How CTRL-C, CTRL-V Saves Lives

Have you ever wondered how we humans can alter our defining characteristics as per individual liking or eliminate genetic combinations that are the root cause of incurable, life-threatening diseases? Well, what once felt like fiction of mind is now emerging as a revolutionary, blooming technology named CRISPR. A gene-editing tool allowing scientists to simply transmute DNA sequences and modify gene function, it has a myriad of potential applications, including correcting genetic defects, treating and averting the spread of diseases, and improving the growth and immunity of crops.

Several diseases coming under the umbrella of genetic disorders are caused by an abnormality in an individual’s genetic makeup. The genetic abnormality can range from minuscule to major -- from an isolated mutation in a single base within the DNA of a single gene to a whole chromosomal abnormality which may even lead to the addition or subtraction of an entire chromosome.

The factor that adds to the severity of such disorders is that these are generally irremediable and can’t be cured by any pharmaceutical medications, in such cases the CRISPR genome editing technology comes to the rescue of such patients. It has particularly made several recent advances in the treatment of genetic blood disorders like beta-thalassemia and sickle cell anemia.

Focusing our attention on sickle cell anemia (also referred to as SCD-Sickle Cell Disease) we learn that the conventional treatment procedures solely addressed the symptoms and didn’t provide any lasting, persistent cure. However, CRISPR-Cas9 has demonstrated an immense potential to cure the underlying genetic cause of the disease. A new gene therapy, CTX001 has been licensed by Vertex pharmaceuticals along with CRISPR Therapeutics. Patient data from its clinical trials have shown promising results in patients. Victoria Gray was the first person to be treated for SCD using this therapy and in July 2020 it was announced that her disease had shown significant improvement with fetal hemoglobin levels shooting up and bouts of pain disappearing. More significantly, the technology liberated her from the need of undergoing periodic blood transfusions. Despite the strides it has made in the medical field, CRISPR still has a long way to go.

Evaluating the safety and efficacy of the CTX001 in patients is key to gaining the trust of more patients who are reluctant to receive gene-editing treatments.

Along with genetic disorders, the CRISPR technology has also emerged as a ray of hope for patients suffering from cancer, Alzheimer’s disease, HIV, autism, muscular dystrophy, and inherited eye disorders, heralding the advent of the era of transformative medicine.

Bibliography

1.https://clinicaltrials.gov/ct2/show/NCT03745287

2.https://www.synthego.com/blog/crispr-cure-diseases

3.https://www.synthego.com/blog/crispr-cure-diseases

4.https://www.youtube.com/watch?v=4YKFw2KZA5o Written by: Sehreet Kaur