ArticleMolecular therapy. Methods & clinical development2017
Lentivector Iterations and Pre-Clinical Scale-Up/Toxicity Testing: Targeting Mobilized CD34
Article in Molecular therapy. Methods & clinical development, 2017. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Cited by 26 papers.
What it found
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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
26 citing papers in PubMed, 46 citations in OpenAlex.
- Reduced-intensity conditioning in LV-mediated gene therapy for Fabry disease targeting HSPCs.Molecular therapy : the journal of the American Society of Gene Therapy · 2025Article
- Lentivirus-mediated gene therapy for Fabry disease: 5-year End-of-Study results from the Canadian FACTs trial.Clinical and translational medicine · 2025Article
- Development of anMolecular therapy. Methods & clinical development · 2023Article
- Review
- Dysregulated DNA methylation in the pathogenesis of Fabry disease.Molecular genetics and metabolism reports · 2022Article
- Autologous, lentivirus-modified, T-rapa cell "micropharmacies" for lysosomal storage disorders.EMBO molecular medicine · 2022Article
- Promoter considerations in the design of lentiviral vectors for use in treating lysosomal storage diseases.Molecular therapy. Methods & clinical development · 2022Review
- Cholesterol - the devil you know; ceramide - the devil you don't.Trends in pharmacological sciences · 2021Review
- Predicting genotoxicity of viral vectors for stem cell gene therapy using gene expression-based machine learning.Molecular therapy : the journal of the American Society of Gene Therapy · 2021Article
- Retroviral gene therapy in Germany with a view on previous experience and future perspectives.Gene therapy · 2021Review
- Fabry Cardiomyopathy: Current Treatment and Future Options.Journal of clinical medicine · 2021Review
- Fabry disease: what the cardiologist should consider in non-cardiac screening, diagnosis, and management-narrative review.Cardiovascular diagnosis and therapy · 2021Review
- Systemic Treatment of Fabry Disease Using a Novel AAV9 Vector Expressing α-Galactosidase A.Molecular therapy. Methods & clinical development · 2021Article
- Extracellular vesicles from recombinant cell factories improve the activity and efficacy of enzymes defective in lysosomal storage disorders.Journal of extracellular vesicles · 2021Article
- Lentivirus-mediated gene therapy for Fabry disease.Nature communications · 2021Article
- Current Clinical Applications of In Vivo Gene Therapy with AAVs.Molecular therapy : the journal of the American Society of Gene Therapy · 2021Review
- Assessing the role of glycosphingolipids in the phenotype severity of Fabry disease mouse model.Journal of lipid research · 2020Article
- Therapeutic challenges in two adolescent male patients with Fabry disease and high antibody titres.Molecular genetics and metabolism reports · 2020Article
- Current and Investigational Therapeutics for Fabry Disease.Kidney international reports · 2020Review
- Genetic ablation of acid ceramidase in Krabbe disease confirms the psychosine hypothesis and identifies a new therapeutic target.Proceedings of the National Academy of Sciences of the United States of America · 2019Article
Corrections and comments
PubMed lists nothing against this paper. Absence here is not a guarantee, only a check that was made.
Authors and funding
22 authors at 12 institutions in 3 countries.
Funding
No grant is acknowledged in the PubMed record.
Abstract
Fabry disease is a rare lysosomal storage disorder (LSD). We designed multiple recombinant lentivirus vectors (LVs) and tested their ability to engineer expression of human α-galactosidase A (α-gal A) in transduced Fabry patient CD34
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.