Heritable human genome editing: Research progress, ethical considerations, and hurdles to clinical practice

Our genome at conception determines much of our health as an adult. Most human diseases have a heritable component and thus may be preventable through editing. Preventing disease from the beginning life before irreversible damage has occurred is admirable goal, but path to fruition remains unclear. Here, we review significant scientific contributions field editing, unique ethical challenges that cannot overlooked, hurdles must overcome prior translating these technologies into clinical practice. The introduction editing using clustered regularly interspaced short palindromic repeats (CRISPR)-based generated tremendous enthusiasm well controversy within medical public communities. Heritable Human Genome Editing (HHGE) potential treat or even eradicate genetic diseases. By addressing defect amplified cell proliferation during development, HHGE prove more effective than any other treatment being developed today, including somatic gene drug therapy (Figure 1). Somatic therapies are limited in their ability reverse already reach billions cells needed adequately disease. brief vitro culture embryo routinely practiced IVF clinics provides readily accessible window for prevention numerous conditions later difficult manage, let alone cure. This hope rationale research, not yet therapy, criteria efficacy safety met. also raises regulatory questions. Manipulations early highly consequential, both with regard benefits risks. Other reviews discussed emphasizing its understanding function (Lea Niakan, 2019Lea R.A. Niakan K. germline editing.Nat. Cell Biol. 2019; 21: 1479-1489Crossref PubMed Scopus (20) Google Scholar; Rossant, 2018Rossant J. Gene development: concerns practical applications.Development. 2018; 145: dev150888Crossref (16) Plaza Reyes Lanner, 2017Plaza A. Lanner F. 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