Robert147iq
Iron
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- May 4, 2026
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hello, this thread will be about editing genes, or more specifically i will be talking about CRISPR and casgevy.
WHAT IS CRISPR?
CRISPR stands for Clustered Regularly Interspaced Short Palindromic Repeats, and in short is essentially scissors made out of a couple molecules.
i will be talking about the CRISPR-Cas12a, which is a class 2, type V CRISPR RNA-guided endonuclease. what is endonuclease? its a class of enzyme that cuts DNA or RNA by breaking the bonds that hold the strand together.
how cas12a functions, is that its guided by RNA to a specific DNA sequence, which it then cuts the DNA, and creates a break so the cells repair system can be used to add, remove, or change DNA. this is very important, because the cell attempts to repair itself, and thats when the gene changes, or edits itself. the scientists could repair itself with a different DNA template, if provided correctly. doing this could potentially cure genetic diseases, or correct specific gene mutations.
WHAT COULD CRISPR DO?
crispr-cas9 is a tool that has transformed cancer research by enabling precise gene editing. this has demystified cancer years ago (slightly), and could potentially cure certain cancers soon. the first person to be treated with CRISPR, was a woman named Victoria Grey, and it was used to help her sickle cell disease. this happened back in 2019, 4 years before the first CRISPR type of therapy was approved. after the therapy was over, it had completely cured Victoria, and she still to this day experiences no pain, and is completely cured of sickle cell.
there have been only 64 patients that have been through casgevy therapy (a type of therapy using CRISPR), and 99% of those patients have been cured, most of them no longer having to be in hospital anymore, and have never experienced pain crises from their disease.
THE RISKS.
the risks of crispr based therapies is that, before the crispr edited cells can be given, the patient must undergo a conditioning treatment with chemotherapy (like busulfan) to wipe out their existing bone marrow. this can cause severe nausea, mouth sores, the risk of life threatening infections, and potential infertility. and because crispr works like molecular scissors, there is a risk that it could make unintended cuts elsewhere in the genome.
will this be more commonly used? most likely yes. casgevy's competition is other gene therapies like lyfgenia, and casgevy costs 2.2 million per course, while lyfgenia costs 3.1 million per course. a near 1 million dollar difference! casgevy is also deemed safer than lyfgenia, because casgevy finds and replaces, or cuts, while lyfgenia relies on gene insertion, which uses a modified virus to carry an entirely new, artificial hemoglobin gene into the cell. describing lyfgenia, in my opinion, sounds way scarier than casgevy, and casgevy will most likely get picked when a patient is given options.
WHAT IS CRISPR?
CRISPR stands for Clustered Regularly Interspaced Short Palindromic Repeats, and in short is essentially scissors made out of a couple molecules.
i will be talking about the CRISPR-Cas12a, which is a class 2, type V CRISPR RNA-guided endonuclease. what is endonuclease? its a class of enzyme that cuts DNA or RNA by breaking the bonds that hold the strand together.
how cas12a functions, is that its guided by RNA to a specific DNA sequence, which it then cuts the DNA, and creates a break so the cells repair system can be used to add, remove, or change DNA. this is very important, because the cell attempts to repair itself, and thats when the gene changes, or edits itself. the scientists could repair itself with a different DNA template, if provided correctly. doing this could potentially cure genetic diseases, or correct specific gene mutations.
WHAT COULD CRISPR DO?
crispr-cas9 is a tool that has transformed cancer research by enabling precise gene editing. this has demystified cancer years ago (slightly), and could potentially cure certain cancers soon. the first person to be treated with CRISPR, was a woman named Victoria Grey, and it was used to help her sickle cell disease. this happened back in 2019, 4 years before the first CRISPR type of therapy was approved. after the therapy was over, it had completely cured Victoria, and she still to this day experiences no pain, and is completely cured of sickle cell.
there have been only 64 patients that have been through casgevy therapy (a type of therapy using CRISPR), and 99% of those patients have been cured, most of them no longer having to be in hospital anymore, and have never experienced pain crises from their disease.
THE RISKS.
the risks of crispr based therapies is that, before the crispr edited cells can be given, the patient must undergo a conditioning treatment with chemotherapy (like busulfan) to wipe out their existing bone marrow. this can cause severe nausea, mouth sores, the risk of life threatening infections, and potential infertility. and because crispr works like molecular scissors, there is a risk that it could make unintended cuts elsewhere in the genome.
will this be more commonly used? most likely yes. casgevy's competition is other gene therapies like lyfgenia, and casgevy costs 2.2 million per course, while lyfgenia costs 3.1 million per course. a near 1 million dollar difference! casgevy is also deemed safer than lyfgenia, because casgevy finds and replaces, or cuts, while lyfgenia relies on gene insertion, which uses a modified virus to carry an entirely new, artificial hemoglobin gene into the cell. describing lyfgenia, in my opinion, sounds way scarier than casgevy, and casgevy will most likely get picked when a patient is given options.
