Biomedical

CRISPR therapy was personalized for the first time and given to a baby

Biology, Biomedical, Science / By

In August 2024, a boy named KJ Muldoon was born. He wasn’t a normal, healthy baby, though. He had a genetic disorder called a CPS1 deficiency that made it difficult for him to process protein. This simple fact is dangerous in itself, but it also makes him have a lot of ammonia in his blood. Too much ammonia is toxic to the brain, so this disorder is extremely deadly.

However, when he was born, a team of scientists from around the world rallied together to create a personalized gene therapy for him. This first of its kind treatment was given to him when he was six months old. After getting three separate doses of this treatment to edit his genes, the mutation seems to be fixed. This was an incredible process that could revolutionize the future of healthcare. 

How was this treatment developed?

When KJ was first born with this disorder, scientists got right to work. They took steps to create this treatment that usually takes almost 10 years, but they collaborated and got it done in just a few months. In most cases, the only way to treat his disease is to get a liver transplant, but it would be a long time before he would be able to get one. This causes 50% of babies with this disease to die before getting a transplant. 

So, the scientists got the parents’ permission to use base editing to try and fix the mutation. Base editing is when they make selective changes to single-letters in DNA sequences. For the base editing approach, fatty lipid molecules were wrapped around a treatment to stop it from breaking down in his blood when it was travelling to his liver, where it made the edit. The lipids housed instructions on how the cells should create an enzyme that can then change the gene. Also, they bring CRISPR with them, which finds the exact letter in the DNA that has to be changed. 

This treatment was actually possible because of decades of federally funded research into things like CRISPR technology. CRISPR is an acronym for Clustered Regularly Interspaced Short Palindromic Repeats. It’s part of the immune system of bacteria that cuts DNA and we now use it as a kind of molecular scissors to edit genes. There are two parts of this system. First, there’s a CRISPR-associated nuclease whose job is to connect and cut DNA. There’s also the guide RNA sequence whose job is to direct the CRISPR-associated nuclease so it reaches its target. This whole process lets scientists rewrite an organism’s genetic code and do things like create drought-resistant crops. 

They had to use this CRISPR technology to personalize KJ’s treatment because, with CPS1 deficiency being a rare disease, pharmaceutical companies are not willing to spend several years and millions of dollars to develop a large-scale treatment. Also, a lot of the time different mutations cause each individual’s disorder, so a mass produced product would possibly not be as effective as this treatment ended up being.

In fact, after just his first dose, KJ was able to safely eat as much protein as he was supposed to at his age. He continued getting treatments to get his ammonia levels under control and, after only three doses, he was done with the treatment. Now, he is almost a year old, and while scientists are still cautious to call his disorder cured and do not know if he will still need a liver transplant someday, KJ does seem to be doing very well right now. 

Implications for the future

This promising sign could lead to a brighter, healthier future for society. First of all, CRISPR could be used in the future to treat other extremely uncommon genetic diseases. The problem with this gene editing therapy though is that it is extremely expensive, so it would not be a cost-effective solution for most people with these issues. At the same time, now that they’ve treated KJ, they can adapt his treatment to fix other people’s mutations too. That means that future treatments could be cheaper for people.

Even though it seems to have been successful so far, this one personalized treatment took teams of scientists and months of work to pull off. Logistically, this would be impossible to do for everyone with a rare disorder. The possibility of this treatment however does create new possible futures, like one where cystic fibrosis or Huntington’s disease could have a form of treatment. 

Despite all the potential financial issues, personalizing gene therapy will inevitably save lives. People with rare diseases will have more of a chance at survival, and in the future, the technology will develop and likely become more accessible. The success of baby KJ’s treatment is a promising step towards a future where certain genetic disorders are no longer a death sentence, but, instead, with some work done by scientists, they will just be another part of their long, healthy lives. 

