Gene therapy is designed in the process of adding new genes to one’s cells to replace malfunctioning or missing cells.
If a mutated gene leads to the formation of a faulty gene, gene therapy can be used to introduce a normal copy of that specific gene so as to restore the normal function of the protein.
The main difference between cells and genes is that cells are just the basic building blocks of every living organism. The human body is composed of millions of cells. Within each cell, we have thousands of genes that relay the information of production of particular enzymes and protein that make bones, muscles and blood that supports all our body functions such as growing, making energy, and digestion.
How genes therapy works
A gene can mutate or change when you are an adult, or sometimes part or whole gene can have complications or miss right from birth. Any of such changes can alter how the protein will be made, which at the end can lead to serious problems and diseases.
Under gene therapy, doctors can solve such situations by replacing a gene that is defective with another one or turn of the gene that is causing medical problems. Inserting a new cell to your body is done by a process called a vector.
Gene therapy procedures can be utilized to change cells outside or inside your body. If it is inside, a doctor injects a vector that has the gene straight into the part of your body that contains a defective cell.
For the case of gene therapy that is done outside the blood, body, bone marrow, the vector containing the genes is introduced to the cell and left to multiply in the laboratory. They are left to multiply and then injected into the patient.
Gene therapy and sickle cell treatment
Gene therapy has been widely used to treat sickle cells. Gene therapy for sickle cell involves an infusion of the autologous bone marrow transduced with a lentiviral vector that has short hairpin. Gene therapy has been experientially used to treat people with sickle cell diseases that affect more than 100 000 Americans. The results show that gene therapy can be used to treat sickle cell anemia. In the future, gene therapy will be used to treat people who suffer from severe diseases like cancer.