Role of Gene Therapy in Drug Discovery and Development

contract lab service
contract lab service

Medical science is constantly evolving, and researchers are developing innovative ways to treat certain diseases and health conditions. Gene therapy is a promising treatment option with the potential to revolutionize the way we currently treat diseases. This therapy involves securely delivering genetic materials to human body cells to successfully replace, modify, or supplement a damaged or non-working gene. Gene therapy has shown impressive potential for treating various diseases, like infectious diseases, genetic disorders, and deadly conditions, like cancer.

It has enabled researchers and medical experts to use this therapy for drug discovery and development. Many drug manufacturers already use gene therapy through contract lab services to manufacture effective drugs for deadly diseases. Gene therapy is a vital tool for identifying newer therapeutic targets, making it easier to develop newer treatment options. Scientists are using it to understand the genetic base of different diseases that contribute to a particular disease. If you want to learn more about gene therapy’s role in drug discovery and development, read along.

Gene Therapy: Its Role in Drug Discovery & Development

Genes stored in our body cells contain DNA responsible for most bodily functions and forms. From regulating your different body systems to helping you grow taller- everything is controlled by your DNA. When genes stop functioning, they give rise to numerous diseases. It is where gene therapy can help by entirely replacing or modifying faulty/damaged genes. This replacement or modification is done to strengthen your body’s disease-fighting power, allowing you to cure disease.

Although researchers are still examining and finding ways to use this therapy in drug discovery and development, it has established a prominent place in the process. It is being used in contract lab service centers for the following reasons.

  • Identifying New Therapeutic Targets

Researchers can use gene therapy to deduce new therapeutic targets by precisely comprehending the genetic base of different diseases. The process involves studying genetic mutations and genes associated with a particular disease being studied by researchers. For example, when researchers identify a faulty gene(s) causing cystic fibrosis by identifying the genetic base of this disease, they get closer to finding an effective cure. Using gene therapy, the faulty gene can be replaced with a functional one, ending the disease.

  • Developing Better Drugs

Gene therapy has been proven effective in enhancing the overall efficacy of drugs. It achieves this stage by modifying the gene expressions actively involved in drug transportation and metabolism. There are numerous genetic editing tools, like CRISPR, used by researchers to alter DNA cells. Scientists can effectively use such gene editing tools to boost the genetic expression responsible for metabolizing a drug, making it much more effective. Gene therapy also assists in creating medicines that can help target particular body tissues or cells while minimizing the potential side effects.

  • Creating Personalized Medicines

Developing personalized drugs has become easier because of gene therapy. Scientists can now target specific mutations in a patient and use them to create highly effective medicines for the person. The process involves deeply analyzing a person’s DNA present in body cells. Once identified and analyzed, gene therapy can be used to target the said mutations through a therapeutic gene.

For example, if a particular patient has been diagnosed with cancer, healthcare experts can use gene therapy to target the specific mutations present within cancerous cells and develop personalized treatment methods. It may involve taking the help of a contract lab service to develop a highly custom drug, but it is possible. A customized drug will significantly boost the treatment’s success rate. Researchers are using gene therapy to create various personalized drugs that can be used to treat a similar problem faced by numerous people.

  • Enhancing Drug Safety

One of the best benefits of using gene therapy in drug development is its contribution to making the drug safer and more effective. This therapy can modify genetic expressions that create a particular disease or health disorder. When scientists identify the exact gene(s) responsible for creating an illness or disease, they modify the drug to target that particular disease’s genetic base. The potential side effects are also considered during the process, and efforts are made to minimize any potential harm. As a result, better drugs are created with high efficiency and low side effect rates.

  • Assist in Drug Delivery to Specific Tissues/Cells

Another contribution of gene therapy in drug discovery and development is helping researchers efficiently test the drug during the development stage. Once a drug prototype is created, it must be tested to determine its effectiveness. It is where gene therapy comes into the picture. This therapy helps deliver the developed drug(s) to the target body cells and tissues. Precise delivery is achieved using viral vectors engineered for particular body cells. For example, scientists created a viral vector, especially to target cancer cells. This vector can now be used to deliver a fully-functional or therapeutic gene to the cancer cells, prompting them to self-destruct.

Gene Therapy is the Future

Irrespective of the disease or illness a person gets, they can be attributed to certain gene malfunctions. Once the faulty gene gets fixed, the health problem disappears. Gene therapy has emerged as a miraculous tool for treating health conditions by replacing or repairing mutated genes. The therapy is also used in contract lab service centers to improve drug identification and development. This therapy is also being used in clinical trials to treat different diseases, and its results are promising.