Tag

Crispr

All articles tagged with #crispr

cancer-genetics2 days ago•51 min saved

Arm-level CNAs reveal hidden breast cancer drivers via CRISPR screens

Researchers developed the CRISPR-KOALA platform to perform high-throughput bidirectional gene screens on recurrent arm-level copy-number alterations in immunocompetent mouse models of basal-like breast cancer. Screening 3,752 genes, they identified 90 cancer driver genes across signaling pathways such as MAPK, HIPPO and WNT, showing that arm-level CNAs can selectively recruit diverse drivers and that manipulating these genes can bypass the requirement for aneuploidy in TP53-mutant BLBC. Notably, PLGRKT on chromosome 9p emerged as a potent oncogene linked to stress-resistant mitochondria and enhanced ROS detox, illustrating how specific drivers may shape tumor heterogeneity driven by aneuploidy.

via Nature|

#aneuploidy #basal-like-breast-cancer #cancer-genetics

science3 days ago•194 min saved

Embryo base editing nudges germline research toward reality, intensifying designer-baby debate

New studies using base editing in early human embryos show precision editing with fewer chromosomal errors but still face mosaicism and off-target effects. While these results advance understanding of embryo development and disease-related genes, experts warn that safety, ethical, and regulatory hurdles remain, keeping the door to germline editing and “designer babies” firmly closed for now rather than opened.

via CNN|

#base-editing #crispr #embryos

science3 days ago•77 min saved

Prime editing maps essential histone H3 lysines in mammalian cells

Researchers developed a high-throughput CRISPR prime-editing platform to mutate all canonical histone H3 genes in mouse embryonic stem cells and map lysine requirements for cell fitness. The screen pinpointed key residues (H3K4, H3K9, H3K14, H3K18, H3K79) whose disruption reduces fitness, with H3K56 playing a conserved role in genome stability; combinatorial edits reveal functional crosstalk (for example, H3K27R + H3K36R impairs self-renewal and transcription). The approach can occasionally generate kilobase-scale deletions in histone clusters when nicking is used, but careful screening yields clean clones, establishing a functional map of H3 lysines and a versatile toolkit for studying chromatin regulation in mammals.

via Nature|

#crispr #epigenetics #histone-h3

technology13 days ago•10 min saved

Epigenetic editing goes from concept to clinic in biotech startups

A new wave of biotech startups is pursuing targeted epigenetic editing—modifying DNA-associated chemical markers to turn genes on or off—aiming to treat diseases from muscular dystrophy to hepatitis B; companies like Epicrispr and nChroma are leading with dead-Cas9/Cas12F tools and refined methyltransferases, reporting preclinical success and early human dosing, while researchers caution that long‑term safety and off‑target effects remain critical as this approach moves toward the clinic.

via Nature|

#biotechnology-startups #crispr #epigenetic-editing

biology28 days ago•6 min saved

A single gene speeds growth but shortens lifespan in aging killifish

A Nature Communications study shows the vgll3 gene influences how fast African turquoise killifish grow and reach reproductive age, with CRISPR edits accelerating growth and puberty but increasing late-life tumors and shortening lifespan, providing rare in-vertebrate evidence for antagonistic pleiotropy and suggesting a trade-off between early-life benefits and later-life disease; the findings hint that similar mechanisms could partly explain aging in humans and motivate research to separate growth benefits from cancer risks.

via ScienceAlert|

#aging #antagonistic-pleiotropy #biology

science29 days ago•45 min saved

CRISPR’s Next Act: From Bacterial Defense to One-Time Therapies and Global Ethics

Jennifer Doudna describes how CRISPR-Cas9 evolved from a bacterial immune system into a programmable gene-editing tool, its rapid uptake across medicine and agriculture, and the ongoing delivery challenges to different tissues. The discussion covers a landmark 2024 liver-targeted therapy for Baby KJ, the prospect of one-time genetic treatments, and the ethical, regulatory, and accessibility questions raised by germline edits (notably the 2018 case), underscoring the need for cautious public engagement as the CRISPR toolbox expands.

via Quanta Magazine|

#crispr #delivery #ethics

science1 month ago•12 min saved

RNA-Driven Chromatin Shredding Targets Cancer Mutations

Researchers engineered an RNA-guided CRISPR-Cas12a2 nuclease to recognize cancer-specific transcripts and trigger transcript-activated chromatin shredding, leading to DNA damage responses and selective death of cancer cells in experimental systems, offering a potential precision approach to targeting undruggable mutations such as p53.

via Nature|

#cancer #chromatin #crispr

science1 month 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-technology2 months 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

biotech2 months 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

science2 months 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

science2 months 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

health2 months 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

science2 months 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

science2 months 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