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Gene editing using the CRISPR/Cas9 system has been the most important advance in genomic medicine in the last 10-20 years. It allows for efficient and precise correction of genetic mutations and re-purposing of immune cells for personalized therapy of inherited diseases and cancer. It has been covered widely in the lay press, and patients are now coming into the clinic asking about it. Also, it has now entered standard practice in oncology and, increasingly, in medical genetics. This technology is new, largely investigational, and expensive, so there are significant disparaties in access to it. In addition, the genetic disorders to be treated have differing incidence and carrier rates in various ethnic populations, with potential risk of genetic discrimination and stigmatization. Thus, it is important for general clinicians to develop a basic knowledge of its principles and applications, both to address patients’ questions and to make appropriate referrals to the specialized centers offering this cutting-edge therapy.
Learning Objectives
1) Incorporate the scientific principles behind the current revolution in gene editing and its potential to produce highly personalized therapies for cancer and genetic diseases, along with other applications in disease surveillance and infection resistance
2) Confront and analyze the thorny ethical implications of this powerful new technology
3) Counsel patients on the current state of the art and potential clinical utility of gene editing technology for diagnosis and treatment of genetic, neoplastic, and infectious diseases.
How do we know this is a problem?
Porteus MH. A New Class of Medicines through DNA Editing. N Engl J Med. 2019;380:947-959.
Frangoul H, Altshuler D, Cappellini MD, Chen YS, Domm J, Eustace BK, Foell J, de la Fuente J, Grupp S, Handgretinger R, Ho TW, Kattamis A, Kernytsky A, Lekstrom-Himes J, Li AM, Locatelli F, Mapara MY, de Montalembert M, Rondelli D, Sharma A, Sheth S, Soni S, Steinberg MH, Wall D, Yen A, Corbacioglu S. CRISPR-Cas9 Gene Editing for Sickle Cell Disease and β-Thalassemia. N Engl J Med. 2021;384:252-260.
Daley GQ, Lovell-Badge R, Steffann J. After the Storm – A Responsible Path for Genome Editing. N Engl J Med. 2019;380:897-899.
