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Title:           Treatment of infantile-onset Pompe disease in a rat model with muscle-directed AAV gene therapy
Subject:         Molecular Metabolism, 81 (2024) 101899. doi:10.1016/j.molmet.2024.101899
Author:          Sergio Muñoz
Creator:         Elsevier
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     Item: 
      Title: Treatment of infantile-onset Pompe disease in a rat model with muscle-directed AAV gene therapy
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      Children: 
       Item: 
        Title: 1. Introduction
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       Item: 
        Title: 2. Materials and methods
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        Children: 
         Item: 
          Title: 2.1. Animals
          Destination: slink1
         Item: 
          Title: 2.2. AAV vector production and administration
          Destination: slink2
         Item: 
          Title: 2.3. Sample collection
          Destination: slink3
         Item: 
          Title: 2.4. Gene expression analysis
          Destination: slink4
         Item: 
          Title: 2.5. Enzyme activities
          Destination: slink5
         Item: 
          Title: 2.6. Glycogen content quantification
          Destination: slink6
         Item: 
          Title: 2.7. Vector genome copy number
          Destination: slink7
         Item: 
          Title: 2.8. Western blot analyses
          Destination: slink8
         Item: 
          Title: 2.9. Histology and immunohistochemistry
          Destination: slink9
         Item: 
          Title: 2.10. Electron microscopy
          Destination: slink10
         Item: 
          Title: 2.11. Naso-anal length measurement
          Destination: slink11
         Item: 
          Title: 2.12. Four-limb grip strength test
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         Item: 
          Title: 2.13. Statistical analysis and data processing
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       Item: 
        Title: 3. Results
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        Children: 
         Item: 
          Title: 3.1. Generation and characterization of a Pompe rat model
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         Item: 
          Title: 3.2. Pompe rats develop early-onset skeletal muscle pathology
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         Item: 
          Title: 3.3. Pompe rats present severe cardiac alterations and hepatomegaly
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         Item: 
          Title: 3.4. Glycogen storage in CNS of Pompe rats
          Destination: slink17
         Item: 
          Title: 3.5. Systemic administration of a novel muscle-specific AAVMYO3-Gaa gene therapy reverted skeletal muscle pathology of Pompe rats
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         Item: 
          Title: 3.6. Treatment with AAVMYO3-Gaa normalized autophagy and lysosomal homeostasis
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       Item: 
        Title: 4. Discussion
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       Item: 
        Title: CRediT authorship contribution statement
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       Item: 
        Title: Declaration of competing interest
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       Item: 
        Title: Declaration of competing interest
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       Item: 
        Title: Acknowledgements
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       Item: 
        Title: Appendix A. Supplementary data
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       Item: 
        Title: References
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    Title: Treatment of infantile-onset Pompe disease in a rat model with muscle-directed AAV gene therapy
    Author: Sergio Muñoz
    Subject: Molecular Metabolism, 81 (2024) 101899. doi:10.1016/j.molmet.2024.101899
    Creator: Elsevier
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   Type: CRC32
  Checksum: 26897faea7e8d5649b3592d597ed79d5
   Type: MD5
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   Type: SHA-1
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   Type: SHA-256