Tag

Crispr

All articles tagged with #crispr

science4 days ago•9 min saved

Early-Development ZNF804A Linked to Hyper-Excitable Neurons in Schizophrenia

A precision functional genomics study shows the schizophrenia risk gene ZNF804A is most active in glutamatergic neurons during the second trimester. Using CRISPR-Cas9 to suppress ZNF804A in developing neurons increased local protein translation at dendritic tips, raised synapse density, and heightened electrical excitability, linking this genetic risk factor to a specific neurodevelopmental mechanism that may contribute to schizophrenia.

via Neuroscience News|

#crispr #neurodevelopment #schizophrenia

science-and-technology24 days ago•184 min saved

Healthy longevity hinges on beating chronic disease, not aging gadgets

CNN argues that extending healthy life may depend as much on preventing chronic disease as on aging‑themed tech; while CRISPR gene editing and mRNA vaccines offer promising avenues against aging‑linked diseases, both remain early‑stage. In the meantime, proven prevention—Mediterranean diet, regular aerobic and strength training, adequate sleep, and social connection—can add healthy years now.

via CNN|

#aging #chronic-disease #crispr

biotech29 days ago•2 min saved

In vivo CRISPR therapy nears approval after striking Phase 3 results in HAE

Intellia Therapeutics’ Phase 3 study of lonvo-z, its in vivo CRISPR-based treatment for hereditary angioedema, showed an 87% reduction in attack rates versus placebo after a single dose, with over 60% of patients attack-free, prompting an FDA rolling submission and positioning lonvo-z as the second approved CRISPR medicine and the first in vivo gene-editing therapy.

via statnews.com|

#biotech #crispr #fda-rolling-submission

science29 days ago•3 min saved

Intellia's in vivo CRISPR therapy hits Phase 3 for hereditary angioedema, signaling a potential one-time treatment

Intellia Therapeutics said its one-time, in vivo CRISPR-based treatment for hereditary angioedema met its Phase 3 primary endpoint, reducing attacks by 87% vs placebo and achieving 62% attack-free patients at six months after a liver-directed infusion, with a favorable safety profile. The company has started a rolling FDA submission and targets a U.S. launch in 2027 if approved.

via CNBC|

#crispr #gene-editing #hereditary-angioedema

science1 month ago•74 min saved

Bacterial toxin hijacks claudin-4 to cleave E-cadherin, fueling colorectal cancer risk

Researchers identify claudin-4 as the receptor for enterotoxigenic Bacteroides fragilis toxin (BFT) and show that BFT binding to claudin-4 enables direct cleavage of the E-cadherin ectodomain at the cell surface, disrupting the epithelial barrier and triggering pro-carcinogenic signaling. A genome-wide CRISPR screen highlighted claudin-4 as a key determinant of BFT toxicity, with validation in multiple cell lines and a mouse model. Importantly, a soluble claudin-4 analogue blocks BFT activity both in vitro and in vivo, suggesting a potential therapeutic approach to prevent ETBF-associated colorectal neoplasia and infection.

via Nature|

#bft #claudin-4 #colorectal-cancer

health1 month ago•65 min saved

CRISPR-based sickle cell therapy earns $3M Breakthrough Prize for Thein and Orkin

Two researchers, Swee Lay Thein and Stuart Orkin, won the Breakthrough Prize in Life Sciences worth $3 million for work that led to Casgevy, a CRISPR-based therapy that functionally cures sickle cell disease and beta-thalassemia by disabling the BCL11A repressor to boost fetal hemoglobin. The treatment—developed with Vertex—involves editing a patient’s bone marrow cells and reinserting them, a costly, lengthy process with chemotherapy, and while a major milestone, access remains limited as scientists pursue in vivo approaches to broaden use.

via Live Science|

#bcl11a #beta-thalassemia #casgevy

science1 month ago•3 min saved

Researchers test CRISPR/XIST approach to turn off extra chromosome in Down syndrome cells

A study from Beth Israel Deaconess Medical Center and Harvard uses a modified CRISPR/Cas9 to insert the chromosome-silencing XIST RNA into trisomy 21 cells, silencing the extra chromosome in a subset of cell lines and offering a potential path toward chromosomal therapy for Down syndrome, but much work remains to assess safety, delivery, and whether effects translate to animals and humans.

via Gizmodo|

#chromosomal-therapy #crispr #down-syndrome

science1 month ago•2 min saved

Tiny CRISPR enzyme boosts in-body gene editing for targeted therapies

NIH-backed researchers identified the small Al3Cas12f CRISPR enzyme and engineered the Al3Cas12f RKK variant to dramatically improve editing in human cells, raising efficiency from under 10% to over 80% (up to 90% in a key region). This enzyme's size allows packaging into AAV delivery vectors, a major step toward in-body gene therapies for diseases like cancer and ALS; next, researchers will test AAV-packaged delivery to move toward clinical use.

