This is the third blog post in the Biopharmaceutical Basics series.
Sometimes the whole or part of a gene is defective or missing from birth. This is typically referred to as a genetically inherited mutation.
In addition, healthy genes can change (mutate) over the course of our lives. These acquired mutations can be caused by environmental exposures. The good news is that most of these genetic changes (mutations) do not cause disease. But some inherited and acquired mutations can cause developmental disorders, neurological diseases, and cancer.
Depending on what is wrong, scientists can do one of several things through gene therapy.
What is gene therapy?
Gene therapy is an evolving strategy for disorders caused by a missing or faulty gene and may involve addition, inhibition, editing, or functional replacement of a gene.
Gene therapies can be one-time treatments and are designed to target the genetic root cause of diseases.
How does it work?
Gene therapy can be used to modify cells inside or outside the body. When gene therapy is used to modify cells inside the body, a doctor will inject a vehicle called a “vector,” carrying the gene directly into the patient.
When gene therapy is used to modify cells outside the body, doctors take blood, bone marrow, or another tissue, and separate out specific cell types in the lab. The vector containing the desired gene is introduced into these cells. The cells are later injected into the patient, where the new gene is used to produce the desired effect.
There are four types of gene therapy:
Gene replacement – Gene replacement uses a new, working gene to replace the function of a nonworking or missing gene. This gene then provides the instructions for the body to make the missing or insufficient protein.
Gene addition – Gene addition involves the introduction of a new gene into the body to target a specific aspect of what causes the disease.
Gene inhibition – Gene inhibition involves deactivating or “silencing” the expression of a mutated or faulty gene that codes for a toxic protein or too much protein.
Gene editing – Gene editing corrects the mutation in a gene that is causing disease.
Why is gene therapy important?
Advances in genetics and bioengineering have enabled the development of gene therapy and, with it, the unprecedented potential to treat diseases caused by recessive gene disorders, acquired genetic diseases, and some viral infections. Technologies for manufacturing and analysis of gene therapy products are evolving with continued efforts to be fully optimized.
What solutions are available?
With the right analytical tools, scientists can expedite the development of safe, high-quality gene therapy products while reducing time to market and costs of manufacturing.
Solutions from Thermo Fisher Scientific are evolving, right along with the gene therapy advancements themselves. Our robust assays are tailored for the analysis of gene therapy products to enable fast results with better accuracy and reproducibility to facilitate the development of your gene therapy product and accelerate its progression to market. There are several recombinant DNA technology protocols, in which the gene-of-interest or healthy gene is inserted into a vector, which can be a viral, plasmodial, or nanostructured.
Messenger RNAs (mRNA) therapies — which upon introduction to the target cell are translated into desired proteins — are being explored in the use of personalized immunotherapy.