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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

Twincretin drug Tirzepatide shows unmatched efficacy in weight loss and diabetes management

Tirzepatide, a novel twincretin drug, significantly improves weight loss and glycemic control in obese and type 2 diabetes patients, outperforming traditional treatments in clinical trials.

15 Jul 2024

Lifestyle changes can greatly reduce heart disease risk in people with high genetic risk

Study highlights the significant impact of genetic risk and lifestyle on early- and late-onset cardiovascular diseases, showing that lifestyle improvements can notably reduce CVD risk, especially in high genetic risk individuals.

14 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

Neighborhood disadvantage linked to higher expression of stress-related genes in prostate tumors

Those living in disadvantaged neighborhoods have significantly higher activity of stress-related genes, new research suggests, which could contribute to higher rates of aggressive prostate cancer in African American men.

12 Jul 2024

Optical metabolic imaging reveals how Toxoplasma infection changes host cell metabolism

Toxoplasma gondii is a parasite responsible for toxoplasmosis, a lifelong chronic infection prevalent in about 30% of the human population.

12 Jul 2024

Immune cell inflammation may be responsible for some severe symptoms in lysosomal storage diseases

Researchers at The Hospital for Sick Children (SickKids) have found that inflammation in an immune cell may be responsible in part for some severe symptoms in a group of rare genetic conditions called lysosomal storage diseases (LSDs).

12 Jul 2024

Enzymatic RNA synthesis paves the way for sustainable therapeutics

While the COVID-19 vaccines introduced many people to RNA-based medicines, RNA oligonucleotides have already been on the market for years to treat diseases like Duchenne Muscular Dystrophy and amyloidosis.

12 Jul 2024

Study finds early, unnecessary opioid prescriptions for teen surgery

A multi-institutional study found that 1 in 6 youths fill an opioid prescription prior to surgery, and 3% of patients were still filling opioid prescriptions three to six months after surgery, indicating persistent opioid use and possible opioid dependence.

12 Jul 2024

New tech shows promise for mental wellbeing

One of the intriguing abilities of the human mind is daydreaming, where the mind wanders off into spontaneous thoughts, fantasies and scenarios, often without conscious effort, allowing creativity and reflection to flow freely.

12 Jul 2024

How lipoproteins shape your metabolic health

Study provides a comprehensive review of the lipoprotein pathway and its impact on healthy metabolism, highlighting the roles of HDL, LDL, and other lipoproteins in cardiovascular health and disease.

11 Jul 2024

Tiny force sensors in embryos pave way for new birth defect treatments

A group of scientists at UCL have successfully created mechanical force sensors directly in the developing brains and spinal cords of chicken embryos, which they hope will improve understanding and prevention of birth malformations such as spina bifida.

11 Jul 2024

ARPP21 gene mutation could be the cause of ALS

Researchers from the Neuromuscular Diseases Group and the Dementia Neurobiology Group of the Sant Pau Research Institute (IR Sant Pau) and the Memory Unit of the Sant Pau Hospital, led by neurologist Dr. Ricard Rojas-García, have identified a new mutation in the ARPP21 gene that could be the cause of Amyotrophic Lateral Sclerosis (ALS), a devastating neurodegenerative disease.

11 Jul 2024

Unveiling late-onset combined immunodeficiency in patients with 18q del syndrome

Chromosome 18q deletion (18q del) syndromeis a rare genetic condition disorder, affecting approximately 1 in 40,000 to 55,000 individuals, caused by the deletion of genetic material on the long arm of chromosome 18.

11 Jul 2024

Researchers discover a new mechanism of resistance to KRAS inhibitors in pancreas cancer

Mutations in the KRAS gene are the primary driver of pancreatic cancer. A new class of drugs known as KRAS inhibitors is showing initial promise in clinical trials, but many tumors quickly develop resistance to the treatment.

11 Jul 2024

New study reveals the genetic cause of Parkinson's disease

According to Science Alert, neuroscientists from Johns Hopkins University have recently discovered a new treatment for Parkinson's disease using an FDA-approved cancer drug.

11 Jul 2024

Study reveals how DDM1 protein suppresses 'jumping genes' in plants

International joint research led by Akihisa Osakabe and Yoshimasa Takizawa of the University of Tokyo has clarified the molecular mechanisms in thale cresses (Arabidopsis thaliana) by which the DDM1 (Decreased in DNA Methylation 1) protein prevents the transcription of "jumping genes."

11 Jul 2024

Distinct T-cell signatures identified in children with type 1 diabetes

A study conducted at the University of Eastern Finland found distinct signatures in CD8-positive T cells in blood samples from children with newly diagnosed type 1 diabetes and in autoantibody-positive children who later developed type 1 diabetes.

11 Jul 2024

Prime editing offers promising path to permanent treatment for cystic fibrosis

Cystic fibrosis is one of the most common genetic disorders, causing thick mucus build-up in the lungs and other parts of the body, breathing problems, and infection.

11 Jul 2024

Novel Co-STAR cells show promise in targeting and eradicating cancer

Using genetic engineering techniques, investigators at the Johns Hopkins Kimmel Cancer Center and its Ludwig Center, the Lustgarten Laboratory and Bloomberg~Kimmel Institute for Cancer Immunotherapy have designed a novel type of cell to recognize and fight cancer.

11 Jul 2024