학술논문

Intravital imaging of muscle damage and response to therapy in a model of Pompe disease.
Document Type
Article
Source
Clinical & Translational Medicine. Mar2024, Vol. 14 Issue 3, p1-6. 6p.
Subject
*GLYCOGEN storage disease type II
*TREATMENT effectiveness
*ENZYME replacement therapy
*MOLECULAR biology
*GREEN fluorescent protein
Language
ISSN
2001-1326
Abstract
This article discusses the use of intravital imaging to study muscle damage and response to therapy in a mouse model of Pompe disease. Pompe disease is a lysosomal glycogen storage disorder that primarily affects muscle tissues. The researchers used a newly developed gene therapy approach and applied intravital microscopy to visualize and quantify the effectiveness of the treatment in live animals. They found that the gene therapy resulted in a reversal of pathology in multiple tissues, including the limb and tongue muscles. This study provides valuable insights into the disease progression and the potential of gene therapy as a treatment option for Pompe disease. [Extracted from the article]