Can neonatal exposure improve the effectiveness of cystic fibrosis gene therapy re-dosing?
Dr Nigel Farrow
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.