Gene Editing News and Research

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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

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

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

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

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

Blocking nidogen-2 enhances chemotherapy efficacy in pancreatic cancer

Researchers at the Garvan Institute of Medical Research have identified a potential new therapeutic target for pancreatic cancer, one of the most lethal cancer types with limited treatment options.

9 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

AI uncovers potential cancer drivers hidden in 'junk' regions of DNA

Using artificial intelligence, Garvan Institute researchers have found potential cancer drivers hidden in so-called 'junk' regions of DNA, opening up possibilities for a new approach to diagnosis and treatment.

2 Jul 2024

CHARMs: A new hope for prion disease therapy

Drug development is typically slow: the pipeline from basic research discoveries that provide the basis for a new drug to clinical trials to production of a widely available medicine can take decades.

27 Jun 2024

New curative treatment for sickle cell disease shows promising results in international trial

Vanderbilt University Medical Center (VUMC) researchers are touting data from a multicenter, international phase 2 clinical trial showing a new, curative treatment for sickle cell disease (SCD).

25 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

Artificial variants of Withanolides act highly specifically against leukemia cells

Nightshade plants produce a diverse array of compounds with therapeutic potential. Researchers at CeMM have now identified an artificial variant inspired by the Withanolides group that acts highly specifically against leukemia cells.

21 Jun 2024

Promising gene editing therapy shows hope in sickle cell disease cure

Researchers have presented the latest findings from a clinical trial aimed at discovering a cure for sickle cell disease, a painful genetic blood disorder with limited treatment options.

14 Jun 2024

Efficient whole-gene insertions can be achieved with improved gene-editing technology

Scientists at the Broad Institute of MIT and Harvard have improved a gene-editing technology that is now capable of inserting or substituting entire genes in the genome in human cells efficiently enough to be potentially useful for therapeutic applications.

10 Jun 2024