Gene Editing News and Research

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Breakthrough research identifies pathway used by glioblastoma to infiltrate the brain

A new pathway that is used by cancer cells to infiltrate the brain has been discovered by a team of Canadian and American research groups led by the Singh Lab at McMaster University.

2 Aug 2024

Using advanced CRISPR to boost efficiency of CAR-T cell therapies

CAR-T cells are highly effective in treating selected blood cancers. However, challenges remain with this new therapy, which was first approved in 2017 in the USA and a year later in Europe for treating acute lymphoblastic leukemia (ALL).

2 Aug 2024

Understanding iron overload in β-thalassemia: Mechanisms, diagnosis, and treatments

β-Thalassemia is a genetic disorder characterized by reduced or absent synthesis of the beta chains of hemoglobin, leading to ineffective erythropoiesis and severe anemia.

29 Jul 2024

Scientists explore early life factors contributing to SMA

Spinal muscular atrophy (SMA) is a severe neurological disease for which there is presently no cure, although current therapies can alleviate symptoms. In the search for better treatment options, scientists at DZNE and the Dresden University of Technology are now drawing attention to previously unnoticed abnormalities in embryonic development. They base their argument on studies of so-called organoids: Laboratory-grown tissue cultures that can reconstruct disease processes. Their findings are published in the journal Cell Reports Medicine.

27 Jul 2024

Understanding the epigenetic regulation of hepatic stellate cells in NAFLD

Non-alcoholic fatty liver disease (NAFLD) has emerged as a leading cause of chronic liver disease worldwide, affecting approximately 38% of the global population from 2016 to 2019.

25 Jul 2024

Creating the first genetically susceptible mice for late-onset Alzheimer's

Mice don't get Alzheimer's - and while that's good news for mice, it's a big problem for biomedical researchers seeking to understand the disease and test new treatments.

24 Jul 2024

Broken String Biosciences appoints Steve Becker as Chief Commercial Officer

Broken String Biosciences (“Broken String”), a genomics company driving development of the next generation of more precise, safe, and effective cell and gene therapies, today announced it has appointed Steve Becker as Chief Commercial Officer.

24 Jul 2024

USC scientists transform B cells into cancer and HIV destroyers

USC scientists have discovered a way to turn the body's B cells into tiny surveillance machines and antibody factories that can pump out specially designed antibodies to destroy cancer cells or HIV, two of medicine's most formidable foes.

22 Jul 2024

Common blood thinner can be repurposed as an inexpensive antidote for cobra venom

Scientists at the University of Sydney and Liverpool School of Tropical Medicine have made a remarkable discovery: a commonly used blood thinner, heparin, can be repurposed as an inexpensive antidote for cobra venom.

18 Jul 2024

CU Boulder research uncovers role of endogenous retroviruses in cancer

Peek inside the human genome and, among the 20,000 or so genes that serve as building blocks of life, you'll also find flecks of DNA left behind by viruses that infected primate ancestors tens of millions of years ago.

18 Jul 2024

Harnessing cell's natural delivery system for targeted drug delivery

Each cell in the body has its own unique delivery system that scientists are working on harnessing to move revolutionary biological drugs - molecules like proteins, RNA and combinations of the two - to specific diseased parts of the body.

17 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

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