Lentiviral Vector Development for Fanconi Anemia

Through continuous testing and long-term research, Creative Biolabs' experienced team has now launched a lentiviral vector (LV) development platform to offer various LV services for gene therapy for Fanconi anemia (FA). These services not only make breakthroughs in the biotechnology industry, but also complement our platform to meet more customers' requirements. So, no matter what difficulties you have encountered in the relevant research work, you can contact us to get your customized service.

Introduction of Fanconi Anemia

FA is a genetic disease which caused by mechanism defection in repairing damaged DNA. Mutations in at least 15 genes will cause FA, and the proteins encoded by these genes are involved in a cellular process called FA pathway associated with abnormal DNA repair. This DNA repair process is mediated by a combination of eight FA-related proteins to form FA core complex to activate both FANCD2 and FANCI proteins, and therefore allows DNA repair proteins enter the interstrand cross-link (ICL) region to eliminate ICL and continue DNA replication. Topically, 80% to 90% of FA diseases are caused by mutations in three genes, FANCA, FANCC, and FANCG. The proteins encoded by these genes instruct the formation of FA core complexes. When they are unable to function normally, the FA pathway is blocked, and eventually leads to the accumulation of ICL, causing various diseases. Patients may have symptoms of bone marrow failure, physical abnormalities, organ defects, and increased risk of cancer. The most common symptom is bone marrow failure. In general, 90% of bone marrow function is affected, resulting in aplastic anemia with reduced blood cell production.

Figure 1. Fanconi anemia.

Gene Therapy for Fanconi Anemia

At present, the treatment for patients with FA is mainly to supplement the missing blood cells of patients by using drugs or blood transfusions. It is feasible to cure FA by transplanting healthy human bone marrow, but this method has relatively high risk and is not suitable for everyone. Bone marrow transplantation itself is also a very dangerous operation.

Therefore, the use of gene therapy to cure FA is currently the best solution. The patient's bone marrow stem cells are corrected by using the modified virus to transfer the normal gene to the FA patient without causing infection. Firstly, patients receiving treatment will take some special growth factors to transfer blood-forming stem cells from the bone marrow to the blood. Adults can also use drugs that increase the production of stem cells in the peripheral blood. Secondly, after collecting enough stem cells, the cells are treated with a lentiviral vector carrying a normal gene in the laboratory to correct the loss-of-function genes. Finally, these repaired cells are periodically introduced into the patient's body to cure these hereditary diseases.

Lentiviral Vector Development for Gene Therapy

The use of lentiviral vectors in FA gene therapy will significantly increase the success rate of treatment and minimize the potential risks. In the existing cure examples, the patient receiving the treatment is not only in a stable improvement, but also presents no sign of any viral gene insertion. Creative Biolabs is committed to providing you with a lentiviral vector construction service that allows you to get more efficient transfection, and more stable lentiviral vectors. 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.