Gene Therapy Delivery

Gene therapy is a rapidly growing field of biotechnology that uses genetic material to treat or prevent diseases caused by genetic mutations. Dendrimers are a class of highly branched and symmetric macromolecules that have been explored as delivery materials for gene therapy. Dendrimers have several unique properties that make them attractive for gene delivery, such as a well-defined and monodisperse size, a high degree of surface functionalization, and the ability to encapsulate or complex with DNA or RNA molecules.

Introduction into Gene Therapy

The goal of gene therapy is to replace, remove, or repair a defective or missing gene, or to introduce a new gene into a patient's cells to provide a therapeutic benefit. The basic concept of gene therapy is to deliver a therapeutic gene to the patient's cells using a vector, such as a virus, that can enter cells and deliver the gene to the nucleus. The vector can be modified to target specific cells or tissues. Once inside the cell, the therapeutic gene can integrate into the genome, replace a missing or defective gene, or provide a new function to the cell. Gene therapy has the potential to provide a long-term or even permanent cure for genetic diseases that currently have no effective treatments.

• DNA Delivery
  In recent years, the introduction of DNA into target cells can achieve gene therapy for the treatment of many serious incurable diseases such as cancer and genetic diseases.
• siRNA Delivery
  After delivery of siRNA to target cells, target genes can be reversibly silenced to achieve gene therapy.
• mRNA Delivery
  As a new class of nucleic acid drugs, mRNA can be used in gene therapy, vaccine, gene editing and other fields after delivery to target cells.

Delivery System for Gene Therapy

Gene therapy is a rapidly evolving field that offers a promising approach to treating genetic disorders. Gene therapy involves the delivery of therapeutic genes to target cells to correct or replace the defective genes. The success of gene therapy depends on the efficient delivery of therapeutic genes to target cells using safe and effective delivery materials. Some of the common delivery system for gene therapy include:

• Liposomes
• Peptides
• Polymeric Vectors
• Gold Nanoparticles
• Viral Vectors

Dendrimers are Promising Polymeric Vectors of Gene Therapy

Some of the advantages of using dendrimers for gene therapy include:

• High Transfection Efficiency
  Dendrimers can efficiently deliver genes to target cells and have been shown to have higher transfection efficiency compared to other non-viral gene delivery vectors.
• Low Toxicity
  Dendrimers have a low toxicity profile and are less immunogenic compared to viral vectors.
• Controlled Size and Shape
  Dendrimers have a uniform size and shape, which allows for the control of the physical and chemical properties of the delivery system.
• High Surface Functionality
  Dendrimers have a high degree of surface functionality, which allows for the attachment of targeting ligands, imaging agents, and other functional molecules.
• Versatility
  Dendrimers can be synthesized with various types of functional groups and can be modified to tune their properties for specific applications.

Gene transfection mechanism of dendrimers, with emphasis on the benefits brought by fluorinated dendrimers. (Yang J, et al., 2015)

How We Can Help

Based on an advanced technology platform and experienced scientists, CD BioSciences is committed to providing you with innovative dendrimer products and high quality, hassle-free custom services that will make a significant contribution to the success of your project. We are confident that we can break through all the difficulties of a dendrimer research project and meet each of your specific needs. All our deliverables will undergo rigorous therapeutic testing and be delivered on time. For more detailed information, please feel free to contact us or send us an inquiry directly.

Reference

1. Yang J.; et al. Surface-engineered dendrimers in gene delivery. Chem Rev. 2015, 15: 5274-300.