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Treatment of heritable diseases using CRISPR: Hopes, fears, and reality
Natalie Kofler1, Katherine L Kraschel2
1Founder, Editing Nature and Associate Research Scientist, Yale Institute of Biospheric Studies, Yale Interdisciplinary Center for Bioethics, 21 Sachem St., New Haven, CT 06511, United States.
Abstract:
CRISPR gene editing is poised to transform the therapeutic landscape for diseases of genetic origin. The ease and agility by which CRISPR can make specific changes to DNA holds great promise not only for the treatment of heritable diseases, but also their prevention through germline editing. CRISPR-based therapeutic strategies are currently under development for numerous monogenic diseases. These strategies range from proof of concept studies demonstrating pre-fertilization gamete editing to recently initiated clinical trials for postnatal ex vivo therapies. The promise of CRISPR's human genome editing potential has captivated the public's attention. It is of paramount importance that medical professionals who work with patients who may have or carry a monogenic heritable disease understand CRISPR technology in order to have informed and compassionate discussions with their patients. Understanding CRISPR means understanding its evolving therapeutic applications' nuances, limitations, and barriers to access as well as the regulatory landscape they inhabit. In this piece we provide a review of the promises and pitfalls of CRISPR germline gene editing and their implications for patient decision-making throughout various stages of the reproductive process.
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