ArticleDNA and cell biology reports2025
Correction of Disease Phenotype in Pompe Disease Knockout Mice Following Cationic Lipid-GL-67-Mediated Gene Therapy.
Article in DNA and cell biology reports, 2025. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Not yet cited in PubMed.
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
0 citing papers in PubMed.
No citing paper in PubMed yet.
Corrections and comments
PubMed lists nothing against this paper. Absence here is not a guarantee, only a check that was made.
Authors and funding
4 authors.
Funding
No grant is acknowledged in the PubMed record.
Abstract
Background: Genetic deficiency of lysosomal acid α-glucosidase or acid maltase (GAA) results in Pompe disease (PD) or glycogen storage disease type II, encompassing subtypes of varying severity. The infantile form presents as hypotonia, muscle weakness, and congestive heart failure in the first year, while the adult onset forms are limited to skeletal muscle. Gene therapy for diseases that affect muscles or organs (as PD) may be very difficult due to their size or location and our ability to transfect these organs with high efficiency. Methods: We evaluated a plasmid vector carrying the cytomegalovirus promoter linked to the human Results: A human PD fibroblast cell line was transiently transfected with increasing amounts of the cationic lipid GL-67 complexed to the human Conclusions: These data suggest that cationic lipid mediated gene delivery of the human
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.