Genetic Therapy

* This product is for research use only. Not intended for use in the treatment or diagnosis of disease.

Summary

Gene therapy is a new technology that combines modern medicine and molecular biology by introducing the target gene into the patient and achieving the target gene product. Gene therapy as a new approach to disease treatment has already had some successful applications, and scientific breakthroughs will continue to push gene therapy into mainstream medicine.

Gene therapy can work through a variety of mechanisms:

  • Replace the disease-causing gene with a healthy copy of the gene.
  • Inactivate or turn off disease-causing genes that do not work properly.
  • Turn on a gene to help fight the disease.
  • Removing a section of DNA that can impair the function of a gene and cause disease.
  • Introducing new or modified genes into the body to help treat disease.

A variety of vectors have been developed to carry the target genes into the body, these vectors can be injected directly or intravenously into specific tissues in the body, where it is taken up by individual cells. Alternatively, samples of patient cells can be taken in a laboratory setting and exposed to the carrier. Cells containing the vector are then returned to the patient. If the treatment is successful, the new gene delivered by the carrier will produce a functional protein, or the edited molecule will correct the DNA error and restore protein function.

  • Plasmid DNA

Circular DNA molecules can be genetically engineered to carry therapeutic genes into human cells.

  • Viral vectors

Viruses have the natural ability to deliver genetic material to cells, so some gene therapy products are derived from viruses (Retroviruses, adenoviruses, adeno-associated viruses, etc). Once a virus has been modified to eliminate its ability to cause infectious disease, these modified viruses can be used as vehicles to carry therapeutic genes into human cells.

  • Bacterial vectors

Bacteria can be modified to prevent them from causing infectious diseases and then used as vehicles to carry therapeutic genes into human tissues.

Application

  • CAR T cell therapy (or chimeric antigen receptor T cell therapy) is an example of cell-based gene therapy. CAR T cell therapy introduces genes into human T cells, which are a type of immune cell. The gene provides instructions for making a protein, called a chimeric antigen receptor (CAR), that attaches to cancer cells. The modified immune cells can specifically attack cancer cells.
  • Adenovirus Gendicine is a gene therapy based on the p53 gene, which is used to treat head and neck cancers.
  • Genome editing is a new technique that could be used in gene therapy. Instead of adding new genetic material, genome editing introduces gene-editing tools that can alter existing DNA in cells. Genome editing technology allows the addition, removal, or alteration of genetic material at precise locations in the genome. CRISPR-Cas9 is a well-known type of genome editing.

BOC Sciences' Transfection Kits

Product Information
Catalog No.Product NameDescription
BT-000001DNA in vivo Delivery KitReagents designed for plasmid DNA in vivo transfection.
BT-000044siRNA/DNA Transfection ReagentBOC Sciences siRNA/DNA transfection reagent is a highly efficient, low toxicity, ideal siRNA and plasmid DNA transfection reagent for mammalian cells.
BT-000030DOTAP-Lipo Transfection ReagentReagents for DNA, RNA and protein transfection.
BT-000023Broad Spectrum DNA Transfection ReagentReagents for DNA transfection.
BT-000046CRISPR RNP Transfection ReagentBOC Sciences' CRISPR RNP transfection reagent is a polymer-based transfection reagent with a membrane fusion virus mechanism that can efficiently deliver Cas9 ribonucleoprotein (RNP) in vitro.
BT-000005Cas9 Transfection ReagentOptimized lipid nanoparticle transfection reagent for CRISPR-Cas9 protein delivery
BT-000042HTT mRNA Transfection ReagentReagents for high-efficiency delivery of mRNA into hard-to-transfect cells.
BT-000041mRNA Transfection ReagentTransfection reagent for mRNA.
BT-000043siRNA Transfection ReagentBOC Sciences' siRNA transfection reagent is an efficient, low toxicity, ideal siRNA transfection system for mammalian cells.
BT-000012Hela CRISPR Transfection ReagentProprietary preparation reagent optimized for delivery of CRISPR/Cas9 gene modification system into Hela cells.
BT-000014HepG2 CRISPR Transfection ReagentProprietary preparation reagent optimized for delivery of CRISPR/Cas9 gene modification system into HepG2 cell line.
BT-000022Astrocytes Transfection ReagentReagents optimized for high-efficient delivery of DNA and RNA into Astrocyte cells.
BT-000021Neuro-2a Transfection ReagentReagents designed for efficient delivery of DNA and RNA into neuroblastoma cells.
BT-000020Neuro-Transfection ReagentReagents designed for high-efficient transfection of DNA into nerve cells.

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