purdue University Indianapolis · US

Funding

Whole body single AAV microgene therapy in canine DMDR01NS090634 · NINDS · UNIVERSITY OF MISSOURI-COLUMBIA · PI Dongsheng Duan · 2015 to 2026
$4.2M
Treatment of Duchenne Muscular Dystrophy with the Muscle Calcium PumpR01AR070517 · NIAMS · UNIVERSITY OF MISSOURI-COLUMBIA · PI DUAN, DONGSHENG · 2016 to 2020
$3.1M
NIAMS NIH HHS R01 AR070517NINDS NIH HHS R01 NS090634
6 · The paper itself

Abstract

The immune response is a primary hurdle in the development of gene therapy for neuromuscular diseases. Both innate and adaptive immune responses have been observed in human trials. The canine model is an excellent platform to understand immunological consequences of gene therapy. Over the last several decades, we have conducted gene replacement and gene repair therapies in the canine model of Duchenne muscular dystrophy (DMD) using adeno-associated virus (AAV)-mediated expression of the highly abbreviated micro-dystrophin gene, the larger mini-dystrophin gene, and the Cas9-based CRISPR genome editing machinery. We have evaluated the innate, humoral, and cellular immune responses to the AAV vector and the transgene product. In this chapter, we share our experience in collecting and processing of the dog blood samples for immunological assays, and our protocols for quantitative evaluation of cytokines and chemokines, antibodies, and T-cell responses.

Indexed as

DystrophinMuscular Dystrophy, DuchenneAnimalsDogsGenetic TherapyGenetic VectorsHumansImmunity, HumoralDystrophinBloodCanine modelDMDDuchenne muscular dystrophyELISAELISpotLuminexPBMCsPlasmaSerum

Identifiers

PMID36401038
OpenAlexW4309329467

What OpenQuestion holds

Textmetadata
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.