Fong, Yuman, Hackett, Perry B
Elsevier BV
Biochemistry, Genetics and Molecular Biology, Pharmacology, Toxicology and Pharmaceutics
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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 nucle-ases [ZFNs], clustered regularly interspaced short palindromic re-peats [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. 3 Decisions on which diseases as well as how to regulate and pay for treatments are increasingly debated. 4 Much of the debate on gene engineering involves the ethics of genetically altering germ cells and embryos. 5 In this Editorial, we wish to discuss other pertinent issues on the application of precision genetic technologies to encourage open discussion, debate, and planning. There are some diseases that essentially everyone would agree should be treated with somatic cell gene editing. These include adenosine deaminase deficiency, hemophilia, b-thalassemia, cystic fibrosis, and many others, where targeted gene correction of cells to produce structural or secretory proteins would alleviate or abrogate devas-tating effects of the disease. The potential savings in human suffering, societal expense, and gain in human life and productivity would justify gene-editing therapy.

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