Acceptance and Access to Gene Editing: Science and Our Obligations to Mankind

DOI: http://dx.doi.org/10.1016/j.ymthe.2016.12.012

It has been six decades since the elucidation of the structure of DNA,1 four decades since the introduction of restriction endonucleases opened up recombinant DNA technology, and a decade and a half since the sequencing of the human genome.2 We are on the verge of therapies that could permanently remedy genetic diseases. Gene editing using cellular, viral, and synthetic nucleotide templates, accompanied by engineered site-specific nucleases (zinc finger nucleases [ZFNs], clustered regularly interspaced short palindromic repeats [CRISPR]/Cas, transcription activator-like effector nucleases [TALENs]) now offer possibilities of treatment for diseases such as hemophilia, thalassemia, or cancers related to genetic mutations.

Published online: December 27, 2016