MULTICOMPONENT SYSTEMS FOR SITE-SPECIFIC GENOME MODIFICATIONS

Abstract

The invention includes systems, compositions, and. methods for the making of modular gene editing through reverse transcriptase related processes. Systems and methods that use modified nucleotides and peptides are specifically provided. Transgene introduction into eukaryotic genomes offers vast opportunities to improve, correct and/or alter genetic expression, and concomitantly serve to treat or ameliorate disease symptoms. Successful transgene insertion would allow for rescue from loss-of-function mutations, inhibition of gain-of-function mutations, the exogenous control of RNA and/or protein expression, the introduction of isoform expression specificity, engineered gene and protein expression, and other useful outcomes.

Documents