Fig. 1: Tools for editing the genomes of cells within the body.

Activities of the SCGE Consortium converge on editing the genome of cells inside the human body. a, Targets of the genome editors (right) range from DNA within the nucleus of a cell to other nucleic acids elsewhere within a cell, such as DNA within the mitochondria (mtDNA) or RNA in the cytoplasm. Targets of epigenomic editors (left) produce targeted alteration of the chromatin structure—including remodelling, modification of the 3D structure and the direct modification of histones or DNA—without editing the DNA or RNA sequence. Approaches to editing cells outside of the body, as well as germline editing in embryos, are not directly supported by the SCGE Consortium, nor are strategies for gene augmentation through the addition of exogenous DNA. b, Interoperable tools assembled into an SCGE Toolkit will be disseminated to accelerate the translation of safe and effective genome-editing therapeutics into the clinic. Tools encompass several categories: newly developed genome editors, delivery technologies, reporter-animal systems, and human biological systems.