Gene therapy is a medical technique that aims to treat disease by modifying the genetic code of an individual. The first gene therapy product was a virus-based vector that targeted only liver cells, but newer methods are now being explored that target both muscle and nerve cells as well. Gene therapies can be used to alter the expression of genes within specific tissuesas well as in entire organs. This technology has been shown to improve outcomes for patients suffering from diseases such as hemophilia and certain types of cancer.
Gene Therapy Has Been Used For More Than A Decade To Treat Various Diseases
Gene therapy has been used to treat many different diseases for more than a decade. It is a way to treat or avoid disease by using DNA or RNA. Dr Brian Blick says that gene therapy can be used to treat genetic diseases, cancer, and other illnesses like HIV/AIDS.
Genetic diseases are caused by mistakes in the genes, which tell the body how to make proteins. If these problems aren’t fixed, they can change how the body works or even kill the person. The goal of gene therapy is to replace bad genes with good ones so that the body can work properly again without any problems caused by the mutation that caused the disease.
Production And Delivery Issues Have Hampered Gene Therapy Technologies
Even though the idea of using gene therapy to treat pain is exciting, it is a complicated process that has been slowed by several problems. Making the product is hard and expensive, and getting the gene therapy product to the cells that need it can also be hard. As this technology is being studied and improved, Dr Brian Blick agrees that side effects are also a worry.
New Methods Are Overcoming These Challenges, Advancing Gene Therapy
Gene therapy for pain relief is a relatively new field, but it’s making huge strides. There are still many challenges to overcome, but new methods have been developed that will allow gene therapy to progress even further.
These advances include:
- New methods of delivery.
- New understanding of human genetics and how genes work within the body.
Novel Gene Delivery Vectors May Improve Efficiency And Reduce Side Effects
One of these advances is the use of new vectors to deliver genes, which could make the process more effective and lower unwanted side effects. RNA interference, which is also known as RNAi, is an example of a new carrier. It works by putting short RNA molecules into cells, which can stop genes from being turned on. The adeno-associated virus is another example. This virus has been used to send genes to treat diseases like hemophilia B and Parkinson’s.
Human Genetics And Gene Function Have Improved Gene Therapy
In addition to the progress made in gene therapy, we have also learned a lot about human genetics and how genes work in the body. Both of these things have helped the field a lot. Through genetic engineering, new drugs and treatments for illnesses like cancer, diabetes, and heart disease have been made. Because it has worked so well, genetic engineering is now used in several types of medical research:
- Gene therapy: changing the DNA sequence of an organism to treat or avoid disease.
- Gene editing: changing specific genes by hand with high-tech tools or zinc finger nucleases.
Stem cell research: using pluripotent stem cells for medical reasons.