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Review Article Volume 12 Issue 2
Current CRISPR-Cas gene editing systems and therapeutic
Foo Suanhow, Tawil Bill
Received: May 25, 2025 | Published: June 9, 2025
Citation: Suanhow F, Bill T. Current CRISPR-Cas gene editing systems and therapeutics. J Appl Biotechnol Bioeng. 2025;12(2):86-92. DOI: 10.15406/jabb.2025.12.00389
Abstract
In 2023 Casgevy (Exagamglogene autotemcel) became the first CRISPR-Cas therapeutic to be approved in the United States. This therapeutic is used to treat sickle cell anemia and represents the culmination of research in the field of CRISPR-Cas systems as a human therapeutic. As of 2022, there are around 71 clinical trials related to CRISPR in various stages. Unfortunately, CRISPR-Cas therapeutics can be expensive, as Cagevy is reported to cost 2.2 million USD per treatment. Fortunately, there is a good possibility the cost of CRISPR therapeutics can be reduced, as researchers today have more tools at their disposal to create gene edited products. These tools include more options at the Cas effector level, such as Cas9, Cas 12, Cas13, and xCas9. In addition, increased efficiency for CRISPR- Cas editing can be improved using guide RNA spacer modifications and RNA aptamers to improve Cas binding to the guide RNAs. Software such as CHOPCHOP and Synthego for guide RNA designs can help researchers visualize and improve guide RNA strategies. This paper will discuss current CRISPR-Cas systems and strategies used to make gene editing more efficient and provide a glimpse into the future of gene editing treatments by discussing current CRISPR-Cas therapeutics in clinical trials.
Keywords: Base editing, casgevy, Cas12, Cas13, CRISPR-Cas9, gRNA, PAM, prime editing, tracrRNA
©2025 Suanhow, et al. This is an open access article distributed under the terms of the, which permits unrestricted use, distribution, and build upon your work non-commercially.