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

ABSTRACT: 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 respon

SUBMITTER: Clark RDE 

PROVIDER: S-EPMC11283059 | biostudies-literature | 2024 Sep

REPOSITORIES: biostudies-literature