Emerging non-viral vectors for gene delivery

Area of Science:

• Biomedicine
• Molecular Biology
• Materials Science

Background:

• Gene therapy aims to treat diseases by delivering functional genes into cells.
• Clinical gene therapy is hindered by the absence of safe and efficient gene delivery vehicles.

Purpose of the Study:

• To review novel, fast-growing, and efficient non-viral gene delivery vectors.
• To discuss research progress, development directions, and challenges of these vectors.
• To provide an overview of approved non-viral gene therapeutics and clinical trials.

Main Methods:

• Comprehensive literature review of non-viral gene delivery systems.
• Analysis of research progress and potential development of vectors.
• Overview of current clinical applications and trials.

Main Results:

• Identified key non-viral vectors: liposomes, lipid nanoparticles (LNPs), poly(β-amino ester) (HPAE), single-chain cyclic polymers (SCKP), poly(amidoamine) (PAMAM) dendrimers, and polyethyleneimine (PEI).
• Detailed research progress, future directions, and existing challenges for each vector type.
• Summarized currently approved non-viral gene therapeutics and ongoing clinical trials.

Conclusions:

• Non-viral gene vectors are crucial for advancing clinical gene therapy.
• Continued research in materials science and molecular biology will enhance gene delivery efficiency and safety.
• Non-viral vectors are increasingly vital in the expanding field of clinical gene therapy.