Gene Therapy
The CRISPR DNA editing has many jobs, one of the biggest being gene therapy. CRISPR has been tested and used to cure many different diseases such as Cystic Fibrosis, Sickle Cell Anemia, and hemophilia. While not available yet, scientists have also been working with CRISPR to see if it can cure diseases such as cancer, Heart Disease, HIV and different forms of mental illness. Though the gene editing tool is far from perfect, CRISPR could eventually be used to solve many of the medical mysteries we know today.
What Diseases Will CRISPR Help?
Cystic Fibrosis
Cystic Fibrosis is a condition that occurs in the lungs and digestive system. People with CF have a mutation in the Cystic Fibrosis trans membrane conductance regulator (CFTR) and therefore can not produce functional CFTR proteins. This causes a build up of mucus in the lungs and can block the airways. When inserted, the CRISPR CAS-9 protein complex can be used to replace the mutated gene with the correct sequence, allowing cells to produce CFTR proteins, curing people of Cystic fibrosis.
Sickle Cell Anemia
Sickle Cell anemia occurs because of a beta globin mutation located on the eleventh chromosome. The mutation causes hemoglobin proteins to attach together and create a flatter, less flexible, red blood cell shaped like a crescent. Because of i ts shape, the mutated red blood cell can block blood vessels and prevent blood flow through out the body. Using CRISPR, Scientist can fix the beta globin mutation and produce healthy red blood cells, thus curing sickle cell anemia.
Hemophilia
Hemophilia Is caused when a persons blood can not clot properly, thus causing uncontrollable bleeding. A mutation on the X chromosome causes a person to not make specific proteins called clotting factors. Without clotting factors, blood can not clot, and a someone affected could die from loss of blood. By using CRISPR to cut out the mutation and repair it using the correct sequence, people suffering from hemophilia can start producing clotting proteins.
Cancer
While still in its early stages, CRISPR could also be used to cure different types of cancers. In general, Cancer is caused by a mutation that allows a cell to move through mitosis at a rapid speed and keep reproducing and growing. A protein called cyclin regulates mitosis and the stages in which a cell moves through during reproduction. if a signal to turn off cyclin is never sent, a cell will continue to move through the cell cycle creating many mutated cells very fast. This process creates a tumor. By inserting CRISPR, scientist may one day be able to "turn off" cyclin production and disable the genes associated with it.
Heart Disease
Heart disease is cause by the blockage of arteries, lack of blood follow through the heart, heart failure, and many more factors. There are many different forms of heart disease, but the most common are coronary artery disease, high blood pressure, cardiac arrest, congestive heart failure, arrhythmia, peripheral artery disease, stroke, and congenital heart disease. People who have heart disease suffer from life threatening health issues and could easily die. CRISPR could one day be used to help lower cholesterol rates helping those who suffer from heart disease.
HIV
Human immunodeficiency virus (HIV) is a sexually transmitted disease that is spread through blood and semen. When first contracted, the virus only causes flu like symptoms such as a fever, cough, and fatigue. Though untreated, HIV can lead to AIDS which causes weight loss, fever, cold sweats, and recurrent infections. Overall, HIV suppresses the body's immune system and prevents it from fighting off infections. If left untreated the person infected will most likely die. When inserted, CRISPR can turn off the specific genes HIV uses to replicate and stops HIV from reproducing.