title: Salient Features of Endonuclease Platforms for Therapeutic Genome Editing.
abstract: Emerging gene-editing technologies are nearing a revolutionary phase in genetic medicine: precisely modifying or repairing causal genetic defects. This may include any number of DNA sequence manipulations, such as knocking out a deleterious gene, introducing a particular mutation, or directly repairing a defective sequence by site-specific recombination. All of these edits can currently be achieved via programmable rare-cutting endonucleases to create targeted DNA breaks that can engage and exploit endogenous DNA repair pathways to impart site-specific genetic changes. Over the past decade, several distinct technologies for introducing site-specific DNA breaks have been developed, yet the different biological origins of these gene-editing technologies bring along inherent differences in parameters that impact clinical implementation. This review aims to provide an accessible overview of the various endonuclease-based gene-editing platforms, highlighting the strengths and weakness of each with respect to therapeutic applications.
authors.affiliation: bluebird bio, Cambridge, Massachusetts, USA.
authors.forename: Michael T
authors.initials: MT
authors.lastname: Certo
authors.affiliation: bluebird bio, Cambridge, Massachusetts, USA.
authors.forename: Richard A
authors.initials: RA
authors.lastname: Morgan
mesh_terms: D000818:Animals; D004720:Endonucleases / Q000378:metabolism*; D000072669:Gene Editing / Q000266:history / Q000379:methods; D018014:Gene Transfer Techniques; D005818:Genetic Engineering / Q000266:history / Q000379:methods; D015316:Genetic Therapy / Q000379:methods; D005822:Genetic Vectors / Q000235:genetics; D016678:Genome; D049673:History, 20th Century; D006801:Humans; D014161:Transduction, Genetic
chemical_list: D004720:Endonucleases