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#CRISPR

3 posts3 participants1 post today

Le Cong, one of the pioneers of applying #CRISPR in eukaryotic cells, and his team now have developed an agent to apply #ChatGPT #LLM technology to automate and enhance CRISPR-based gene-editing design and data analysis.
In #nature Biomedical Engineering.
nature.com/articles/s41551-025

NatureCRISPR-GPT for agentic automation of gene-editing experiments - Nature Biomedical EngineeringAn agent-based large language model (LLM) tool helps with CRISPR-based gene-editing experimental design and analysis.

**Megatrend #12** - Personalized, Precision Medicine: "Tomorrow's cure lies not in a universal pill, but within our own genetic code." - Futurist Jim Carroll

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(Futurist Jim Carroll is writing a series on 30 Megatrends, which he first outlined in his book Dancing in the Rain: How Bold Leaders Grow Stronger in Stormy Times. The trends were shared in the book as a way of demonstrating that, despite any period of economic volatility, there is always long-term opportunity to be found. The book is now in print - learn more at dancing.jimcarroll.com)

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The one-size-fits-all era in medicine is ending. Treatments, nutrition, and wellness approaches tailored to individual genetic profiles are becoming the standard, not the exception. As with every post in this series, there's a PDF here that goes into more depth on this trend.

There's a little bit of duplication with some of the material covered in previous megatrends with this one, but it's important ot note that this trend is powerful enough on its own to deserve its own post.

What's it all about? We are combining what we might call 'next-generation genomic sequencing', AI, and CRISPR gene editing, to come up with a new health paradigm that involves 'fixing people before they are sick rather than after.' Why these 3 trends? The cost for genomic sequencing is collapsing, AI is accelerating the ability to do so, and gene editing allows us to correct the genetic causes of disease at the source.

Before going further, it's important to define the revolution by understanding two key terms.

The first is Personalized Medicine, which uses an individual's unique genomic, environmental, and lifestyle information to guide decisions for prevention, diagnosis, and treatment of disease. It's based on the idea that because no two individuals are the same, they should not receive the same healthcare.

The second term is Precision Medicine. While often used interchangeably, it more accurately describes the strategy of stratifying individuals into small populations based on shared characteristics. It focuses on identifying which healthcare and pharmaceutical approaches will be most effective for those small, specific groups of people, rather than creating unique treatments for each individual.

While its potential is immense, the transition to personalized medicine is not merely a technological upgrade but a disruptive force that challenges the foundational structures of healthcare delivery, research, and regulation.

Even so, over the long term, it's one of the biggest megatrend opportunities of our time.

**#Personalized** **#Medicine** **#Genomics** **#CRISPR** **#AI** **#Precision** **#Healthcare** **#Genetics** **#Innovation** **#Future**

Original post: jimcarroll.com/2025/07/decodin

Just mind bogglingly amazing - modern #GeneEditing therapies! Doesn't get more #PrecisionMedicine than this!

"First Personalized CRISPR Gene Editing Therapy Patient Baby KJ Discharged

Three hundred and seven days after he was first admitted to Children’s Hospital of Philadelphia (CHOP), the 10-month-old went home after being treated with a bespoke base editing approach"

#Science #CRISPR #genetics
insideprecisionmedicine.com/to

Inside Precision Medicine · First Personalized CRISPR Gene Editing Therapy Patient Baby KJ DischargedBy Jonathan D. Grinstein, PhD

A Gene Editing Strategy to Repair Huntington's Defects

"A lot more studies would be needed before we can know if disrupting these repeats with a base editor could be a viable therapeutic strategy to treat patients. But being able to illuminate the biological consequences of interrupted repeats is a really useful and important milestone," explained senior study author Dr David Liu.

#CRISPR #genetics #research #biology #biotechnology #science #Huntingtons

labroots.com/trending/genetics

Continued thread

This is an example of what is being eliminated as Republicans in Washington, DC and this administration make catastrophic cuts to scientific research (among other things) to provide tax cuts to and curry favor with billionaires:

"World’s first patient treated with personalized CRISPR gene editing therapy at Children’s Hospital of Philadelphia"
pennmedicine.org/News/worlds-f
#ScienceMatters #science #CRISPR #antiscience #DOGE

Baby Is Healed With World’s First Personalized Gene-Editing Treatment.

The technique used on a 9½-month-old boy with a rare condition has the potential to help people with thousands of other uncommon genetic diseases.
nytimes.com/2025/05/15/health/ #genetics #CRISPR #genetherapy

(Just another example of why we should treasure our researchers and scientists)

Continued thread

Good news from @npr, as shared by our @ScienceDesk. A baby born in Philadelphia last August with a life-threatening genetic disorder was successfully treated with a gene-editing infusion developed using CRISPR technology. His genetic defect appears to have been fixed, at least partially reversing his condition.

npr.org/sections/shots-health-

When KJ Muldoon was born at Children's Hospital of Philadelphia (CHOP) in August, doctors noticed that he was lethargic. They carried out tests that revealed he had a genetic metabolic disorder that leads to the buildup of ammonia and can cause brain damage and death. Gene sequencing helped them determine the exact location of the error that led to him being unable to make a vital enzyme. This allowed doctors to use CRISPR technology to create a treatment tailored specifically towards his unique mutation. After three infusions containing billions of gene editors, his defect appears to have been corrected and his condition, at least partially reversed. "This is an important first step towards an entirely new type of personalized medicine. I think it's going to utterly transform the way we practice medicine, particularly in the area of rare diseases," says Dr. Kiran Musunuru, a professor for translational research at the University of Pennsylvania, who worked on KJ's case. See the @npr story at the first link. There's a link to the original article in the New England Journal of Medicine at the second link.

flip.it/M_l8MJ

flip.it/R-WrOX