ReviewCellular immunology2019
Immunomodulatory, liver depot gene therapy for Pompe disease.
Review in Cellular immunology, 2019. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Cited by 19 papers.
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.
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.
Who cites it
19 citing papers in PubMed, 21 citations in OpenAlex.
- Collagen Type II-Targeting Lentiviral Gene Therapy for Mucopolysaccharidosis IVA.Current issues in molecular biology · 2025Article
- Correction of Disease Phenotype in Pompe Disease Knockout Mice Following Cationic Lipid-GL-67-Mediated Gene Therapy.DNA and cell biology reports · 2025Article
- Latest Advancements in Treatment Options for Infantile-Onset Pompe Disease: A Comprehensive Narrative Review.Clinical medicine insights. Pediatrics · 2025Review
- Gene therapy for glycogen storage diseases.Journal of inherited metabolic disease · 2024Review
- Screening data from 19 patients with late-onset Pompe disease for a phase I clinical trial of AAV8 vector-mediated gene therapy.JIMD reports · 2023Article
- Phase I study of liver depot gene therapy in late-onset Pompe disease.Molecular therapy : the journal of the American Society of Gene Therapy · 2023Article
- Lentiviral gene therapy prevents anti-human acid α-glucosidase antibody formation in murine Pompe disease.Molecular therapy. Methods & clinical development · 2022Article
- Preclinical Research in Glycogen Storage Diseases: A Comprehensive Review of Current Animal Models.International journal of molecular sciences · 2020Review
- Design of efficacious somatic cell genome editing strategies for recessive and polygenic diseases.Nature communications · 2020Article
- Pompe Disease: New Developments in an Old Lysosomal Storage Disorder.Biomolecules · 2020Review
- Molecular Approaches for the Treatment of Pompe Disease.Molecular neurobiology · 2020Review
- Gene therapy for glycogen storage diseases.Human molecular genetics · 2019Review
- Liver depot gene therapy for Pompe disease.Annals of translational medicine · 2019Review
- Progress and challenges of gene therapy for Pompe disease.Annals of translational medicine · 2019Review
- Immunological challenges and approaches to immunomodulation in Pompe disease: a literature review.Annals of translational medicine · 2019Review
- Pompe Disease: From Basic Science to Therapy.Neurotherapeutics : the journal of the American Society for Experimental NeuroTherapeutics · 2018Review
- Therapeutic Benefit of Autophagy Modulation in Pompe Disease.Molecular therapy : the journal of the American Society of Gene Therapy · 2018Article
- The impact of Pompe disease on smooth muscle: a review.Journal of smooth muscle research = Nihon Heikatsukin Gakkai kikanshi · 2018Review
- Neonatal gene therapy with AAV2/8-LSPhGAA improves hypertrophic cardiomyopathy in the GaaMolecular genetics and metabolismArticle
Corrections and comments
PubMed lists nothing against this paper. Absence here is not a guarantee, only a check that was made.
Authors and funding
3 authors at 1 institution in 1 country.
Funding
Abstract
Pompe disease is caused by mutations in acid alpha glucosidase (GAA) that causes accumulation of lysosomal glycogen affecting the heart and skeletal muscles, and can be fatal. Enzyme replacement therapy (ERT) with recombinant human GAA (rhGAA) improves muscle function by reducing glycogen accumulation. Limitations of ERT include a short half-life and the formation of antibodies that result in reduced efficacy. By harnessing the immune tolerance induction properties of the liver, liver-targeted gene delivery (with an adeno-associated virus vector containing a liver specific promoter), suppresses immunity against the GAA introduced by gene therapy. This induces immune tolerance to rhGAA by activating regulatory T cells and simultaneously, corrects GAA deficiency. Potentially, liver-targeted gene therapy can be performed once with lasting effects, by administering a relatively low dose of an adeno-associated virus type 8 vector to replace and induce immune tolerance to GAA.
Indexed as
Identifiers
What OpenQuestion holds
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.