Genome Editing News and Research

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One-time prime editing targets common genetic cause of cystic fibrosis

Researchers optimized prime editing for the efficient correction of the cystic fibrosis transmembrane conductance regulator F508del mutation in human airway epithelial cells, achieving high correction efficiencies and functional restoration with minimal off-target effects.

15 Jul 2024

Gene editing silences mutant microRNA, restores hearing in adult mice

Researchers have used gene editing to restore hearing in adult mice with a type of inherited hearing loss. They showed that shutting down a damaged copy of a gene called a microRNA (miRNA) enabled the animals to regain hearing. The approach by a research team supported by the National Institutes of Health (NIH), reported in Science Translational Medicine, may eventually lead to potential treatments for inherited hearing loss in people.

13 Jul 2024

New gene editing technique offers hope for millions

A team led by Mass Eye and Ear researchers has demonstrated for the first time a successful restoration of hearing through a novel, in vivo genome editing approach in an adult mouse model with a form of inherited deafness caused by mutations in microRNA.

11 Jul 2024

Efficient multi-site genome editing with retron-derived multitrons

Genome editing has become a widely adopted technology to modify DNA in cells, allowing scientists to study diseases in the lab and develop therapies that repair disease-causing mutations.

10 Jul 2024

Scientists identify over 5,000 genetic variants that enable certain cancers to thrive

Researchers performed saturation genome editing of the BAP1, whose dysfunction is related to cancer and impaired neurodevelopment.

10 Jul 2024

Scientists identify over 5000 high-risk cancer gene variants

Over 5,000 genetic variants that enable certain cancers to thrive have been identified by scientists, along with a potential therapeutic target to treat or even prevent these cancers from developing.

8 Jul 2024

Scientists map all the possible outcomes of changes to key tumor-suppressing gene

Researchers at the Francis Crick Institute have mapped all the possible outcomes of changes to a tumor-suppressing gene called VHL, the first step in a huge research endeavor to unpick the outcomes of tens of thousands of genetic variations in genes associated with cancer.

5 Jul 2024

New hope for solid tumors with enhanced CAR-T cells

A promising therapy that treats blood cancers by harnessing the power of the immune system to target and destroy cancer cells could now treat solid tumors more efficiently. Thanks to a recent study published in Molecular Therapy – Methods & Clinical Development from Dan Cappabianca and Krishanu Saha at the Wisconsin Institute for Discovery, Chimeric Antigen Receptor (CAR) T-cell therapy can be improved by altering the conditions the T cells are grown in. And it was all discovered by chance.

3 Jul 2024

Study provides resource for investigating diabetes-linked transcription factors

Scientists have generated a comprehensive map of the gene targets regulated by the transcription factors HNF4A and HNF1A in human pancreatic beta cells and liver cells. Published in the journal Nature Communications, the study revealed common and tissue-specific molecular pathways regulated by HNF4A and HNF1A, two proteins that possess important functions governing the development and function of the pancreas and liver.

26 Jun 2024

KU researchers uncover role of Src gene in nervous system development

Many people are familiar with oncogenes -; genes long known to be involved in cancers in humans, such as the gene "Src."

24 Jun 2024

Sphere Fluidics launches Cyto-CellectPLUS to accelerate cell line development workflows

Next-generation assay provides a rapid and cost-effective solution to quantify IgG production in single cells.

From Sphere Fluidics 4 Jun 2024

Two new studies offer insights into the molecular biology of neuropsychiatric disease

Working under the umbrella of the PsychENCODE Consortium, the mental health research project established in 2015 by the National Institutes of Health, a team of Mount Sinai scientists has uncovered important new insights into the molecular biology of neuropsychiatric disease through two new studies published in a special issue of Science on Friday, May 24.

24 May 2024

New MOBE technology revolutionizes multiplexed genome editing

The human genome consists of around 3 billion base pairs and humans are all 99.6% identical in their genetic makeup.

21 May 2024

New study finds Sfrp1 as a potential therapeutic target in cancer treatment

Cancer is a leading cause of death globally. One of the primary reasons why cancer is such a deadly disease is the ability of cancer cells to become drug-resistant.

15 May 2024

New modular epigenome editing platform sheds light on gene regulation mechanisms

Understanding how genes are regulated at the molecular level is a central challenge in modern biology.

9 May 2024

FAU researchers receive grants to tackle Alzheimer's disease

Three Florida Atlantic University researchers at the forefront of Alzheimer's disease (AD) research have each received a $350,000 grant from the Florida Department of Health's "Ed and Ethel Moore Alzheimer's Disease Research Program."

8 May 2024

CRISPR gene editing shows promise for treating individuals with a form of inherited blindness

Results from a groundbreaking clinical trial of CRISPR gene editing in 14 individuals with a form of inherited blindness show that the treatment is safe and led to measurable improvements in 11 of the participants treated.

6 May 2024

GenE-HumDi's strategy for involving young scientists in cutting-edge research

COST Actions are meant to be inclusive which means ensuring a geographic spread of participants, encouraging gender balance, and involving young researchers.

3 May 2024

Timelapse breakthrough: Scientists capture secrets of early embryonic cell division

The beginning of life is shrouded in mystery. While the intricate dynamics of mitosis is well-studied in the so-called somatic cells – the cells that have a specialized function, like skin and muscle cells – they remain elusive in the first cells of our bodies, the embryonic cells.

24 Apr 2024

Meta-analysis uncovers stress-responsive genes in Arabidopsis

Plants can be temperamental. Even weeds along the side of highways or pushing their way up in the cracks of concrete sidewalks can get stressed out by dehydration, cold, excess salt and more.

12 Apr 2024