Lentiviral Vector Development for X-linked Chronic Granulomatous Disease
Creative Biolabs' highly efficient and rigorous team of scientists is ready to provide professional technical support for your research. By continuously improving our research and development capabilities, we have established the world's leading lentiviral vector technology platform, which can be used to develop specific vectors based on the treatment needs of different diseases. The gene therapy of X-linked chronic granulomatous disease (CGD) has always been one of the areas we valued. No matter what difficulties you have encountered in the relevant research work, you can contact us to get your own customized service.
Introduction of X-linked Chronic Granulomatous Disease
GCD is a rare genetically heterogeneous primary immunodeficiency disease caused by defects in the nicotinamide adenine dinucleotide phosphate (NADPH) oxidase complex. This disease can cause the patient's phagocytic cells do not normally produce superoxide and mediate the killing of intracellular microorganisms. And patient is more susceptible to chronic inflammation and bacterial or fungal infection.
Among all the mutations that cause CGD, the most common is X-linked CGD caused by mutations in the CYBB gene, which accounts for more than 70% of the overall diseases. This gene encodes the key transmembrane protein gp91-phox in the NADPH oxidase complex. To date, there are more than 700 different CYBB gene mutations recorded in the database. The treatment method of CGD is mainly based on antibiotics and antifungal drugs. Bone marrow transplantation (BMT) can cure CGD, however, this method has higher requirements for transplant recipients and donors, and this specific process of treatment is relatively complicated, moreover, the costs and risks make it cannot be easily universalized. Therefore, the development of gene therapy technology will make it the first choice for the treatment of CGD and other related gene defects in the future.
Figure 1. Chronic granulomatous disease.
Gene Therapy for X-linked Chronic Granulomatous Disease
In-depth research in molecular biology has led us to a more comprehensive understanding of the CYBB gene encoding the gp91-phox subunit, making it possible to cure X-linked CGD by gene therapy. The specific treatment program is to extract some blood-making cells from the patient using a technique called apheresis, and then uses a special virus-retrovirus to transfect the normal CYBB gene into the cells, so that these blood-making cells can produce normal gp91-phox subunit. In this process, treated patients require a small dose of busulfan chemotherapy, but the risk is much lower than traditional allogeneic BMT treatment. Finally, when we get the genetically repaired cells, we can re-implant them into the patient.
Lentiviral Vector Development for Gene Therapy
The emergence of lentiviral vector technology and its successful application in the field of gene therapy is a breakthrough in human life and health. The ability of this vector to infect undifferentiated or terminally differentiated cells greatly expands the target of disease that can be used for gene therapy. In addition, lentiviral vectors increase the efficiency of gene transfer to target cells, reduce the difficulty of the transfection process, and reduce the need for in vitro cell manipulation, making the gene therapy safer and more efficient. Therefore, the development and optimization of lentiviral vectors will become a technical means that needs to be paid more attention for gene therapy in the future.
Creative Biolabs provides lentiviral vectors for X-linked CGD gene therapy according to customer needs through rigorous quality control. We will optimize the vector to make it more genetically stable and can be permanently integrated into the genome of transduced cells to provide long-term stable gene expression in vivo or in vitro. If you have any questions or have any difficulties, you can contact us by email or send us an inquiry to find a complete solution.
