Gene Therapy Products & Technology for Effective Research
Gene therapy offers the prospect of long term and potentially curative benefits to patients with genetic or acquired diseases by harnessing the body’s natural bioprocesses, instead of relying on foreign substances.
Gene therapy uses vectors to deliver a payload that has a therapeutic benefit to the patient. It can involve the direct expression of a therapeutic protein or restore the expression of an under-expressed protein. Payloads include plasma DNA, short interference, and short hairpin RNA (siRNA, shRNA), and micro and messenger RNA, among others. Vectors include viruses, such as AAV, AV, Lenti, and Retro, and non-viral approaches that include cationic lipids and electroporation. Therapeutic payloads can be delivered directly into the patient (viral), or into a cell provided by the patient or a non-related donor.
These approaches can be labeled as in-vivo or ex-vivo and may overlap with the development of therapeutic cell therapy applications. Virus-like particles (VLP) were previously developed for vaccine products and are gaining popularity in gene therapy applications and drug delivery vectors because they can supplement the absence or defective form of an essential protein. VLPs can be expressed in either microbial or mammalian expression systems depending on their complexity.
Avantor® products can help maintain the integrity and activity of gene therapy products during upstream and downstream purification through final fill. Choose from a broad range of cell culture components, fermentation media and supplements, growth factors, reagents and excipients, and single-use use products to help enable aseptic fluid transfer that supports “closed-system” cell culture.
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What Is Gene Therapy?
Gene therapy is an experimental technique that involves inserting genes into a patient’s cells to treat diseases or disorders without using drugs or surgery. Researchers are studying how to use gene therapy to treat several conditions, including inherited disorders, cancer, and some types of viral infections. Because gene therapy technology modifies a person’s genes, the FDA’s Center for Biologics Evaluation and Research (CBER) tightly regulates gene therapy products.
How Does Gene Therapy Work?
Gene therapy involves the direct expression of a therapeutic protein or restoration of the expression of an under-expressed protein. Cell and gene therapy use vectors to deliver a payload that can provide therapeutic benefits to patients. Payloads include plasma DNA, short interference and short hairpin RNA (siRNA, shRNA), and micro or messenger RNA. Vectors include viruses (AAV, AV, Lenti, and Retro) and non-viral approaches (cationic lipids and electroporation). Therapeutic payloads can be delivered directly into the patient or a cell provided by the patient or a non-related donor.
What Are the Different Types of Gene Therapy?
There are two primary types of gene therapy:
- Somatic cell gene therapy
- Germline therapy
Most of the human body is non-reproductive somatic cells. Gene therapy affects only the targeted somatic cells in a patient. Future generations do not inherit alterations in those cells. Germline therapy transforms reproductive cells, such as eggs or sperm, and the results are permanent genetic modifications that impact the genes of future generations.
Somatic gene therapy approaches are in vivo and occur within a living organism, while ex vivo takes place outside the body of a living organism. In fact, in vivo and ex vivo are Latin terms that mean “in the living” and “out of the living.” Virus-like particles (VLP) were developed for vaccine products and are now gaining popularity in gene therapy applications and drug delivery vectors to supplement the absence of or a defective form of essential proteins. Depending on their complexity, VLPs can be expressed in either microbial or mammalian expression systems.
Advance Your Gene Therapy Progress with Avantor
Avantor® can help you maintain the integrity and activity of gene therapy products during upstream and downstream purification through the final fill. Choose from a broad range of materials cell culture components for aseptic fluid transfer supporting “closed-system” cell culture, including:
- Cell culture components
- Fermentation media and supplements
- Growth factors
- Reagents and excipients
- Protein purification systems
- Single-use use products for fluid transfer that supports “closed-system” cell culture
- We carry a comprehensive range of products from brands you trust for gene therapy to genomics applications.
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Our Bioprocessing Solutions team can help you choose gene therapy products tailored to your needs to obtain superior results!