CRISPR & Genetic Engineering News and Discussions

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CRISPR's efficiency triples in lab tests with DNA-wrapped nanoparticles
https://phys.org/news/2025-08-crispr-ef ... b-dna.html
by Northwestern University

With the power to rewrite the genetic code underlying countless diseases, CRISPR holds immense promise to revolutionize medicine. But until scientists can deliver its gene-editing machinery safely and efficiently into relevant cells and tissues, that promise will remain out of reach.

Now, Northwestern University chemists have unveiled a new type of nanostructure that dramatically improves CRISPR delivery and potentially extends its scope of utility.

Called lipid nanoparticle spherical nucleic acids (LNP-SNAs), these tiny structures carry the full set of CRISPR editing tools—Cas9 enzymes, guide RNA and a DNA repair template—wrapped in a dense, protective shell of DNA. Not only does this DNA coating shield its cargo, but it also dictates which organs and tissues the LNP-SNAs travel to and makes it easier for them to enter cells.

In lab tests across various human and animal cell types, the LNP-SNAs entered cells up to three times more effectively than the standard lipid particle delivery systems used for COVID-19 vaccines, caused far less toxicity and boosted gene-editing efficiency threefold. The new nanostructures also improved the success rate of precise DNA repairs by more than 60% compared to current methods.

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Gene-edited immune cells show promise for universal 'off-the-shelf' cancer therapy
https://medicalxpress.com/news/2025-09- ... shelf.html
by Pralhad Gupta, Peking University

Peking University scientists have developed a cancer therapy that could make life-saving treatment accessible to any patient, anywhere. A team led by Professor Wei Wensheng from Peking University, collaborating with the PLA General Hospital and biotech company EdiGene Inc., has developed a novel cancer therapy that could make advanced treatment accessible to many more patients. Their study is published in the journal Cell.

CAR-T therapy is one of the most exciting breakthroughs in cancer treatment. It works by taking a patient's own immune cells, reprogramming them to recognize cancer, and putting them back into the body to fight the disease. Many patients with leukemia, lymphoma, and other blood cancers have seen dramatic recoveries thanks to this method.

But there are big problems: It takes weeks to prepare each patient's treatment, it is very expensive, and some very sick patients don't have healthy cells to work with. Scientists have long dreamed of an "off-the-shelf" version of CAR-T made from healthy donor cells so treatment could be ready right away. The challenge is that the body usually rejects donor cells, or the donor cells attack the patient, making them unsafe.

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Small nuclear RNA base editing offers a safer alternative to CRISPR, researchers find
https://phys.org/news/2025-09-small-nuc ... safer.html
by University of California - San Diego

Genetic editing holds promise to treat incurable diseases, but the most popular method—CRISPR—sometimes does more harm than good. A new study from University of California San Diego and Yale University researchers highlights an innovative alternative approach that may be safer. The study was published in Nature Chemical Biology on Sept. 18, 2025.

CRISPR—short for clustered regularly interspaced short palindromic repeats—is a method of genetic editing that uses RNA and bacterial proteins to edit DNA. It was adapted from a method used by bacteria as an immune defense against the DNA of viruses.

When the method is used by scientists to edit human DNA, however, there can be unintended consequences. These can include accidental edits that can cause life-threatening health conditions in the near term. What's more, the long-term effects of CRISPR editing are unknown and may include an increased risk of cancer.

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How a 'speech gene' could help treat Huntington's

by Lisa Kim, Stanford University
https://medicalxpress.com/news/2025-09- ... ngton.html

In fatal genetic diseases like Huntington's and spinocerebellar ataxia, proteins develop long stretches of repeating letters that are prone to sticking together like Velcro.

As the proteins clump up in the brain, they damage and kill neurons, triggering severe cognitive decline. Clinicians don't have any treatments targeting these disease-causing clumps, and Huntington's and related diseases have no cure.

But there's a mystery: Some proteins naturally have the same repetitive regions—called polyglutamine or polyQ expansions—that are just as long, or even longer, and they don't clump at all.

A study from Stanford Medicine, published in Cell in April, dives into that mystery. The research team focused on FOXP2, a gene famously tied to human speech and language.

To their surprise, the protein contains one of the longest known polyQ stretches in the human body, but it usually doesn't clump or cause disease.

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A more precise CRISPR platform enables large-scale gene screening in live mouse brains
https://medicalxpress.com/news/2025-09- ... large.html
by Ingrid Fadelli, Phys.org

Over the past few decades, biomedical researchers and neuroscientists have devised increasingly advanced techniques to study and alter neurophysiological processes. These include CRISPR (Clustered Regularly Interspaced Short Palindromic Repeats), a sophisticated tool to edit specific genes in some animals, including mice, rats, zebrafish and fruit flies.

Researchers at University of California, San Francisco led by Martin Kampmann recently introduced a more precise CRISPR screening platform that can be applied directly in living tissue, enabling the screening of a larger number of genes at once. The new technique, called CRISPR screening by AAV episome sequencing (CrAAVe-seq), was introduced in a paper published in Nature Neuroscience.

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Polyunsaturated fatty acid therapy reverses age-related vision decline in mice

by University of California, Irvine
https://medicalxpress.com/news/2025-09- ... erses.html

Changes in vision are often a common sign of aging. But what if we could reverse age-related visual decline? In a new study, UC Irvine researchers explore a possible therapy for addressing "aging" in the eye and for preventing diseases such as age-related macular degeneration (AMD).

"We show the potential for reversing age-related vision loss," says Dorota Skowronska-Krawczyk, Ph.D., an associate professor in the Department of Physiology and Biophysics and the Department of Ophthalmology and Visual Sciences. The study was a collaboration between researchers from UC Irvine, the Polish Academy of Sciences, and the Health and Medical University in Potsdam, Germany.

