Rationally Designed MicroRNA-Based Genetic Classifiers Target Specific Neurons in the Brain.

ABSTRACT: Targeting transgene expression to specific cell types in vivo has proven instrumental in characterizing the functional role of defined cell populations. Genetic classifiers, synthetic transgene constructs designed to restrict expression to particular classes of cells, commonly rely on transcriptional promoters to define cellular specificity. However, the large size of many natural promoters complicates their use in viral vectors, an important mode of transgene delivery in the brain and in human gene therapy. Here, we expanded upon an emerging classifier platform, orthogonal to promoter-based strategies, that exploits endogenous microRNA regulation to target gene expression. Such classifiers have been extensively explored in other tissues; however, their use in the nervous system has thus f

SUBMITTER: Sayeg MK 

PROVIDER: S-EPMC4547530 | biostudies-literature | 2015 Jul

REPOSITORIES: biostudies-literature