Therapeutic Gene Editing with CRISPR: A Laboratory Medicine Perspective.

Elan Hahn, Matthew Hiemenz

Journal: Clinics in laboratory medicine 2021;40(2):205-219

PMID: 32439069

Full Text on Elsevier Science View on PubMed

Abstract

Therapeutic gene editing with the clustered regularly interspaced short palindromic repeat (CRISPR)-Cas system offers significant improvements in specificity and programmability compared with previous methods. CRISPR editing strategies can be used ex vivo and in vivo with many theoretic disease applications. Off-target effects of CRISPR-mediated gene editing are an important outcome to be aware of, minimize, and detect. The current methods of regulatory approval for personalized therapies are complex and may be proved inefficient as these therapies are implemented more widely. The role of pathologists and laboratory medicine practitioners is vital to the clinical implementation of therapeutic gene editing.

Address: Department of Laboratory Medicine and Pathobiology, University of Toronto, Medical Sciences Building, Room 6231, 1 King's College Circle, Toronto, Ontario M5S 1A8, Canada. Electronic address: [email protected].; Department of Pathology and Laboratory Medicine, Children's Hospital Los Angeles, Los Angeles, California 90027, USA; Department of Pathology, Keck School of Medicine of USC, Los Angeles, California 90033, USA.

DOI: 10.1016/j.cll.2020.02.008

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MeSH Terms: CRISPR-Cas Systems, Gene Editing, Genetic Therapy, Humans, Immunotherapy, Adoptive, Sequence Analysis, DNA, Transcription Activator-Like Effector Nucleases, Zinc Finger Nucleases