References

Kolata, G. (2025, May 15). Baby Is Healed With World’s First Personalized Gene-Editing Treatment. The New York Times. Retrieved June 7, 2025, from https://www.nytimes.com/2025/05/15/health/gene-editing-personalized-rare-disorders.html

Ledford, H. (2025, May 15). World’s first personalized CRISPR therapy given to a baby with genetic disease. nature. Retrieved June 7, 2025, from https://www.nature.com/articles/d41586-025-01496-z

What is CRISPR: The Ultimate Guide to CRISPR Mechanisms, Applications, Methods & More. (n.d.). Synthego. Retrieved June 7, 2025, from https://www.synthego.com/learn/crispr

Exploring the Science Behind Allergies

Bio Chemistry, Biomedical, Biotech, Engineering, Immunology, Science, Technology / By

As alarming as it sounds, even a lick of peanut butter could be life-threatening. Allergies. What is it? Let’s see. Had the peanut in peanut butter been harmful to everyone it wouldn’t be called an allergy. Only if something reacts in an unprecedented way to a select few is then called an allergy.

So the question arises, How do I know if I’m allergic and what I am allergic to?

Allergies come in forms, ranging from water to even nickel coins. One can’t possibly predict what substances react weirdly with your body without ever being exposed to it. This is why allergy tests are done.

Well, Only a medical professional could let you know your allergies unless something you had eaten or been exposed to previously didn’t sit right with you. Symptoms of an allergy range from a runny nose to breathlessness and of course, the scary and itchy hives. 

Let’s take a look at what the doctor is doing behind the scenes, shall we?

An immunologist or allergist usually does the test which involves a skin prick or a patch test. The image above, from Westhillsaaa, illustrates a medical personnel checking for unusual reactions in a patient’s skin through various triggers.

The tests could range from injecting the allergens into your skin from an injection to taking out a blood sample. The choice of tests varies according to the patient’s data including their medical history, their condition, and suspected triggers.

Something to note about allergies is that a person can outgrow them with time. This is commonly seen in children getting rid of food allergies but some allergies like that of pollen and medications persist for a long time or even all your life.

Although you can’t possibly get rid of an allergy that still persists in adulthood, you can take certain medications and tests described accordingly to reduce complications.

A common medication is desensitization which is basically building tolerance for your allergen by exposing your body to it periodically under small concentrations. 

A personal suggestion is that you should have an emergency action plan including an EpiPen ready just in case things go south after eating/reacting to something new.

In the near future, who’s to deny that at the rate medical technology is growing, maybe we could even have a permanent remedy for allergies? That’s a topic up for discussion.

Unlocking Limb Regeneration: The Salamander’s Clue to Ending Phantom Pain

Biology, Biomedical, Engineering, Medicine, Science / By

You wake up in the morning with some arm pain. Sounds pretty normal, no? But what if you were told that that pain was all in your head? Alright, well obviously it’s in your head, your brain is what detects the pain, but bear with me. What if your arm wasn’t… there? What if your arm hadn’t been there for years, only for you to still feel it being there despite this objective truth? If all of that applies to you, then you are a victim of phantom pain. 

Over 500 people lose their limbs each day, whether it’s through the brutality of warfare, a freak accident at a job, or otherwise. Of these individuals, approximately 80% of them experience phantom pain. Not to mention that all of them suffer some degree of reduction in quality of life and even mental health for some. For what seems like its conception, limbs and their loss seemed like an unfortunate reality of the world. We all are only given one pair of parts, if we lose them somehow, that’s on us. That’s it. No redos, no replacements, no takesies-backsies. 

But what if there were redos? What if there was a way to replace the irreplaceable? What if there was a way to grow the ungrowable? That may very be possible through researching an animal that most of us have yet to encounter in the flesh: the humble salamander. 

What makes the salamander so special lies in its capability to regrow its limbs in its entirety. It could lose all of its arms and legs: as long as the stump is not destroyed, it can regrow them again and again without fail. These were only possible through the salamander’s natural capabilities to salvage what was left and prevent the wound from festering. The blood vessels quickly contract and a layer of skin cells swiftly encase the wound site. This wasn’t what made the salamander’s capability to regrow limbs so odd though, it was that it had other parts of its body (namely the opposite side of the missing limb) chip in to regrow the lost part. Although it would sometimes appear in a slightly different place, it would, for all anatomically sound purposes, be a perfectly functional limb. 

