From my understanding there talking about using CRISPR-Cas9 to treat including rheumatoid arthritis (RA), inflammatory bowel diseases (IBD), systemic lupus erythematosus (SLE), multiple sclerosis (MS), type 1 diabetes mellitus (DM), psoriasis, and type 1 coeliac disease.

QUOTE Autoimmune diseases are disorders that destruct or disrupt the body's own tissues by its own immune system. Several studies have revealed that polymorphisms of multiple genes are involved in autoimmune diseases. Meanwhile, gene therapy has become a promising approach in autoimmune diseases, and clustered regularly interspaced palindromic repeats and CRISPR-associated protein 9 (CRISPR-Cas9) has become one of the most prominent methods. It has been shown that CRISPR-Cas9 can be applied to knock out proprotein convertase subtilisin/kexin type 9 (PCSK9) or block PCSK9, resulting in lowering low-density lipoprotein cholesterol. In other studies, it can be used to treat rare diseases such as ornithine transcarbamylase (OTC) deficiency and hereditary tyrosinemia. However, few studies on the treatment of autoimmune disease using CRISPR-Cas9 have been reported so far. In this review, we highlight the current and potential use of CRISPR-Cas9 in the management of autoimmune diseases. We summarize the potential target genes for immunomodulation using CRISPR-Cas9 in autoimmune diseases including rheumatoid arthritis (RA), inflammatory bowel diseases (IBD), systemic lupus erythematosus (SLE), multiple sclerosis (MS), type 1 diabetes mellitus (DM), psoriasis, and type 1 coeliac disease. This article will give a new perspective on understanding the use of CRISPR-Cas9 in autoimmune diseases not only through animal models but also in human models. Emerging approaches to investigate the potential target genes for CRISPR-Cas9 treatment may be promising for the tailored immunomodulation of some autoimmune diseases in the near future. QUOTE

What is this CRISPR-Cas9 how would they treat it? Are they changing the genes or turning the genes off?

PubMed

Genome Editing Using CRISPR-Cas9 and Autoimmune Diseases:...

Autoimmune diseases are disorders that destruct or disrupt the body's own tissues by its own immune system. Several studies have revealed that polymorphisms of multiple genes are involved in autoim...

3 hours ago, Moon99 said:

QUOTE Autoimmune diseases are disorders that destruct or disrupt the body's own tissues by its own immune system. Several studies have revealed that polymorphisms of multiple genes are involved in autoimmune diseases. Meanwhile, gene therapy has become a promising approach in autoimmune diseases, and clustered regularly interspaced palindromic repeats and CRISPR-associated protein 9 (CRISPR-Cas9) has become one of the most prominent methods. It has been shown that CRISPR-Cas9 can be applied to knock out proprotein convertase subtilisin/kexin type 9 (PCSK9) or block PCSK9, resulting in lowering low-density lipoprotein cholesterol. In other studies, it can be used to treat rare diseases such as ornithine transcarbamylase (OTC) deficiency and hereditary tyrosinemia. However, few studies on the treatment of autoimmune disease using CRISPR-Cas9 have been reported so far. In this review, we highlight the current and potential use of CRISPR-Cas9 in the management of autoimmune diseases. We summarize the potential target genes for immunomodulation using CRISPR-Cas9 in autoimmune diseases including rheumatoid arthritis (RA), inflammatory bowel diseases (IBD), systemic lupus erythematosus (SLE), multiple sclerosis (MS), type 1 diabetes mellitus (DM), psoriasis, and type 1 coeliac disease. This article will give a new perspective on understanding the use of CRISPR-Cas9 in autoimmune diseases not only through animal models but also in human models. Emerging approaches to investigate the potential target genes for CRISPR-Cas9 treatment may be promising for the tailored immunomodulation of some autoimmune diseases in the near future. QUOTE

What is this CRISPR-Cas9 how would they treat it? Are they changing the genes or turning the genes off?

PubMed

Genome Editing Using CRISPR-Cas9 and Autoimmune Diseases:...

