crispr

Rebecca Cavallaro discusses the recently approved gene editing therapy called CRISPR. CRISPR studies how bacteria's immune system fights viruses by removing foreign DNA. It uses a protein called Cas9 and a guide RNA to recognize and edit specific DNA sequences. The FDA has approved CRISPR for treating sickle cell disease, where patients' cells are edited in the lab and returned without the defect. Studies show that patients no longer experience painful crises. This therapy has the potential to revolutionize healthcare and improve lives. Hi, my name is Rebecca Cavallaro. Today I'm going to be talking about something that is very interesting to me and on Monday was actually just approved by the FDA for the first time. I'm going to be talking about gene editing based therapy more known as CRISPR. So the first how it started was two ladies were studying this, one from the US and one from France. They won a Nobel Prize in 2020. CRISPR is basically studying how the virus affects bacteria, observed how certain strand of bacteria developed in a primitive immune system to fight the virus. It is noticed how the strain of bacteria had a protein when edited its genome and removed the foreign DNA sequence of the virus, the protein CRISPR gene. The virus inserts, any virus that we have inserts its DNA sequence into the host DNA synchronous. So every virus is either the DNA or an RNA and the primitive protein cuts it out. So CRISPR has two components. The first is the Cas9 protein that can cut out DNA and a guide RNA that can recognize a sequence of DNA to be edited. First it recognizes the sequence of DNA that is causing the health problem. Then it creates a specific RNA to recognize that strand of DNA and then the guide RNA attaches itself to the Cas9 DNA protein and then that component is attached to the target cells. It locates the target letter sequence and cuts out that part of the DNA. It's like a cut and paste tool and this is hopefully can have critical advances in patient care and in cure lifelong inherited diseases. So on Monday, it was the first time that the FDA approved the CRISPR for the sickle cell disease. So this is a very it's a very sad disease that patients have and it takes their patients own cells to the lab, edit the cells and give them back their cells without the defect or with the defect corrected. So the patients no longer have the painful crises called VOCS and it's very painful. The studies show that patients who undergo this therapy no longer have this the VOCS. It's a very precise gene editing and there have studies showing that four years of patients that they've done this therapy to they have no no effects of the gene editing. This has been something that has been interesting to me. Both of my parents are in the medical field and I believe that this is there's been a lot of talk about this and a lot of opinions, but I believe personally that this is going to be something that's very going to be innovative in healthcare in the future and I'm excited to see how this integrates in our healthcare system and improves people's lives. Thank you.