What Disease Can Be Treated With Gene Therapy?

Getting diagnosed with a lethal disease completely shambles one’s willpower. To prevent this, scientists came up with a miracle called gene therapy. This can add more years to your life!

Gene therapy manipulates the construction of genes to treat medical disorders. The faulty gene is either repaired or replaced with a much healthier version. This way genetic conditions can be cured directly.

Most importantly, you have to have knowledge about the blueprints of your gene and how editing them can affect your health. Nowadays many health clinics are introducing gene therapy to their system for helping people with a more comfortable future.

A wide variety of diseases can be treated thanks to gene therapy. We have collected data from healthcare professionals to present you with possible success stories. So tune in!

What Possible Diseases Can be Treated with Gene Therapy?

The ailments targeted by gene therapy are often complex. It takes immense patience and relentless efforts for researchers to come up with these possible techniques. Some of the diseases currently treated by gene therapy are:

Sickle Cell Anaemia

Donut-shaped red blood cells are usually considered healthy. But sickle cell anemia modifies them to a more abnormal C-shaped structure. These irregularly shaped cells often get trapped while passing through your blood vessels. They also clump up and cause oxygen deficiency in your bloodstream.

Gene therapy helps to edit these faulty cells with a tool called CRISPR-Cas9. This tool snips off the damaged parts of your sickle cell and switches them with a better one.

Another approach is activating your dormant genes. Fetal hemoglobin is replaced with adult hemoglobin once a baby is born. So gene therapy turns on your inactive fetal hemoglobin to establish a balance with your contaminated adult hemoglobin. This causes the emergence of healthy red blood cells.

Hereditary Blindness

Degenerative eyesight impairment can be cured using gene therapy. This causes a gradual loss of your light-sensing cells leading to blindness. Your retina is properly shielded from the immune system. It makes it a safe spot to perform surgery.

A gene therapy called Luxterna is used to treat patients with mutations in their genes. Luxterna assists our eyes to respond to light by encoding retinal proteins. Missing proteins can be made up by adding a healthy copy of your mutated gene. It means providing the right proteins can make up for your vision loss.

To deliver the proteins you need vectors like AAV (adeno-associated virus). It is injected into both your eyes for repairing your genes. This is successful in restoring your vision.

Severe Combined Immunodeficiency (SCID)

Severe Combined Immunodeficiency comes in a pack. This is caused by damaging your infection-fighting immune cells. It makes people extremely sensitive to even minor infections. In fact, it is more common in infants. Also, it cannot be treated by injection doses as your body becomes susceptible to low-strength viruses present in the shots.

Gene therapy can effectively correct the root cause of your SCID. Here, retroviruses are used as they can pierce through cells. The viruses are injected with a healthier copy of the faulty gene which is then delivered to the affected area. Damaged cells from the SCID patient are then immunized.

Side effects like leukemia are slowly reduced. This means our immune system can be reinforced using this technique.

Crohn’s disease

Crohn’s disease primarily swells the gastrointestinal tract. It sometimes develops other passageways between our gut and the anus. This causes major complications as waste materials that are supposed to be ejected from our body enter our gut. As a result, the excrement of feces becomes extremely difficult.

To solve this problem gene therapy delivers anti-inflammatory proteins to the inflamed parts of the GI tract. This reduces the swelling and creates an immune response. Again the immune response can be altered using gene therapy. It helps in maintaining a perfect immune equilibrium.

Not only that, gene therapy promotes the growth of new gut cells to replace the deformed ones. This therapy helps people in achieving a healthier gut.

Hemophilia

Hemophilia causes patients to bleed out profusely even with minor cuts. This typically happens as your blood lacks beneficiary proteins that induce blood clots. It makes you lose gallons of blood both internally and externally. This can even lead to death if not taken under control.

A vector usually assists in the delivery of a therapeutic gene to the affected area inside your body. This causes the faulty gene to be replaced leading to reduced bleeding incidents. It means that proper blood clots can now be formed to prevent large amounts of blood from leaving our bodies.

