Designing Cell-Type-Specific Promoter Sequences Using Conservative Model-Based Optimization.

ABSTRACT: Gene therapies have the potential to treat disease by delivering therapeutic genetic cargo to disease-associated cells. One limitation to their widespread use is the lack of short regulatory sequences, or promoters, that differentially induce the expression of delivered genetic cargo in target cells, minimizing side effects in other cell types. Such cell-type-specific promoters are difficult to discover using existing methods, requiring either manual curation or access to large datasets of promoter-driven expression from both targeted and untargeted cells. Model-based optimization (MBO) has emerged as an effective method to design biological sequences in an automated manner, and has recently been used in promoter design methods. However, these methods have only been tested using large trai

SUBMITTER: Reddy AJ 

PROVIDER: S-EPMC11213138 | biostudies-literature | 2024 Jun

REPOSITORIES: biostudies-literature