Long-Term Functional Correction of Pompe Disease and Increased α-Glucosidase Expression after Gene Therapy with Novel Combinations of Muscle-Targeted Transcriptional Cis-Regulatory Elements

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  FWOAL1073: HERCULES: Development of an innovative muscle-directed gene therapy platform for lysosomal storage disorders

  VandenDriessche, T. (Administrative Promotor)

  1/01/23 → 31/12/26

  Project: Fundamental

  • Regulatory Sequence 100%
  • Muscle 83%
  • Skeletal Muscle 78%
  • Adeno Associated Virus Vector 71%
  • Glycogen 64%