CRISPR & Genetic Engineering News and Discussions

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Electrical synapses genetically engineered in mammals for first time

Scientists have used gene editing to produce artificial electrical synapses in mice, where they can be targeted to make the animals more sociable or reduce their risk of OCD-like symptoms

14 April 2025

Electrical synapses that carry messages through the brain have been artificially engineered in mammals for the first time, altering their behaviour. This could have potential for preventing or treating a range of mental health conditions, including obsessive compulsive disorder (OCD).

Connections, or synapses, between nerve cells are either electrical or chemical. Chemical ones, which are more common in mammals, involve molecules called neurotransmitters, whereas electrical synapses rely on proteins called connexins.

Many mental health conditions seem to occur when something goes wrong with the neurotransmitter-based signalling system, says Kafui Dzirasa at Duke University in Durham, North Carolina. “We wanted to know if we could engineer a way to bypass the chemical synapses between cells by putting an electrical synapse there.”

First, Dzirasa and his colleagues looked for proteins from other organisms that could be used to build an electrical synapse in mice. Similar work was previously done in the nematode worm Caenorhabditis elegans, but that animal only has 302 neurons, so it was relatively simple, whereas mice have about 71 million neurons.

“We found [the connexins] by searching an incredible amount of literature to find proteins with exactly the properties that we’d want to engineer a human system with,” says Dzirasa.

https://www.newscientist.com/article/24 ... irst-time/

[firestar464]

Engineering 'bespoke enzymes': Machine learning expands CRISPR toolbox

https://phys.org/news/2025-04-bespoke-e ... olbox.html

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Gene-editing therapy shows early success in fighting advanced gastrointestinal cancers
https://medicalxpress.com/news/2025-05- ... anced.html
by Alex Smith, University of Minnesota Medical School

Researchers at the University of Minnesota have completed a first-in-human clinical trial testing a CRISPR/Cas9 gene-editing technique to help the immune system fight advanced gastrointestinal (GI) cancers. The results, recently published in The Lancet Oncology, show encouraging signs of the safety and potential effectiveness of the treatment.

"Despite many advances in understanding the genomic drivers and other factors causing cancer, with few exceptions, stage IV colorectal cancer remains a largely incurable disease," said Emil Lou, MD, Ph.D., a gastrointestinal oncologist with the University of Minnesota Medical School, Masonic Cancer Center and M Health Fairview, and clinical principal investigator for the trial. "This trial brings a new approach from our research labs into the clinic and shows potential for improving outcomes in patients with late-stage disease."

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World’s first gene-edited spider produces red fluorescent silk
By Jay Kakade
May 19, 2025

https://newatlas.com/biology/worlds-fir ... cent-silk/
For years, the CRISPR-Cas9 genome technology has been reshaping genetic engineering, a precision tool to transform everything from agriculture to medicine. With its incredible efficiency, this molecular tool has been applied to plants, animals, and even bacteria. But until now, no one has used CRISPR-Cas9 on spiders.

Researchers at the University of Bayreuth have recently successfully bred the world’s first CRISPR-Cas9-modified spider to produce red fluorescent silk. For this work, they used a species of common house spider (Parasteatoda tepidariorum).

Since many spiders are cannibalistic, with a diverse and complex genome architecture, genetic manipulation and nurturing the resulting offspring poses significant challenges. These factors have left them underrepresented in laboratory research. To overcome these hurdles, the researchers developed a novel CRISPR solution containing the gene sequence for a red fluorescent silk protein and injected it into unfertilized spider eggs.

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World's first therapy to reverse spinal cord injury enters human trial
By Bronwyn Thompson
May 26, 2025
https://newatlas.com/chronic-pain/spina ... l-therapy/

A paradigm shift in the way we treat spinal injuries is now in sight, with the world's first regenerative cell therapy being granted approval for a registrational Phase I clinical trial. It's a historical milestone that could successfully treat what has, until now, been an incurable condition.

This week, the US Food and Drug Administration (FDA) and China’s National Medical Products Administration (NMPA) approved the global clinical trial to treat spinal cord injury (SCI), which is estimated to affect more than 15 million people worldwide. SCI impacts people across demographics, and most often results from traffic accidents, sporting injuries and other trauma, including serious falls and workplace incidents. There's no real cure; treatment is more management, with surgery and rehabilitation to restore some degree of quality of life. Sufferers, however, are often left with paralysis or severe disability for life.

Now, Chinese biotechnology company XellSmart has the potential to change this forever, as its allogeneic induced pluripotent stem cells (iPSC) regenerative therapy has been given the green light by both US and Chinese health administrations to enter a clinical trial. Pluripotent stem cells are, of course, immature stem cells that can develop into specific cells. In this case, the kind to replace the damaged or dead neural cells caused by SCI. The treatment aims to not just repair the injury but provide the foundation to regrow all the cells needed to return function to the damaged region.

