Extracellular Vesicles as Promising Vectors for CRISPR/Cas9 Delivery: Advantages, Challenges, and Clinical Implications

Abstract

 The CRISPR/Cas system, with its capacity to modify DNA sequences and thus change cellular and organ traits, holds promise for gene study and therapeutic interventions. However, the absence of reliable and efficient delivery mechanisms curtails its clinical usage. Extracellular vesicles (EVs) present an appealing avenue for transporting CRISPR/Cas9. They offer multiple benefits over other delivery vehicles, especially in safety, protection, carrying capacity, tissue penetration, targeting precision, and adaptability. As a result, there’s a growing trend in employing EVs for in vivo delivery of CRISPR/Cas9. This article provides an overview of the strengths and weaknesses associated with various CRISPR/Cas9 delivery mechanisms and vehicles. It underscores the unique attributes of EVs as carriers, delving into their inherent features, physiological and pathological roles, safety profile, and targeting proficiency. The piece also delves into the origins of EVs, methods for their isolation, and techniques for packing CRISPR/Cas9 within them. In its conclusion, the article charts a path forward, emphasizing the pivotal areas of focus for optimizing EVs as carriers in CRISPR/Cas9’s clinical utilization, namely the safety, storage potential, uniformity, production rate, and precise targeting of EVs.