Gene Editing News and Research

RSS

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

CRISPR screening uncovers microproteins involved in fat storage

The obesity rate has more than doubled in the last 30 years, affecting more than one billion people worldwide.

7 Aug 2025

Cedars-Sinai study finds new way to combat Alzheimer’s in preclinical models

In a preclinical study, Cedars-Sinai investigators found that boosting angiotensin converting enzyme (ACE) in key immune cells called microglia in the brain helped protect the brains of specially bred laboratory mice against Alzheimer's disease.

6 Aug 2025

Prime editing reverses symptoms of severe childhood neurological disease

Scientists use a precise form of gene editing called prime editing to correct the most common genetic mutations that cause alternating hemiplegia of childhood, a rare and severe neurological disorder that begins in infancy.

From The Jackson Labs 6 Aug 2025

Apple snail eye regeneration offers new insights into human sight restoration

The eye of the apple snail is unusually similar to a human eye-but, unlike human eyes, it can regrow itself if injured or even amputated.

6 Aug 2025

Targeting lactate metabolism offers new avenues for cancer treatment

Lactic acid (LA) has transitioned from being perceived as a mere glycolytic waste product to a pivotal regulator of tumor-immune crosstalk.

5 Aug 2025

Researchers classify how specific genetic mutations correspond to rare disorders

The NF-κB signaling pathway plays a pivotal role in how our bodies fight infection, heal, and regulate inflammation. At the heart of this pathway is the NEMO protein, encoded by the IKBKG gene.

30 Jul 2025

New RNA therapy reverses symptoms of progeria in mouse models

Children who develop deep wrinkles, stunted growth, and rapidly aging bones and blood vessels as early as 1 to 2 years of age may be suffering from Hutchinson-Gilford Progeria Syndrome (HGPS), a rare and incurable genetic disorder that affects approximately one in eight million people.

29 Jul 2025

New method genetically blocks mosquitoes from transmitting malaria

Mosquitoes kill more people each year than any other animal. In 2023, the blood-sucking insects infected a reported 263 million people with malaria, leading to nearly 600,000 deaths, 80% of which were children.

23 Jul 2025

Astrocytes play key role in coordinating neurons for visual information

Cells called astrocytes are about as abundant in the brain as neurons, but scientists have spent much less time figuring out how they contribute to brain functions.

22 Jul 2025