Non-Viral CRISPR carriers: transient delivery with lasting effects

Area of Science:

• Genomic Medicine
• Gene Therapy Delivery Systems

Background:

• CRISPR-Cas9 technology has transformed genome editing capabilities.
• Traditional gene therapies heavily rely on viral vectors for delivery.
• Non-viral delivery methods are gaining traction for in vivo CRISPR applications.

Purpose of the Study:

• To systematically review the current clinical trial landscape for in vivo CRISPR therapies.
• To discuss the various delivery strategies employed in these trials.
• To highlight the shift towards non-viral vectors in CRISPR delivery.

Main Methods:

• Systematic survey of ongoing clinical trials involving in vivo CRISPR therapies.
• Analysis of utilized delivery strategies, focusing on viral versus non-viral vectors.
• Review of CRISPR technologies and non-viral delivery platforms in clinical use.

Main Results:

• As of December 2025, 136 CRISPR trials are active globally.
• 36 of these trials utilize in vivo delivery of CRISPR components.
• A significant trend shows a clear shift towards non-viral vectors for in vivo delivery.

Conclusions:

• Non-viral delivery strategies are increasingly favored for in vivo CRISPR gene editing.
• Transient delivery of CRISPR components is advantageous for therapeutic effects and safety.
• Further development of non-viral platforms is crucial for future CRISPR delivery advancements.