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A Protocol for the Production of Integrase-deficient Lentiviral Vectors for CRISPR/Cas9-mediated Gene Knockout in Dividing Cells

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Published on: December 12, 2017

Nonviral gene delivery: principle, limitations, and recent progress.

Mohammed S Al-Dosari¹, Xiang Gao

¹Department of Pharmacognosy, College of Pharmacy, King Saud University, P.O. Box 2457, Riyadh, 11451, Saudi Arabia. msdosari@yahoo.com

The AAPS Journal

|October 17, 2009

Summary

Nonviral gene therapy offers a safer alternative to viral vectors for treating genetic disorders. Enhancing gene delivery efficiency is crucial for its clinical success.

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Area of Science:

Biomedical Engineering
Molecular Biology
Gene Therapy

Background:

Gene therapy is a promising treatment for genetic and acquired disorders.
Nonviral gene transfer vectors are gaining attention due to their safety profile (low immunogenicity, low toxicity) and potential for tissue specificity, offering an alternative to viral vectors.
Despite promising preclinical and clinical results, the efficiency of gene delivery remains a significant challenge for nonviral methods.

Purpose of the Study:

This review critically examines current and novel chemical and physical methods for in vivo delivery of therapeutic nucleic acids.
It highlights the limitations of existing nonviral gene delivery systems.
The review discusses recent advancements aimed at improving nonviral gene delivery approaches.

Main Methods:

The review synthesizes information from preclinical studies and human clinical trials.
It analyzes various chemical and physical strategies employed for nonviral gene delivery.
Focus is placed on evaluating the effectiveness and challenges of these methods.

Main Results:

Nonviral gene delivery methods have shown therapeutic benefits in animal models.
However, achieving sufficient gene delivery efficiency in vivo remains a major hurdle for clinical translation.
Current nonviral approaches face challenges related to targeting, stability, and cellular uptake.

Conclusions:

Nonviral gene therapy holds significant potential, but overcoming delivery efficiency barriers is essential for widespread clinical application.
Continued research into refined chemical and physical delivery methods is critical.
Advancements in nonviral vector design and delivery strategies are needed to fully realize the therapeutic promise of gene therapy.