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95% of kids with “bubble boy” disease cured by one-time gene therapy
By Paul McClure
October 16, 2025

A one-time gene therapy using a patient’s own stem cells has effectively cured a deadly immune disorder in 95% of treated children, offering a lasting, donor-free solution to ADA-SCID, known as the “bubble boy” disease.

Those who are old enough may remember “the boy in the bubble,” David Vetter, born with a rare hereditary condition called severe combined immunodeficiency (SCID). It affected his immune system, making even the mildest of infections life-threatening. Vetter died in 1984, at age 12.

In a new study, researchers from UCLA, University College London, and Great Ormond Street Hospital, London, trialed an experimental gene therapy for children born with severe combined immunodeficiency due to adenosine deaminase deficiency (ADA-SCID), demonstrating a 95% long-term success rate.

https://newatlas.com/disease/ada-scid-g ... rapy-cure/

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One-off lipid-lowering gene therapy a success in world-first human trial
By Paul McClure
November 10, 2025

In a world-first trial, scientists used a one-off CRISPR gene edit to switch off a liver “fat brake” gene, slashing stubborn LDL cholesterol and triglycerides in patients whose levels refused to budge on standard treatments.

The angiopoietin-like protein 3 (ANGPTL3) gene is a liver fat brake, telling the body to slow the clearance of fats such as low-density lipoprotein (LDL, the “bad”) cholesterol and triglycerides from the blood. Some people are born with a mutation that turns this brake off leaving these lipids unusually low.

Scientists from the Victorian Heart Hospital and the Victorian Heart Institute, in partnership with Monash University, have conducted the first-in-human clinical trial of a breakthrough gene therapy that mimics this condition in people with hard-to-treat lipid disorders.

https://newatlas.com/medical-tech/gene- ... lycerides/

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How to fix genetic ‘nonsense’: versatile gene-editing tool could tackle a host of diseases

https://www.nature.com/articles/d41586-025-03770-6

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Boy with rare condition amazes doctors after world-first gene therapy

Published
24 November 2025, 00:24 GMT

A three-year-old boy has astounded doctors with his progress after becoming the first person in the world with his devastating disease to receive a ground-breaking gene therapy.

Oliver Chu has a rare, inherited condition called Hunter syndrome - or MPSII - which causes progressive damage to the body and brain.

In the most severe cases, patients with the disease usually die before the age of 20. The effects are sometimes described as a type of childhood dementia.

Due to a faulty gene, before the treatment Oliver was unable to produce an enzyme crucial for keeping cells healthy.

In a world first, medical staff in Manchester have tried to halt the disease, external by altering Oliver's cells using gene therapy.

https://www.bbc.co.uk/news/articles/c5y0y56x6veo

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Watch: Glowing tool seeks out DNA damage in real time
By Pranjal Malewar
December 09, 2025
https://newatlas.com/imaging-diagnostic ... lf-repair/

DNA can sustain serious injuries called double strand breaks, in which both strands of the helix snap. These breaks are among the most dangerous forms of DNA damage and immediately trigger the cell's damage response. Because DNA damage constantly threatens the stability of our genome, the ability to repair it is vital. When repair systems weaken, diseases can arise. That's why scientists study the molecular pathways of DNA repair: they could lead to new therapies.

But there's a challenge. To truly see how cells fix themselves, scientists need tools that can watch DNA damage in real time. Until recently, this wasn’t possible. Most methods involved stopping and preserving cells at different stages, which only gave snapshots instead of a live view of the repair process.

Now, researchers at Utrecht University have built glowing sensors that show exactly where DNA breaks happen. For the first time, they can watch DNA damage and repair play out live inside cells. It’s like opening a window into the cell’s repair system, revealing the process in real time.

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Genetic trick to make mosquitoes malaria resistant passes key test

The rollout of a type of genetic technology called a gene drive for tackling malaria could be edging closer after a lab study supports its success

By Michael Le Page
10 December 2025

https://www.newscientist.com/article/25 ... -key-test/

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Gene editing takes a major step toward ending daily cholesterol pills
By Bronwyn Thompson
January 25, 2026
https://newatlas.com/heart-disease/chol ... ng-crispr/

A new CRISPR-based one-off procedure that lowers "bad" cholesterol has been approved to enter Phase I human trial. If successful, it could be the first approved genetic-silencing method on the market, replacing the need for long-term medication and slashing the risk of cardiovascular disease.

There are high hopes for US biotech company Scribe Therapeutics' STX-1150 treatment, which epigenetically silences the PCSK9 gene in the liver to reduce low-density lipoprotein cholesterol (LDL-C). This is, of course, not the first of its kind – we wrote about Verve Therapeutics' effort in 2023 and CRISPR Therapeutics' CTX310 candidate more recently. However, they're both still in their trial stages.

STX-1150 targets hypercholesterolemia, a key driver of atherosclerotic cardiovascular disease (ASCVD). It epigenetically silences PCSK9 to reduce LDL-C without making any permanent DNA changes.

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RNA therapy may be a solution for infant hydrocephalus

https://medicalxpress.com/news/2026-01- ... halus.html

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This is nice. I bet dollars to donuts that you could pretty much do 90% of everything imaginable with gene editing.

[firestar464]

No, this has nothing to do with gene engineering. This is about the gut microbiome, and the study does not mention superhuman strength; only that having this bacterium in the gut as an older adult is better for grip strength. The same effect is not noticed in younger adults; in other words, it's not a universal boost.

https://gut.bmj.com/content/early/2026/ ... 025-336980