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Crispr

All articles tagged with #crispr

Brain Cells with a Built-In Defense Against Dementia Found
science6 days ago

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
First-Ever Personalized CRISPR Therapy Helps Infant With CPS1 Deficiency Thrive
science22 days ago

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
Diverse autism mutations converge on a shared chromatin-regulation network in stem-cell–derived human cortex
science27 days ago

Diverse autism mutations converge on a shared chromatin-regulation network in stem-cell–derived human cortex

A large hiPSC-based study generated cortical organoids from eight ASD-risk mutations, idiopathic ASD, and controls, profiling gene expression across 25–100 days. Early mutation-specific changes give way to convergent transcriptional and chromatin-regulatory disruptions enriched for ASD risk genes, including SWI–SNF components. CRISPRi validation supports key regulators driving this convergent network, suggesting that diverse genetic risks in ASD propagate through shared transcriptional pathways that affect early neurodevelopment, while idiopathic cases show less convergence.

via Nature|
#autism#crispr#ipsc
CRISPR breakthrough activates genes without DNA cuts
science1 month ago

CRISPR breakthrough activates genes without DNA cuts

Scientists at UNSW Sydney have developed a new CRISPR-based epigenetic editing technique that can turn genes on without cutting DNA, by removing chemical methyl groups that silence genes. This approach could lead to safer gene therapies for conditions like Sickle Cell disease, as it avoids the risks associated with DNA strand breaks. The research demonstrates that methylation directly controls gene activity and opens new possibilities for treating genetic disorders by reactivating silenced genes.

via ScienceDaily|
#crispr#dna-methylation#epigenetic-editing
CRISPR Innovation Promises New Era in Genetic Disease Therapy
science1 month ago

CRISPR Innovation Promises New Era in Genetic Disease Therapy

Researchers at UNSW Sydney have developed a safer CRISPR-based epigenetic editing technique that can switch genes on and off without cutting DNA, offering promising new treatments for genetic diseases like Sickle Cell by reactivating silenced genes through removal of methyl groups, potentially reducing risks associated with traditional gene editing.

via SciTechDaily|
#crispr#dna-methylation#epigenetic-editing
India's First Gene-Edited Sheep Tarmeem: An Update
science-and-technology2 months ago

India's First Gene-Edited Sheep Tarmeem: An Update

India's first gene-edited sheep, Tarmeem, has turned one year old and is thriving, showing increased muscle growth due to CRISPR technology targeting the myostatin gene. Researchers aim to use this technology to enhance meat production sustainably in Kashmir, potentially revolutionizing local agriculture and addressing food security issues. The project marks a significant step in applying gene editing in livestock within India, though regulatory and ethical considerations remain.

via BBC|
#crispr#gene-editing#india
2025 Space Discoveries: New Moons, Mergers, and Future Horizons
science2 months ago

2025 Space Discoveries: New Moons, Mergers, and Future Horizons

Despite political challenges in 2025, significant scientific achievements were made, including the detection of the strongest gravitational signals from black hole mergers, advancements in nuclear fusion, innovative gene therapies, and breakthroughs in chemical recycling and climate research, highlighting a resilient and innovative scientific community.

via Boing Boing|
#black-holes#crispr#gravitational-waves