Can neonatal exposure improve the effectiveness of cystic fibrosis gene therapy re-dosing?

Chief Investigator:

Dr Nigel Farrow

Research Area:

Basic Science

Funding Amount:

$ 34,624


The University of Adelaide


The lung grows and its cells turn over numerous times throughout a person’s lifetime; this continual self-renewal can limit the longevity of gene therapies targeting the lung, leading to the need for re-dosing. Since immunological responses are known to reduce the re-dosing potential of gene therapies, we will assess the theory that neonatal exposure to gene therapy can promote tolerisation, thereby improving the efficacy of re-administered gene therapy later in life. This study will help verify that very early gene therapy initiation is a justified course of action to prevent lung disease in infants with cystic fibrosis.

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