This blog will discuss the topic of CRISPR, a gene editing drug for sickle cell disease and beta thalassemia. The blog will also entail the disadvantages as well as the advantages of the new therapy.

What is sickle cell disease and beta thalassaemia?

Sickle cell is an inherited blood disorder that affects red blood cells. It causes red blood cells to turn into a sickle-like shape, which reduces the ability to transport oxygen because it clogs the vessels.

Thalassaemia is an inherited blood disorder that affects haemoglobin. Beta thalassemia doesn't produce enough haemoglobin so patients with the inherited disorder require frequent blood transfusion.

What is CRISPR and how does it work?

It's one of the first therapies to take a noble prize award despite its new arrival. Gene-editing therapy manipulation of DNA. The therapy targets genes that produce haemoglobin, the protein that carries oxygen in the blood cells. The therapy directs an enzyme called Cas9 to a gene called BCL11A in the bone marrow that prevents the body from making a form of haemoglobin found only in fetuses. The new therapy consists of removing a person's bone marrow stem cells editing them and untreated bone marrow is destroyed and reinfusing the edited cells.

How effective is the treatment?

According to Reardon S, trials are still ongoing and 29 out of the 30 study participants with sickle cells have had no pain for up to a year and 39 out of 42 beta thalassemia patients no longer needed blood or bone marrow transplants for one year after the therapy. Despite it being a new treatment participant have reported positive feedback. This is seen with Jimi Olaghere who stated ‘it's like being born again’ according to BBC news.

Overall, CRISPR gene editing therapy treatment is seen to be effective. However, despite its praises it does have a major disadvantage. One being the price per person making it not accessible for many people. As stated by NHS, sickle cell disease affects people of African origin and thalassaemia affects people of south Asia. The gene editing therapy isn't accessible for everybody as there are 2 bases one in the US and UK. The trials of the therapy are said to be one off, which defeats the purpose of it being for the people to better their lifestyles.

Written by Glanelle

Moderated by Adelene

References:

NHS (2019). Overview - Thalassaemia. [online] NHS. Available at: https://www.nhs.uk/conditions/Thalassaemia/

NHS (2019). Sickle Cell Disease. [online] NHS. Available at: https://www.nhs.uk/conditions/sickle-cell-disease/

Park, S.H. and Bao, G. (2021). CRISPR/Cas9 gene editing for curing sickle cell disease. Transfusion and Apheresis Science, [online] 60(1), p.103060. doi: https://doi.org/10.1016/j.transci.2021.103060

Reardon, S. (2023). FDA Approves First CRISPR Gene Editing Treatment for Sickle Cell Disease. [online] Scientific American. Available at: https://www.scientificamerican.com/article/fda-approves-first-crispr-gene-editing-treatment-for-sickle-cell-disease/"