Correction of Disease Phenotype in Pompe Disease Knockout Mice Following Cationic Lipid-GL-67-Mediated Gene Therapy

These data suggest that cationic lipid mediated gene delivery of the human <i>GAA</i> cDNA may be an effective treatment for PD.

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Publication Details

Journal
DNA and cell biology reports.
Published
2025-04-01
DOI
https://doi.org/10.1089/dcbr.2025.0025
Primary Topic
RNA Interference and Gene Delivery
Type
article
Field-Weighted Citation Impact
0.00
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article

Correction of Disease Phenotype in Pompe Disease Knockout Mice Following Cationic Lipid-GL-67-Mediated Gene Therapy

Adra Mack, Justin Martiniuk, Frank Martiniuk, Seng H. Cheng
DNA and cell biology reports.
RNA Interference and Gene Delivery
article

Correction of Disease Phenotype in Pompe Disease Knockout Mice Following Cationic Lipid-GL-67-Mediated Gene Therapy

Adra Mack, Justin Martiniuk, Frank Martiniuk, Seng H. Cheng
article en

Abstract

These data suggest that cationic lipid mediated gene delivery of the human <i>GAA</i> cDNA may be an effective treatment for PD.

DNA and cell biology reports.Vol. 6(1)
Psychogenics (United States) (US), Automatic Data Processing (United States) (US), Alexion Pharmaceuticals (United States) (US), AstraZeneca (United States) (US)
Openalex Percentile: Top 91%
RNA Interference and Gene Delivery
DOIOpen Access PDFOpenAlex
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