Gene editing targeting the AAVS1 site for site specific integration of microdystrophin for treatment of Duchenne Muscular Dystrophy

Arjun Agarwal

Research output: ThesisDoctoral Thesis

Original languageEnglish
Awarding Institution
  • Royal Holloway, University of London
  • Popplewell, Linda, Supervisor
  • Malerba, Alberto, Supervisor
  • Yáñez-Muñoz, Rafael J., Advisor
Thesis sponsors
Award date1 Dec 2023
Publication statusUnpublished - 2023


  • Gene therapy
  • Gene editing
  • Duchenne muscular dystrophy
  • CRISPR/Cas9

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