Therapeutic genome editing: prospects and challenges
Author(s)Cox, David Benjamin Turitz; Platt, Randall Jeffrey; Zhang, Feng
MetadataShow full item record
Recent advances in the development of genome editing technologies based on programmable nucleases have substantially improved our ability to make precise changes in the genomes of eukaryotic cells. Genome editing is already broadening our ability to elucidate the contribution of genetics to disease by facilitating the creation of more accurate cellular and animal models of pathological processes. A particularly tantalizing application of programmable nucleases is the potential to directly correct genetic mutations in affected tissues and cells to treat diseases that are refractory to traditional therapies. Here we discuss current progress toward developing programmable nuclease–based therapies as well as future prospects and challenges.
Available in PMC 2015 July 06
DepartmentHarvard University--MIT Division of Health Sciences and Technology; Massachusetts Institute of Technology. Department of Biological Engineering; Massachusetts Institute of Technology. Department of Biology; Massachusetts Institute of Technology. Department of Brain and Cognitive Sciences; McGovern Institute for Brain Research at MIT
Cox, David Benjamin Turitz, Randall Jeffrey Platt, and Feng Zhang. "Therapeutic genome editing: prospects and challenges." Nature Medicine 21:2 (February 2015), pp.121-131.
Author's final manuscript