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Hemophilia B gene therapy demonstrates long-term success
https://medicalxpress.com/news/2025-06- ... ccess.html
by St. Jude Children's Research Hospital

A gene transfer approach to treating the bleeding disorder hemophilia B remains safe and effective long-term, as scientists from St. Jude Children's Research Hospital and University College London today report thirteen years of follow-up data. Hemophilia B is a rare genetic disorder caused by insufficient levels of a circulating protein called factor IX, which promotes blood clotting.

The researchers used a one-time gene therapy intervention to address the disorder. Published in The New England Journal of Medicine, the 13-year follow-up study is the longest reported for any gene therapy for hemophilia B. The results, including an almost tenfold reduction in annualized bleeding rate, support the long-term viability of gene therapy for the disease's treatment.

Hemophilia B is an X-linked genetic disorder affecting 1 in approximately 25,000 male births. While the disorder can range in severity, frequent spontaneous bleeding and life-threatening hemorrhages occur due to insufficient blood-clotting factor IX. Treatment for hemophilia B has traditionally been expensive for lifelong supplementation of the clotting factor, but gene therapy offers a potentially transformative means to address the disorder.

"The key benefit is that gene therapy is a one-time, simple intravenous infusion that's very straightforward to do and potentially has positive effects for a lifetime," said co-investigator on the study Andrew Davidoff, MD, St. Jude Department of Surgery chair.

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Phase III trial shows gene therapy skin grafts help heal chronic wounds in blistering skin disease
https://medicalxpress.com/news/2025-06- ... erapy.html
by Stanford University Medical Center

Skin grafts genetically engineered from a patient's own cells can heal persistent wounds in people with an extremely painful dermatologic disease, a Stanford Medicine-led clinical trial has shown. The grafts treat severe dystrophic epidermolysis bullosa, or EB, a genetic condition in which the skin is so fragile the slightest touch can cause blistering and wounds, eventually leading to large, open lesions that never heal and are immensely painful.

A Phase III clinical trial showed that EB patients experienced significantly better healing, less pain and less itching from wounds treated with the genetically engineered grafts compared with skin wounds that were not grafted.

The results were published in The Lancet. The skin grafts were granted approval as an EB therapy on April 29, 2025, by the U.S. Food and Drug Administration.

"With our novel gene therapy technique, we successfully treated the hardest-to-heal wounds, which were usually also the most painful ones for these patients," said the study's lead author, Jean Tang, MD, Ph.D., a professor of dermatology who treats children with EB at Lucile Packard Children's Hospital Stanford.

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Gene therapy may slow loss of motor function in ALS
https://medicalxpress.com/news/2025-06- ... ction.html
by Children's Hospital of Philadelphia

Researchers have developed a gene therapy that significantly slowed motor function loss in preclinical models of amyotrophic lateral sclerosis (ALS), offering new hope for treating the devastating neurodegenerative disease.

"Silencing" a gene associated with regulating TDP-43, the protein that accumulates in the brain and causes ALS, with a technique called RNA interference (RNAi) allowed mice to survive an average of 54% longer. Subjects also experienced improvements in strength and reduced inflammation in the brain and spinal cord, according to research from the Perelman School of Medicine at the University of Pennsylvania and Children's Hospital of Philadelphia, published today in Nature Communications.

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Genetically modified gut bacteria show promise for combating kidney stones in clinical trial
https://medicalxpress.com/news/2025-07- ... idney.html
by Krystal Kasal, Phys.org

The human gut microbiome has been shown to impact health in a myriad of ways. The type and abundance of different bacteria can impact everything from the immune system to the nervous system. Now, researchers at Stanford University are taking advantage of the microbiome's potential for fighting disease by genetically modifying certain bacteria to reduce a substance that causes kidney stones. If scientists are successful at modifying gut bacteria, this can lead to therapeutic treatments for a wide range of diseases.

However, the study, published in Science, shows that this is not a simple task. The researchers used the bacterium Phocaeicola vulgatus, which is already found in the microbiome of humans, and modified it to break down oxalate and also to consume porphyran, a nutrient derived from seaweed. The porphyran was used as a way to control the population of Phocaeicola vulgatus by either adding more porphyran or reducing the amount, which should kill off the bacteria due to a lack of food.

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[firestar464]

this is old news

https://www.eurekalert.org/news-releases/1073061

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Damn straight! I wish you could use this tech to enhance your iq as an adult. I'd choose to raise it into the 300s!

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If you can edit the whole thing couldn't it allow for womb growth?

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