Gene Editing News and Research

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New technology embeds unique genetic identifiers in engineered cells

Genetically engineered cell lines used in biomedical research have long been prone to misidentification and unauthorized use, wasting billions of dollars each year and jeopardizing critical scientific discoveries.

5 Sep 2025

Breakthrough reveals how viruses efficiently pack genetic material

Researchers at San Diego State University and Michigan State University are shedding new light on how viruses meticulously pack their genetic material - a breakthrough that could help researchers engineer antivirals and gene therapies.

2 Sep 2025

Nanostructures boost CRISPR delivery for genetic medicine

With the power to rewrite the genetic code underlying countless diseases, CRISPR holds immense promise to revolutionize medicine. But until scientists can deliver its gene-editing machinery safely and efficiently into relevant cells and tissues, that promise will remain out of reach.

2 Sep 2025

Scientists discover molecular brake that limits T cell attack on tumors

In a discovery that could expand the array of current cancer immunotherapy treatments, scientists at Harvard Medical School have identified a new molecular brake that hinders the ability of T cells to attack tumors.

1 Sep 2025

BindCraft reimagines protein binder discovery through structural prediction

Physical interactions between proteins influence anything from cell signaling and growth to immune responses, so the ability to control these interactions is of great interest to biologists.

29 Aug 2025

Clonogenic hepatocytes identified as engines of liver development

In a new study published in the Journal of Hepatology, researchers from the San Raffaele Telethon Institute for Gene Therapy (SR-Tiget) show that only 15–20% of hepatocytes in newborn mice - dubbed clonogenic hepatocytes - are responsible for generating over 90% of the adult liver mass.

29 Aug 2025

Gene editing therapy exa-cel improves quality of life in sickle cell disease and beta thalassemia

Treatment with exagamglogene autotemcel (exa-cel) led to robust and sustained improvements in quality of life for patients with severe sickle cell disease (SCD) or transfusion-dependent beta thalassemia, according to two studies published in Blood Advances.

27 Aug 2025

Yale researchers uncover new RNA editing tools for safer genetic surgery

The ability to correct disease-causing genetic mistakes using genome editors holds great promise in medicine, but it is not without risk.

21 Aug 2025

Using light to control tissue folding in developing embryos

The complex 3D shapes of brains, lungs, eyes, hands, and other vital bodily structures emerge from the way in which flat 2D sheets of cells fold during embryonic development.

21 Aug 2025

New genome-wide CRISPR screening tool boosts natural killer cells to fight cancer

Natural killer (NK) cells became markedly better at killing cancer cells after scientists removed key gene targets identified through a new genome-wide CRISPR screening tool, according to new research from The University of Texas MD Anderson Cancer Center.

21 Aug 2025

Breakthroughs in diagnosis and therapy for transthyretin amyloidosis

Transthyretin amyloidosis (ATTR) is caused by the misfolding and aggregation of the transthyretin (TTR) protein, leading to multi-organ damage.

21 Aug 2025

Improving gene therapy safety with human kidney organoids

Ryuji Morizane, MD, PhD, of the Department of Medicine at Massachusetts General Hospital, is the senior/corresponding author of a new paper published in Signal Transduction and Targeted Therapy, "AAV for gene therapy drives a nephrotoxic response via NFκB in kidney organoids."

19 Aug 2025

New strategies to tackle resistant foodborne pathogens

Innovative approaches in food and beverage analysis are transforming pathogen detection and control, emphasizing the role of natural compounds and sequencing.

19 Aug 2025

Targeting a sugar metabolism pathway offers hope for treating rare and aggressive childhood cancer

A new study delving into the genetic drivers of a rare and aggressive childhood cancer called Malignant Peripheral Nerve Sheath Tumor (MPNST) has revealed metabolic frailties in the cancer cells that might be exploited to improved treatments for patients.

18 Aug 2025

New generation of CRISPR shows safer path to treating genetic diseases

A new generation of CRISPR technology developed at UNSW Sydney offers a safer path to treating genetic diseases like Sickle Cell, while also proving beyond doubt that chemical tags on DNA - often thought to be little more than genetic cobwebs - actively silence genes.

17 Aug 2025

Gene editing restores hearing and balance in adult mice with DFNA41 deafness

Zheng-Yi Chen, DPhil, associate scientist at the Eaton-Peabody Laboratories, and Ines and Fredrick Yeatts Chair in Otolaryngology, at Mass Eye and Ear, is the senior and co-corresponding author of a paper published in the Journal of Clinical Investigation, "Single Dose Genome Editing Therapy Rescues Auditory and Vestibular Functions in Adult Mice with DFNA41 Deafness."

14 Aug 2025

New mouse model reveals key role of osteocytes in osteogenesis imperfecta

Osteogenesis imperfecta (OI) refers to a group of rare genetic bone disorders that results in the formation of fragile bones. In patients with OI, the matrix that makes up the bone has been found to be abnormal, leading to an increased risk of fractures.

13 Aug 2025

New method greatly improves the precision of genome editing

A research team headed by the University of Zurich has developed a powerful new method to precisely edit DNA by combining cutting-edge genetic engineering with artificial intelligence.

12 Aug 2025

Decoding macrophage immune responses with gene editing and machine learning

Fighting off pathogens is a tour de force that must happen with speed and precision. A team of researchers at CeMM and MedUni Vienna led by Christoph Bock and Matthias Farlik has investigated how macrophages-immune cells that are the body's first responders-master this challenge.

8 Aug 2025