Roctavian is the first gene therapy to cure hemophilia by fulfilling the missing factor. This treatment causes a reduction in bleeding episodes.

Parkinson’s Disease

Parkinson’s disease cause an imbalance in the overall movement stability of patients. It happens as the brain cells gradually lose the production capability of dopamine. This disease causes continuous shaking and trembling of limbs. A lot of times it can lead to immobility in people.

For its treatment, enzymes are usually delivered to the brain cells. Glutamic acid decarboxylase boosts the production of gamma-aminobutyric acid to redo the abnormal circuitry of Parkinson’s brain. Again, three essential enzymes are also transferred to the brain area for the important synthesis of dopamine.

These therapies boost dopamine levels in your brain to make up for the loss done by the disease. This is how the clumsiness of Parkinson’s can be reduced.

Multiple Sclerosis

Multiple sclerosis stimulates impaired signaling of your nervous system. This is related to the brain and the spinal cord which are heavily impacted. Here, your immune system mistakenly attacks the myelin sheets of your spinal cord. This leads to inflammation and as a result damage to the nerves. It disrupts electrical impulses from flowing all over your body.

Gene therapy helps in rebuilding the myelin sheet by providing essential proteins to your spinal cord. Blood stem cell transplant can be carried out to switch the damaged immune cells in our brains. But the damage caused to the body cannot be reversed.

Despite mending the damages, gene therapy helps to reduce fatigue and muscle weakness caused by multiple sclerosis.

Melanoma

Going on a family vacation sounds fun until you forget your sunscreen. UV rays penetrating your skin can lead to skin cancer –  Melanoma is one of them. But it does not stay within its boundary. This cancer can seep into your skin layers spreading cancer all over your body parts.

Melanoma can be removed through the surgery of the tumor or its surrounding affected cells. However, the most popular method is through radiation. Radiation destroys cancerous cells and prevents them from emerging in the future. The radiation beam can be subjected to specific spots containing your cancer while removing them at the same time.

Drugs also reduce cancerous cell expansion during melasma. This causes gene therapy for the treatment of skin cancer.

Mesothelioma

Mesothelioma affects the thin tissue in our internal organs. It is another cancer but this time it affects the internal parts. But it is a much more deadlier version of cancer. It mainly affects your lungs and other moist areas inside your body.

Gene therapy causes the modification of gene structure to reduce the cancerous cells. Usually, a foreign gene is modified in labs outside the body. It is then injected into the cells as suicide genes. The cancerous cells make it programmed in the suicide gene. This causes the demise of the overall cancerous cell along with the foreign gene.

This kind of cancerous cells can be manipulated with the introduction of foreign suicide genes.

Blood Cancer

Overproduction of blood cells leads to blood cancer. This is extremely common and internally shatters a person.

Gene therapy is involved in blood stem cell transplants to cure leukemia and other disorders. This is mainly done using radiation from chemotherapy. Chemotherapy kills off cancerous cells protecting the body from further damage. New modified cells are then injected into the body to make up for the lost ones.  A bone marrow transplant is often the answer.

Chimaeric Antigen Receptor T-Cell therapy is also carried out by reprogramming the patient’s mutated genes. Both of them are highly complex procedures and routine treatments take a long period of time. This is how children and young adults can get over leukemia.

Bottom Line

Gene therapy is a savior to mankind. Rare diseases can be cured with the introduction of new genes and replacing faulty ones. Thanks for tagging along and getting to know more about gene therapy treatments.

In a nutshell, gene therapy took the world by storm by implementing its miracles to save human souls. Diseases like blindness can be cured with simple surgeries. Inherited sickle cell diseases along with hemophilia and Severe Combined Immunodeficiency are slowly taken care of. Multiple Sclerosis and bowel diseases like Crohn’s can be immensely reduced. Jerking caused by Perkinson’s disease is stabilized. Most importantly, cancers like blood cancer, mesothelioma, and melanoma are finally treated using radiation.

Therefore, gene therapy is helping people win their life’s battle.