title: Ethical and regulatory aspects of genome editing.
abstract: Gene editing is a rapidly developing area of biotechnology in which the nucleotide sequence of the genome of living cells is precisely changed. The use of genome-editing technologies to modify various types of blood cells, including hematopoietic stem cells, has emerged as an important field of therapeutic development for hematopoietic disease. Although these technologies offer the potential for generation of transformative therapies for patients suffering from myriad disorders of hematopoiesis, their application for therapeutic modification of primary human cells is still in its infancy. Consequently, development of ethical and regulatory frameworks that ensure their safe and effective use is an increasingly important consideration. Here, we review a number of issues that have the potential to impact the clinical implementation of genome-editing technologies, and suggest paths forward for resolving them such that new therapies can be safely and rapidly translated to the clinic.
authors.affiliation: Department of Microbiology, Immunology, and Molecular Genetics, and Department of Pediatrics, David Geffen School of Medicine, and Eli and Edythe Broad Center of Regenerative Medicine and Stem Cell Research, University of California, Los Angeles, Los Angeles, CA;
authors.forename: Donald B
authors.identifier: 0000-0003-1840-6087
authors.initials: DB
authors.lastname: Kohn
authors.affiliation: Department of Pediatrics, Stanford University, Stanford, CA;
authors.forename: Matthew H
authors.identifier: 0000-0002-3850-4648
authors.initials: MH
authors.lastname: Porteus
authors.affiliation: Departments of Pediatrics and Immunology, University of Washington School of Medicine, Seattle, WA; and Program in Cell and Gene Therapy, Seattle Children's Research Institute, Seattle, WA.
authors.forename: Andrew M
authors.identifier: 0000-0002-4292-6114
authors.initials: AM
authors.lastname: Scharenberg
mesh_terms: D000818:Animals; D026688:Bioethical Issues; D000072669:Gene Editing / Q000941:ethics / Q000331:legislation & jurisprudence / Q000379:methods; D006801:Humans; D052416:Targeted Gene Repair / Q000941:ethics / Q000331:legislation & jurisprudence / Q000379:methods