Advances in Engineered Virus-Like Particles for Genome Editing and Therapy

Area of Science:

• Biotechnology
• Gene Therapy
• Molecular Biology

Background:

• Engineered virus-like particles (eVLPs) are emerging as advanced delivery systems for genome editing technologies.
• They combine viral vector entry efficiency with nonviral platform safety for transient delivery of ribonucleoproteins (e.g., Cas9, base editors, prime editors).

Purpose of the Study:

• To review the principles, advances, and therapeutic potential of eVLPs for genome editing applications.
• To discuss the challenges and future directions for eVLP-based gene editing therapies.

Main Methods:

• Summary of structural and production principles of eVLPs.
• Survey of key developmental advances in eVLP design and engineering.
• Analysis of pseudotyping strategies for cell-type-specific targeting.

Main Results:

• Rational engineering and directed optimization have improved eVLP assembly, cargo stability, and editing efficiency.
• Pseudotyping enhances eVLP versatility for targeted delivery.
• Preclinical studies demonstrate eVLP potential in monogenic and complex disease models.

Conclusions:

• eVLPs represent a promising platform for transient delivery of genome editing agents.
• Advancements in eVLP technology support their progression toward clinical translation for various genetic disorders.
• Further development is needed to address future challenges in therapeutic applications.