via National Institutes of Health (.gov)|

#aav #cas12f #crispr

science2 months ago•12 min saved

Colossal’s de-extinction bid stirs science and ethics debate

Dallas-based Colossal Biosciences is pursuing revival of extinct species—including dire wolves and the dodo—using ancient DNA and CRISPR. The effort has drawn praise for pushing conservation tech and public engagement, but also sharp criticism from scientists who question feasibility and ecological risks, warn against conflating revived animals with extinct ones, and worry about undermining habitat protection; supporters see potential conservation benefits and genetic diversity applications, while critics urge caution and rigorous science.

via The Guardian|

#colossal-biosciences #conservation #crispr

health2 months ago•51 min saved

Miracle CRISPR treatment cures stage 4 colon cancer in a 12-patient trial

Emma Weston-Dimery, diagnosed with stage 4 colon cancer at 23, was one of 12 patients in a Phase 1 trial of CRISPR-Cas9 gene-edited immune cells. After cells were edited to better attack the cancer and reinfused in early 2023, two nodules disappeared within a month and the remaining cancer vanished by month two. She is now cancer-free, the only patient in the trial to achieve complete remission. The study showed mixed results for others, with some stabilization and several deaths due to the disease. A Phase 2 trial and a pill-form version of the therapy are planned, and Weston-Dimery is now an advocate for clinical trials and patient awareness.

via Daily Mail|

#cancer-survivor #cancer-trial #colon-cancer

technology2 months ago•16 min saved

Gene-edited fungus could turbocharge meat substitutes and slash environmental footprint

A gene-edited fungus used to make mycoprotein grows protein nearly twice as fast with far less sugar after deleting two genes with CRISPR to thin its cell wall; lifecycle analyses across six countries show substantial reductions in climate emissions, land use, and freshwater pollution, plus improved essential amino-acid quality. Regulators and large-scale production hurdles remain before it reaches diners.

via Earth.com|

#crispr #fungus #gene-editing

science2 months ago•6 min saved

Brain’s DeltaFosB as Switch Behind Cocaine Relapse, Study Finds

Chronic cocaine use rewires the brain by accumulating DeltaFosB in the ventral hippocampus–nucleus accumbens reward-memory circuit, altering gene expression and driving compulsive drug-seeking. CRISPR experiments show DeltaFosB is necessary for these changes, and calreticulin helps rev the brain’s engine toward cocaine seeking. With no FDA-approved meds for cocaine addiction, researchers are pursuing DeltaFosB-targeting compounds as a potential future treatment to reset brain circuits and reduce relapse.

via Neuroscience News|

#calreticulin #cocaine-addiction #crispr

science3 months ago•5 min saved

Brain Cells with a Built-In Defense Against Dementia Found

A CRISPR-based screen in human neurons with disease-causing tau mutations identified the CRL5SOCS4 protein complex as a key driver of tau protein degradation, helping some brain cells resist toxic tau clumps and dementia. Findings align with brain atlas data showing higher CRL5SOCS4 expression correlates with cell survival, and researchers note mitochondrial dysfunction can increase pathogenic tau fragments. Potential therapies include boosting CRL5SOCS4 activity or protecting proteasomes to enhance tau clearance.

via ScienceAlert|

#alzheimers-disease #crispr #mitochondria

science3 months ago•9 min saved

Coffee-Triggered CRISPR: A Reversible Jab at Precision Gene Therapy

Texas A&M researchers report a chemogenetic system that links CRISPR activity to caffeine, enabling controllable, reversible gene editing and T‑cell activation via a “caffebody,” with safety shutoffs using rapamycin. This approach aims for more precise cancer therapies and other long‑term treatments, pending further preclinical studies.

via SciTechDaily|

#caffeine #cancer-therapy #chemogenetics

science3 months ago•43 min saved

First-Ever Personalized CRISPR Therapy Helps Infant With CPS1 Deficiency Thrive

Researchers at Children’s Hospital of Philadelphia and the University of Pennsylvania developed a bespoke base-editing CRISPR therapy delivered to the liver to fix a CPS1 gene variant in a newborn with a rare metabolic disorder. After a first infusion in early 2025 and subsequent doses, the child has tolerated treatment with no adverse effects, has been able to halt medications and gradually reintroduce protein, and is thriving per a New England Journal of Medicine report; the approach is experimental and not FDA-approved, but signals a path toward patient-specific gene therapies that could be scalable to individual needs.

via Good News Network|

#cps1-deficiency #crispr #gene-therapy