Non-viral vectors for gene-based therapy

  title={Non-viral vectors for gene-based therapy},
  author={Hao Yin and Rosemary Lynn Kanasty and Ahmed A. Eltoukhy and Arturo J. Vegas and Joseph R. Dorkin and Daniel G. Anderson},
  journal={Nature Reviews Genetics},
Gene-based therapy is the intentional modulation of gene expression in specific cells to treat pathological conditions. This modulation is accomplished by introducing exogenous nucleic acids such as DNA, mRNA, small interfering RNA (siRNA), microRNA (miRNA) or antisense oligonucleotides. Given the large size and the negative charge of these macromolecules, their delivery is typically mediated by carriers or vectors. In this Review, we introduce the biological barriers to gene delivery in vivo… 

Role of Lipid-Based and Polymer-Based Non-Viral Vectors in Nucleic Acid Delivery for Next-Generation Gene Therapy

This review is centered on non-viral vectors mainly comprising of cationic lipids and polymers for nucleic acid-based delivery for numerous gene therapy-based applications.

Lipid-Based DNA Therapeutics: Hallmarks of Non-Viral Gene Delivery.

Gene therapy is a promising strategy for the treatment of monogenic disorders. Non-viral gene delivery systems including lipid-based DNA therapeutics offer the opportunity to deliver an encoding gene

A new developing class of gene delivery: messenger RNA-based therapeutics.

This review presents the recent progress in mRNA delivery, focusing on comparing the advantages and limitations of non-viral based delivery vectors.

Drug delivery systems for RNA therapeutics

Polymer- based, lipid-based, and conjugate-based drug delivery systems are described, differentiating between those that passively and those that actively target specific cell types.

Design of peptide-based vector for nucleic acid delivery in vivo

A novel peptide specifically for in vivo utilization is designed by using previous knowledge about the physicochemical properties and internalization mechanism of different CPPs and more efficient CPP and nucleic acid complex formulation strategy is developed to prepare more uniformly sized particles to enhance gene delivery and decrease the risk of potential side-effects from aggregated particles.

Non-Viral Vectors for Gene Delivery

The various physical and chemical methods for gene transfer in vitro and in vivo, including tissue specific expression, long lasting gene expression system, enhanced gene transfection efficiency has been achieved with improvement in delivery methods using non-viral vectors.

Non-viral nanocarriers for intracellular delivery of microRNA therapeutics.

This review will present the strategies for intracellular miRNA delivery, and specially focus on rationally designed routes, their mechanisms of action, and potential therapeutics used in the host cells or in vivo studies.

Materials for non-viral intracellular delivery of messenger RNA therapeutics.




Non-viral gene therapy: polycation-mediated DNA delivery

Non-viral vectors, although less efficient at introducing and maintaining foreign gene expression, have the profound advantage of being non-pathogenic and non-immunogenic; they are the subject of this review and could be further modified with cell- targeting ligands to enhance their utility for in vivo applications.

Peptide-guided gene delivery

This review will focus on the application of peptides to mediate nonviral gene delivery, and it is increasingly clear that peptide-guided gene delivery is still in its infancy.

Toxicity of cationic lipids and cationic polymers in gene delivery.

Polyethylenimine-based non-viral gene delivery systems.

Expression of therapeutic proteins after delivery of chemically modified mRNA in mice

In a mouse model of a lethal congenital lung disease caused by a lack of surfactant protein B (SP-B), twice weekly local application of an aerosol of modified SP-B mRNA to the lung restored 71% of the wild-type SP- B expression, and treated mice survived until the predetermined end of the study after 28 days.

Engineering targeted viral vectors for gene therapy

Substantial progress in modifying viral vectors using diverse techniques now allows targeting to many cell types in vitro and, although important challenges remain for in vivo applications, the first clinical trials with targeted vectors have already begun to take place.

Cationic lipids, lipoplexes and intracellular delivery of genes.

  • L. WasunguD. Hoekstra
  • Biology, Chemistry
    Journal of controlled release : official journal of the Controlled Release Society
  • 2006

Current progress of siRNA/shRNA therapeutics in clinical trials

An overview of siRNA therapeutics in clinical trials is provided, including their clinical progress, the challenges they have encountered, and the future they hold in the treatment of human diseases.

Inorganic nanoparticles as carriers of nucleic acids into cells.

The current state of the art of transfection of nucleic acids into living cells is discussed from a chemical viewpoint and advantages and disadvantages of the available methods are compared.