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#CRISPR

3 posts3 participants0 posts today

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

Meine Kollegin Alisa Pankau und ich haben lange über eine passende Überschrift für ihre News nachgedacht. Es flogen großartige - und vor allem auch witzige - Varianten hin und her. 🤩

Letztlich haben wir uns für diese entschieden, denn - sind wir mal ehrlich - um eine Abwandlung des legendären Rambo-Zitates kam man hier nicht drum herum!

#Spinnen #Biotech #CRISPR

t3n.de/news/er-leuchtet-rot-fo

t3n Magazin · Er leuchtet rot: Warum Forschende einen Spinnenfaden verändert haben - t3n – digital pioneersEin Forscher-Team hat die Genschere CRISPR-Cas9 bei Spinnen eingesetzt, sodass ihre Nachkommen rot fluoreszierende Spinnenseide erzeugen. 

At any moment, and largely hidden from sight, bacteria and viruses are battling it out.

These skirmishes have inspired revolutionary technologies: Discoveries related to the #CRISPR-Cas system, for example, which allows bacteria to defend themselves from invading viruses, were recognized with a 2020 Nobel Prize.

New tech gleaned from this ongoing arms race includes potential antiviral medications and tricks for fighting bacteria, Amber Dance writes at Nature.
nature.com/articles/d41586-025

www.nature.comThe hunt for the next CRISPR: how warring microbes are inspiring new technologyGene editing and many other useful biotechnology tools came from studies of bacteria fighting off viral invaders. But scientists have only begun to unlock the secrets of this ancient arms race.