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Category: AAV-mediated

AAV-mediated gene replacement therapy for LRAT-associated retinitis pigmentosa: a proof-of-concept study in a patient-based rat model
  • AAV-mediated

AAV-mediated gene replacement therapy for LRAT-associated retinitis pigmentosa: a proof-of-concept study in a patient-based rat model

  • March 10, 2026
  • 0

Introduction Retinitis pigmentosa (RP) is a monogenic disease that causes progressive vision loss and blindness [1, 2]. RP often has an early onset and has […]

AAV-mediated MUC5AC siRNA delivery to prevent mucociliary dysfunction in asthma
  • AAV-mediated

AAV-mediated MUC5AC siRNA delivery to prevent mucociliary dysfunction in asthma

  • August 23, 2025
  • 0

Introduction Asthma is a chronic lung disease, affecting millions worldwide, adults and children alike [1]. Individuals with asthma typically experience coughing, difficulty breathing and wheezing […]

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