However, there are several technical and ethical considerations that need addressing when considering its use for patient care. The recent advent of CRISPR technology in clinical trials has paved way for the new era of CRISPR gene therapy to emerge. The discovery and development of the CRISPR/Cas9 system has provided a second opportunity for gene therapy to recover from its stigma and prove to be valuable therapeutic strategy. Gene therapy has witnessed both early successes and tragic failures in a clinical setting. Gene therapy as a strategy to provide therapeutic benefit includes modifying genes via disruption, correction, or replacement ( 1). We review the emerging data of recent gene therapy trials and consider the best strategy to move forward with this powerful but still relatively new technology. This review focuses on the evolution of gene therapy and the role of CRISPR in shifting the gene therapy paradigm. Although it has apparent advantages, CRISPR/Cas9 brings its own set of limitations which must be addressed for safe and efficient clinical translation. While viral vectors remain a key delivery vehicle, CRISPR technology provides a relatively simple and efficient alternative for site-specific gene editing, obliviating some concerns raised by traditional gene therapy. Traditional gene therapy has raised some concerns, as its reliance on viral vector delivery of therapeutic transgenes can cause both insertional oncogenesis and immunogenic toxicity. The discovery of Clustered Regularly Interspaced Short Palindromic Repeats (CRISPR) and CRISPR-associated (Cas) proteins has expanded the applications of genetic research in thousands of laboratories across the globe and is redefining our approach to gene therapy. ![]() 2Weill Cornell Medicine, Cornell University, New York, NY, United StatesĪ series of recent discoveries harnessing the adaptive immune system of prokaryotes to perform targeted genome editing is having a transformative influence across the biological sciences.1Department of Medicine, Thoracic Oncology Service, Memorial Sloan Kettering Cancer Center, New York, NY, United States.
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