AAV9-CSA gene therapy

Intervention

1 paper1 finding

  • 1 lab in 1 country
  • Animals1

Outcomes

1

Lifespan

upin mice1

1 study
  1. AAV9-CSA gene therapy extends lifespan in mice.

    “Treatment with AAV9-CSA resulted in a significant increase in lifespan, and broad distribution of human CSA in the brain and heart.”

    AnimalsCsa -/- ;Xpa -/- mouse model of Cockayne syndromeAAV9 vector encoding human CSA under a CBA promoter

    AAV gene therapy for Cockayne syndrome

    bioRxiv (Cold Spring Harbor Laboratory)9 Jun 2025Preprint

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