Autoimmune diseases are disorders that destruct or disrupt the body's own tissues by its own immune system. Several studies have revealed that polymorphisms of multiple genes are involved in autoim...

In the context of autoimmune diseases, the immune system mistakenly attacks the body's own cells, causing chronic inflammation and damage. These diseases are often caused by a combination of genetic and environmental factors. Many genes are involved, including those that control how immune cells recognize the body’s own tissues or how inflammation is triggered and controlled. CRISPR-Cas9 could be used to treat autoimmune diseases by modifying or disabling genes that are causing the immune system to overreact. For example, researchers may use CRISPR to knock out a gene that produces a protein involved in triggering inflammation. By turning off this gene, the level of inflammation could be reduced, and the immune attack could be slowed or stopped.

Whether scientists choose to change the gene or simply turn it off depends on the nature of the disease and the role of the gene. In some cases, the gene may be producing a harmful protein, and turning the gene off entirely might be the best approach. In other cases, the gene might be faulty and producing the wrong version of a protein, so editing it to correct the mutation would be more effective. In either case, CRISPR-Cas9 allows scientists to directly interact with the DNA of the cells to either silence the harmful gene or to repair it. Some researchers are also experimenting with CRISPR methods that do not involve cutting the DNA, but instead temporarily silence a gene by interfering with its expression. This could offer a safer, reversible way to modulate the immune system without permanently changing the DNA.

Right now, CRISPR is not yet being widely used in humans to treat autoimmune diseases, but it has already been tested in humans for other conditions. For example, CRISPR has been used in clinical trials for sickle cell anemia, certain cancers, and inherited forms of blindness. Most autoimmune disease studies are still in the research phase, using cell cultures or animal models to identify target genes and test delivery methods. Because autoimmune diseases are complex and involve many genes and pathways, scientists need to be careful in selecting the right targets and ensuring that editing one gene does not create new problems elsewhere. However, the research is progressing, and the article you quoted summarizes how CRISPR might eventually be used in both animal and human models to selectively and safely adjust the immune response.

Hope this helped 😁

1 hour ago, Sohan Lalwani said:

Many genes are involved, including those that control how immune cells recognize the body’s own tissues or how inflammation is triggered and controlled. CRISPR-Cas9 could be used to treat autoimmune diseases by modifying or disabling genes that are causing the immune system to overreact. For example, researchers may use CRISPR to knock out a gene that produces a protein involved in triggering inflammation. By turning off this gene, the level of inflammation could be reduced, and the immune attack could be slowed or stopped.

That is a very unlikely approach. For the most part, we need our genes and just deleting one or even a full knockdown is normally not good news. It would also not use the actual benefit of CRISPR/Cas9, which is targeted editing (rather than full knockout or just a knockdown). What it can be used is to introduce functional alleles of genes to counter the whatever genetic issues there might be.

Another idea is to to directly edit stem cells to replace the harmful with a non-harmful variant, but that can be a bit tricky. Usually you only get part of the reproducing cells (usually using a lentiviral vector) so it is often better to introduce something that can withstand the genetic issue. This is case the for sickle cell disease treatment, where a modified hemoglobin is introduced.

1 hour ago, CharonY said:

That is a very unlikely approach. For the most part, we need our genes and just deleting one or even a full knockdown is normally not good news. It would also not use the actual benefit of CRISPR/Cas9, which is targeted editing (rather than full knockout or just a knockdown). What it can be used is to introduce functional alleles of genes to counter the whatever genetic issues there might be.

Another idea is to to directly edit stem cells to replace the harmful with a non-harmful variant, but that can be a bit tricky. Usually you only get part of the reproducing cells (usually using a lentiviral vector) so it is often better to introduce something that can withstand the genetic issue. This is case the for sickle cell disease treatment, where a modified hemoglobin is introduced.

