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Evaluation of anti-vector immune responses to adenovirus-mediated lung gene therapy and modulation by αCD20.

Robert D E ClarkFelix RabitoFerris T MunyonhoT Parks RemchoJay K Kolls
Published in: Molecular therapy. Methods & clinical development (2024)
Although the last decade has seen tremendous progress in drugs that treat cystic fibrosis (CF) due to mutations that lead to protein misfolding, there are approximately 8%-10% of subjects with mutations that result in no significant CFTR protein expression demonstrating the need for gene editing or gene replacement with inhaled mRNA or vector-based approaches. A limitation for vector-based approaches is the formation of neutralizing humoral responses. Given that αCD20 has been used to manage post-transplant lymphoproliferative disease in CF subjects with lung transplants, we studied the ability of αCD20 to module both T and B cell responses in the lung to one of the most immunogenic vectors, E1-deleted adenovirus serotype 5. We found that αCD20 significantly blocked luminal antibody responses and efficiently permitted re-dosing. αCD20 had more limited impact on the T cell compartment, but reduced tissue resident memory T cell responses in bronchoalveolar lavage fluid. Taken together, these pre-clinical studies suggest that αCD20 could be re-purposed for lung gene therapy protocols to permit re-dosing.
Keyphrases
  • gene therapy
  • cystic fibrosis
  • immune response
  • nk cells
  • pseudomonas aeruginosa
  • lung function
  • zika virus
  • multidrug resistant
  • quality improvement
  • small molecule
  • copy number
  • air pollution