Date of Award

8-11-2020

Document Type

Open Access Thesis

Degree Name

MS in Physician Assistant Studies (PA)

Department

Physician Assistant Studies

First Advisor

Holly Levine, MD

Abstract

Gene editing capabilities have expanded enormously since researchers first demonstrated the robust, accurate, and efficient endonuclease activity of the CRISPR/Cas riboprotein enzyme. Since this gene editing technique was first described in 2013, the prospect of gene therapy as a viable clinical tool has improved immensely. CRISPR/Cas techniques and the methods by which they are delivered into cells, have evolved rapidly since the technology􏰿s inception, and successful, intentional genetic alterations of numerous mammalian cell lines have been reported. As a research tool, CRISPR/Cas9 has already proven itself indispensable in a brief period of time. While there are not numerous clinical trials involving CRISPR/Cas technology, CRISPR has absolutely contributed to the acceleration and expansion of gene therapy-based clinical trials in the past five years. Here, the current capabilities of CRISPR/Cas gene editing are evaluated as they relate to the clinical setting, including potential for CRISPR/Cas to function as: a new cancer therapeutic agent, a means of correcting or relieving genetic disease, and a potent disrupter of antimicrobial resistance genes in virulent MDR bacterial strains. Additionally, the future possibilities of CRISPR/Cas-related therapies, and issues preventing the achievement of these therapies in the clinical setting, are discussed.

Identifier

SC 11.PAS.2020.Piper.J

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