I believe overall it is true that in many cases we need our genes to function correctly, and deleting a gene entirely can lead to unintended side effects, particularly when the gene in question plays multiple roles in different tissues. However, there are situations in autoimmune disease research where targeted knockouts or partial gene silencing can be beneficial, especially when focused on non-essential inflammatory mediators or tissue-specific immune pathways. For example, silencing certain cytokine genes that are overactive in diseases like rheumatoid arthritis or inflammatory bowel disease has shown promise in preclinical studies.

Regarding your point about stem cells and delivery, it is accurate that modifying all hematopoietic stem cells can be challenging, especially with viral vectors. But this area is advancing quickly. Non-viral delivery systems such as electroporation of Cas9 protein and guide RNA complexes are now widely used and can achieve high editing efficiencies while reducing the risk of off-target effects. In some trials, such as those for sickle cell disease and beta thalassemia, edited stem cells have achieved therapeutic levels of correction. This shows that while full replacement of all stem cells is difficult, editing a sufficient proportion can still be clinically meaningful.

Also I never said this before, but thank you 😀

You are very helpful when I am discussing medically related topics

Is this CRISPR-Cas9 or some other CRISPR?

First gene-edited islet transplant in a human passes functional trial.

Uppsala University Hospital-led investigators report that gene-edited donor islet cells survived 12 weeks inside a man with long-standing type 1 diabetes without any immunosuppressive medication.

https://medicalxpress.com/news/2025-08-gene-islet-transplant-human-functional.html

And why did they deigned it to only last 12 weeks? After 12 weeks would problem not come back?

Why did they not use gene editing on his own cells?

No, they used CRISPR-CAS12b. There are technical nuances between those two systems but I don't think they are relevant to your question. Using his own cells would be a bit pointless as they lost their function resulting in type I diabetes. If they wanted to use the patient cells, they would first need to harvest cells, which is hugely invasive (the donor in this case is deceased), figure out everything that is wrong (type I can be caused by many mutations and likely include issues that are not yet known) and in many cases it would be beyond CRISPR-CAS to make them functional again. In this case, using a donor with with functioning pancreas and just knock out antigenic regions that could result in rejection is way easier and more practical.

The study only described the results after 12 weeks under observation. It is likely that funding term is over and they published their results they had at that point. It does not mean that they are not doing follow-ups and publish e.g. what happens after 1 year. Also the clinical trial registration ran out at this point and more paperwork might be needed to extend.

3 hours ago, CharonY said:

No, they used CRISPR-CAS12b. There are technical nuances between those two systems but I don't think they are relevant to your question. Using his own cells would be a bit pointless as they lost their function resulting in type I diabetes. If they wanted to use the patient cells, they would first need to harvest cells, which is hugely invasive (the donor in this case is deceased), figure out everything that is wrong (type I can be caused by many mutations and likely include issues that are not yet known) and in many cases it would be beyond CRISPR-CAS to make them functional again. In this case, using a donor with with functioning pancreas and just knock out antigenic regions that could result in rejection is way easier and more practical.

The study only described the results after 12 weeks under observation. It is likely that funding term is over and they published their results they had at that point. It does not mean that they are not doing follow-ups and publish e.g. what happens after 1 year. Also the clinical trial registration ran out at this point and more paperwork might be needed to extend.

Could they not use stem cells and grow stem cells than transplant that in him? Or this guy needed new organ?

That is even more complicated and also useless.. Stem cells are non-differentiated cells and cannot really do anything. You would need to control their differentiation in such a way that they become islet cells to be able to implant them. That only is very tricky. But even worse, they still would have same mutation as already existing islet cell, so essentially you just did a very complicated thing that your body would do on its own without really gaining a benefit at the end of the day.

What that guy needed is just a handful of functioning islet cells, basically a group of cells in the pancreas, that are able to produce hormones such as glucagon and especially in this context, insulin. And since the patient got type I diabetes, their existing cells are unable to fully do that for a range of possible genetic reasons. So if you take cells from the patients, they will have the same underlying issue. And as mentioned before, if you do not know the exact reason, and/or the reason has to be fairly localized (e.g. to specific locus), you cannot hope to reverse the issue with a targeted approach such as CRISPR/CAS.