Abstract
While research in gene editing technology has been active since the 1990s, recent advances in CRISPR ease of use have sparked significant enthusiasm in the biomedical community. Clustered Regularly Interspaced Short Palindromic Repeats (CRISPR) gene editing has the potential to shape the therapeutic landscape of genetically-defined diseases, with key players such as CRISPR Therapeutics, Intellia, and Editas leading the way in β-thalassemia and sickle cell disease. In coming years, clinical development of CRISPR technology promises to change how we think about monogenic and polygenic diseases, though data is still in its infancy. While concerns regarding safety and administration of CRISPR persist and researchers are still seeking to better understand its effect on the body's ability to recognize and repair damaged DNA, the applications for personalized medicine are exciting and wide-ranging. This review examines potential applications of CRISPR for human therapeutics, with a private sector focus detailing anticipated clinical advances and evolving challenges along the way.
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