You’re reading a story. You get to the climax. Katniss is about to risk her whole life for Peeta in District 12.
You’re sitting on your couch. You pray and pray that she comes to her senses and just stays in her hut surviving with the food she already has.
You turn to the next page.
Your whole world just ended. You wish you could have created your own ending to the story.
Fortunately, this incident happened in an alternate universe made up by a director that probably deleted this ending thousands and thousands of times to make sure the story had the perfect ending, just the right one to screw you over.
Turns out, scientists have the same power as directors.
Through the field of gene-editing, scientists are now able to delete parts of the genome that are harmful to the patient and that can result in further complications.
The question is, how is this done?
CrisprCas9 is a technology that enables researchers to add, remove, or alter sections of the DNA sequence. The RNA binds to the Cas9 enzyme. The modified RNA is then used to identify the DNA sequence that needs to be changed. The Cas9 protein binds at the part that needs to be cut off and cuts this portion of the DNA.
If we now have the power to delete sections of our DNA, then don’t we have the power to delete certain diseases as well?
The answer is, yes.
Sickle cell anemia is a disease that affects approximately 100 000 Americans. It occurs in about 1 out of 365 African-American births.
Treatments and therapies have been discovered, but we are still waiting on a cure. This is one of the biggest areas where I, and Jennifer Doudna, a.k.a the pioneer of this technology, think CRISPR-Cas9 has potential.
Sickle cell anemia is caused by a mutation in a person’s hemoglobin. This mutation is called HBB, or Hemoglobin Subunit Beta.
The problem with this mutation in hemoglobin is that it causes the red blood cells in your body to turn into a “sickle” shape. This change in shape is inefficient for your red blood cells because this prevents them from flowing through your blood vessels effectively.
However, fetal hemoglobin is a natural protector for sickle cell anemia that is created by your body. The only problem is, as you grow older, you start to make less and less of it. Therefore, editing your DNA to produce more fetal hemoglobin would allow the patient to outnumber the amount of sickled red blood cells in their body with healthy ones.
This is where CRISPR-Cas9 comes into play.
We can take the patient’s hematopoietic stem cells, which produce both red and white blood cells, and use CRISPS-Cas9 to edit the gene to produce more fetal hemoglobin.
Companies like CRISPR Therapeutics are currently working on doing this.
That shows you that this is legit.
Their project CTX001 is going through clinical trials right now, and is predicted to be completed by May 2022.
Gene-editing has the ability to cure diseases and problems that have been looked at for years and years. It has given us the ability to solve many problems, that also raise ethical issues as well. This is where we encounter setbacks in the process.
If you would like to discuss more about these ethical issues, or talk more about sickle cell anemia, please don’t hesitate to shoot me an email at email@example.com or connect with me on LinkedIn!