A comprehensive analysis of scRNA-Seq and RNA-Seq unveils B cell immune suppression in the AAV-loaded brain.

ABSTRACT: The use of AAV vectors for in vivo gene therapy has demonstrated the potential to permanently correct genetic diseases by delivering functional gene copies to the nuclei of affected tissues. AAV vectors, as tools for in vivo gene delivery, are particularly appealing and have shown safety and long-term efficacy in numerous organ-targeted experiments. Nevertheless, employing AAV vectors for gene therapy in the brain faces a notable hurdle in the shape of immune responses, chiefly instigated by the brain's resident immune cells, microglia. Additionally, lower levels of AAV vector-neutralizing antibodies have been detected in the cerebrospinal fluid compared to the circulatory system. This research, leveraging transcriptomic and single-cell RNA sequencing (scRNA-seq) data in conjunction with M

SUBMITTER: Wu S 

PROVIDER: S-EPMC11882665 | biostudies-literature | 2025 Mar

REPOSITORIES: biostudies-literature