Challenges of Assessing Exon 53 Skipping of the Human DMD Transcript with Locked Nucleic Acid-Modified Antisense Oligonucleotides in a Mouse Model for Duchenne Muscular Dystrophy

  • Sarah Engelbeen
  • , Daniel O'Reilly
  • , Davy Van De Vijver
  • , Ingrid Verhaart
  • , Maaike Van Putten
  • , Vignesh Hariharan
  • , Matthew Hassler
  • , Anastasia Khvorova
  • , Masad J. Damha
  • , Annemieke Aartsma-Rus

Research output: Contribution to journal › Article › peer-review

7 Scopus citations

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Keyphrases

Biochemistry, Genetics and Molecular Biology

Immunology and Microbiology

Pharmacology, Toxicology and Pharmaceutical Science