Exploring Advanced CRISPR Delivery Technologies for Therapeutic Genome Editing

Abstract

The genetic material within cells plays a pivotal role in shaping the structure and function of living organisms. Manipulating an organism's genome to correct inherited abnormalities or introduce new traits holds great promise. Genetic engineering techniques offers promising pathways for precisely altering cellular genetics. Among these methodologies, clustered regularly interspaced short palindromic repeat (CRISPR), honored with the 2020 Nobel Prize in Chemistry, has garnered significant attention for its precision in editing genomes. However, the CRISPR system faces challenges when applied in vivo, including low delivery efficiency, off-target effects, and instability. To address these challenges, innovative technologies for targeted and precise delivery of CRISPR have emerged. Engineered carrier platforms represent a substantial advancement, improving stability, precision, and reducing the side effects associated with genome editing. These platforms facilitate efficient local and systemic genome engineering of various tissues and cells, including immune cells. This review explores recent advances, benefits, and challenges of CRISPR-based genome editing delivery. It examines various carriers including nanocarriers (polymeric, lipid-derived, metallic, and bionanoparticles), viral particles, virus-like particles, and exosomes, providing insights into their clinical utility and future prospects. This review examines the potential of clustered regularly interspaced short palindromic repeat (CRISPR) technology in cellular genetics, focusing on challenges like low delivery efficiency and off-target effects. It discusses innovative carriers-viral, lipid-based, polymeric particles, and exosomes-that improve stability, reduce side effects, and enhance editing capabilities, aiming to optimize CRISPR delivery for therapeutic use.image (c) 2024 WILEY-VCH GmbH