Although humans and salamanders are not the same, we both possess some form of a regeneration factor (with the former resorting to healing and the latter resulting in completely regrowing a limb). It is just a matter of time before we transform our natural capability to heal into being able to regrow lost limbs, perhaps indefinitely. No more will those who are missing an arm or a leg through horrid circumstances have to suffer a poor quality of life. No more will they have to make do with painkillers and accept the never-ending pain. No more will they have to live their life to a fraction of their potential all because of an unfortunate sequence of events. With the help of some salamanders, these circumstances may very well become a thing of the past. 

Image Credit: CAS.org, depiction of limbs regenerating.

Sources

  1. https://acl.gov/sites/default/files/programs/2021-04/llam-infographic-2021.pdf
  2. https://www.montefiore.org/limb-loss-facts
  3. https://my.clevelandclinic.org/health/diseases/12092-phantom-limb-pain
  4. https://www.ucl.ac.uk/news/2014/jun/limb-regeneration-do-salamanders-hold-key

AI can now use the help of CRISPR to precisely control gene expressions in RNA

Artificial Intelligence, Bio Chemistry, Biology, Biomedical, Engineering, Medicine, Science, Technology / By

Almost all infectious and deadly viruses are caused due to their RNA coding. Researchers from established research universities, such as NYU and Columbia, alongside the New York Genome Center, have researched and discovered a new type of CRISPR technology that targets this RNA and might just prevent the spread of deadly diseases and infections.

A new study from Nature Biotechnology has shown that the development of major gene editing tools like CRISPR will serve to be beneficial at an even larger scale. CRISPR, in a nutshell, is a gene editing piece of technology that can be used to switch gene expression on and off. Up until now, it was only known that CRISPR, with the help of the enzyme Cas9, could only edit DNA. With the recent discovery of Cas13, RNA editing might just become possible as well.

https://theconversation.com/three-ways-rna-is-being-used-in-the-next-generation-of-medical-treatment-158190

RNA is a second type of genetic material present within our cells and body, which plays an essential role in various biological roles such as regulation, expression, coding, and even decoding genes. It plays a significant role in biological processes such as protein synthesis, and these proteins are necessary to carry out various processes. 

RNA viruses

RNA viruses usually exist in 2 types – single-stranded RNA (ssRNA), and double-stranded RNA (dsRNA). RNA viruses are notoriously famous for causing the most common and the most well-known infections – examples being the common cold, influenza, Dengue, hepatitis, Ebola, and even COVID-19. These dangerous and possibly life-threatening viruses only have RNA as their genetic material. So, how can/might AI and CRISPR technology, using the enzyme Cas13 help fight against these nuisances?

Role of CRISPR-Cas13

RNA targeting CRISPRs have various applications – from editing and blocking genes to finding out possible drugs to cure said pathogenic disease/infection. As a report from NYU states, “Researchers at NYU and the New York Genome Center created a platform for RNA-targeting CRISPR screens using Cas13 to better understand RNA regulation and to identify the function of non-coding RNAs. Because RNA is the main genetic material in viruses including SARS-CoV-2 and flu,” the applications of CRISPR-Cas13 can promise us cures and newer ways to treat severe viral infections.

“Similar to DNA-targeting CRISPRs such as Cas9, we anticipate that RNA-targeting CRISPRs such as Cas13 will have an outsized impact in molecular biology and biomedical applications in the coming years,” said Neville Sanjana, associate professor of biology at NYU, associate professor of neuroscience and physiology at NYU Grossman School of Medicine. Learn more about CRISPR, Cas9, and Cas13 here. 

Role of AI

Artificial intelligence is becoming more and more reliant as days pass by. So much so, that it can be used to precisely target RNA coding, especially in the given case scenario. TIGER (Targeted Inhibition of Gene Expression via guide RNA design), was trained on the data from the CRISPR screens. Comparing the predictions generated by the model and laboratory tests in human cells, TIGER was able to predict both on-target and off-target activity, outperforming previous models developed for Cas13 

With the assistance of AI with an RNA-targeting CRISPR screen, TIGER’s predictions might just initiate new and more developed methods of RNA-targeting therapies. In a nutshell, AI will be able to “sieve” out undesired off-target CRISPR activity, making it a more precise and reliable method.