What Diseases Have Been Cured by CRISPR?

Diseases Cured by CRISPR: Breakthroughs in Gene Editing

Clustered Regularly Interspaced Short Palindromic Repeats (CRISPR) is changing the way of treatment. It enables scientists to cut and edit DNA with high accuracy. Ever since its discovery, it has shown great potential for curing genetic conditions. Hence, understanding about the diseases which have been cured by CRISPR is essential.

How Does CRISPR Work?

CRISPR works like molecular scissors, which allows scientists to edit DNA at particular locations. It uses a guide RNA to locate a targeted DNA sequence and enzymes to cut the DNA at that exact spot.

After the DNA is cut, cells naturally repair the break. Scientists and researchers use this process to either remove a faulty gene or insert a corrected version of the gene. This kind of method is faster and more accurate than older gene-editing tools.

What is CRISPR gene editing used for?

CRISPR gene editing has a wide prevalence in medical research for curing genetic disorders, such as sickle cell anaemia and beta thalassaemia. In agriculture, it enhances crop resistance and yield. Scientists also use CRISPR to study gene functions and develop potential cures for cancer therapies.

Its accuracy and efficiency have now revolutionised genetic engineering. CRISPR offers promising solutions in healthcare, food security and biological research.

List of Diseases Effectively Treated by CRISPR

While CRISPR is not widely available, clinical trials using CRISPR have led to functional cures for certain patients. Here are a few diseases where CRISPR has made the greatest impact so far:

Sickle Cell Disease

Sickle Cell Disease (SCD) was one of the first conditions to be treated by CRISPR. It is a blood disorder which causes the red blood cells to become misshapen. It can result in pain, infections and organ damage.

In 2019, researchers used CRISPR to treat a patient named Victoria Grey. Doctors used CRISPR to edit her bone marrow cells, disrupting a gene that suppresses foetal haemoglobin production. This allows the cells to produce foetal haemoglobin, which prevents sickling.

After treatment, her symptoms improved dramatically. She stopped requiring blood transfusions, and the pain episodes also dropped. Since then, more clinical trials have shown similar results in other patients as well.

Beta Thalassaemia

Similarly, like sickle cell, beta thalassaemia affects haemoglobin. Patients with this condition cannot make healthy red blood cells and require frequent blood transfusions.

Since 2019, landmark clinical trials have used CRISPR to edit bone marrow cells in patients with severe beta thalassemia and sickle cell disease. In these trials, many patients, including the first reported case, have produced enough healthy red blood cells to stop requiring transfusions entirely. 

List of Diseases that CRISPR Can Cure

CRISPR is transforming medicine by targeting the genetic roots of many inherited and infectious diseases. It ranges from rare disorders to widespread illnesses. The revolutionary technology of CRISPR offers real hope for long-term cures. Here is a list of diseases that CRISPR could potentially cure in the future:

• Inherited Blindness : CRISPR offers a promising solution for certain forms of inherited blindness, like Leber congenital amaurosis. These conditions stem from a single gene mutation which affects the retina. By editing the faulty gene directly with retinal cells, CRISPR may restore partial vision or prevent any further escalation of the condition.
• Cystic Fibrosis : Cystic fibrosis is a life-threatening condition. It occurs due to mutations in the CFTR gene. It affects the lungs and digestive system. CRISPR could potentially correct this mutation at the DNA level and offer a permanent solution rather than symptom management. Scientists are now developing methods to deliver the gene editor directly to the affected lung tissue. While research is going on, a successful application can significantly improve life expectancy and reduce the need for intensive daily treatment.
• Huntington's Disease : Huntington’s disease is very fatal. It is an inherited brain disorder which happens due to a genetic mutation that leads to progressive nerve damage. CRISPR offers hope by targeting and removing the faulty gene that is responsible. Researchers are now exploring whether editing brain cells early on in the disease can slow its progression.
• Cancer : CRISPR is being researched to enhance next-generation cancer treatments, particularly by improving engineered immune cells (CAR-T cells). Early-stage clinical trials are testing CRISPR-edited CAR-T cells for blood cancers like leukemia. CRISPR can also help identify and eradicate the cancer-driving mutations in tumours.
• HIV/AIDS : Extremely early-stage research is exploring whether CRISPR could one day be used to edit immune cells to resist HIV or to cut the HIV genome out of infected cells. This remains a theoretical concept facing significant scientific and delivery challenges and is not a current treatment. This kind of approach goes beyond the current antiretroviral treatments, which manage but do not cure the virus.

An application of CRISPR in the medical field has not only shown tangible results in curing certain diseases but also made rapid progress in tackling conditions like sickle cell anaemia and beta thalassaemia. As the trials expand and technology improves, CRISPR can redefine what is possible in modern medicine shortly.