Precision Gene Editing and Applications in Nephrology

The expanding field of precision gene editing is empowering researchers to directly modify DNA. Gene editing is made possible using synonymous technologies: 1) a DNA targeting platform to molecularly locate user-selected genomic sequences, and 2) an associated biochemical activity that serves as a functional editor. The advent of accessible DNA-targeting molecular systems, like transcription activator like effectors (TALEs) and Clustered Regularly Interspersed Short Palindromic Repeats (CRISPR)-Cas9 gene editing systems, has unlocked the ability to target nearly any DNA sequence with nucleotide-level precision. Progress has also been made in how we can harness endogenous DNA repair machineries to functionally manipulate genetic sequences. The more that is understood about how DNA damage results in deletions, insertions, and modifications the more predictably mutable the genome becomes. These genomic targeting platforms are also useful for locus-specific epigenetic changes and transcriptional enhancement and suppression. This new genome engineering technology builds on a long history of renal science, enabling new animal models of disease as well as novel therapeutic options.