Mutation News and Research

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Targeting protein fragment may improve Huntington disease treatments

Treatments that target a fragment of the mutant protein that causes Huntington's disease might be more effective than treatments, now in clinical trials, that target the whole protein but leave this fragment intact, a new study in mice suggests.

18 Mar 2026

Key gene mutation disrupts the brain’s ability to update beliefs in schizophrenia patients

One of the symptoms of schizophrenia is difficulty incorporating new information about the world. This can lead patients to struggle with making decisions and, eventually, to lose touch with reality.

18 Mar 2026

Study identifies brain pathway crucial for proper functioning of working memory

Working memory is a cognitive function that is essential for carrying out everyday activities and temporarily retaining information.

17 Mar 2026

Clinical trial tests a newer, targeted therapy for IDH mutant astrocytoma

A new clinical trial led by the Alliance for Clinical Trials in Oncology will investigate if a combination of drug therapies after radiation therapy improves outcomes for people with newly-diagnosed, grade 3 IDH-mutant astrocytoma, a type of brain cancer.

16 Mar 2026

Gene mutation that aids high-altitude survival could repair myelin sheath damage

A genetic mutation that helps animals like yaks and Tibetan antelopes survive at high altitudes may hold the key to repairing nerve damage in conditions such as cerebral paralysis and multiple sclerosis (MS).

13 Mar 2026

Combined CDK4/6 and EGFR inhibition improves pancreatic cancer therapy

Clinically available KRAS inhibitors mainly target G12C, which is rare in PDAC and often acquires resistance. Oncogenic KRAS inactivates RB1 via CDK4/6, while RB1 mutation is rare.

13 Mar 2026

Experimental therapy eliminates pancreatic precancerous cells in mice study

A new preclinical study in mice shows that precancerous cells in the pancreas can be eliminated before they have the chance to become tumors.

12 Mar 2026

Transposable elements cause majority of lethal mutations in fruit flies

Most lethal mutations in wild fruit flies are driven by newly transferred jumping genes, not small DNA errors, according to a new study from Duke University.

10 Mar 2026

Microbial ecosystems influence success of cancer checkpoint immunotherapy

Immune checkpoint inhibitors targeting PD-1 and PD-L1 have revolutionized cancer therapy, delivering long-term survival benefits in several malignancies.

9 Mar 2026

New optovolution technique uses light to guide protein evolution

Evolution is biology's powerful method of engineering. It works by generating many variants of DNA, RNA, and proteins inside cells and letting nature "select" the organism that performs best.

6 Mar 2026

Wnt signaling in fibroblasts drives gastric cancer metastasis to the liver

Researchers at the Cancer Research Institute and the Nano Life Science Institute (WPI-NanoLSI), Kanazawa University, have uncovered a critical mechanism that enables gastric cancer to spread to distant organs.

6 Mar 2026

MSK uncovers how interacting mutations shield breast cancer

Researchers at Memorial Sloan Kettering Cancer Center (MSK) have made an important discovery about how genetic mutations in breast cancer patients can interact and drive resistance to certain drugs called CDK4/6 inhibitors. This finding, published in Nature, suggests a new strategy for predicting and preventing resistance to specific therapies based on the tumor's genetic profile.

6 Mar 2026

AI trained on 9 trillion DNA letters predicts harmful mutations and designs new genomes

Scientists developed Evo 2, a large biological foundation model trained on about 9 trillion DNA base pairs that can analyse genomic sequences across bacteria, plants, and humans. The model predicts functional effects of genetic variants and can generate genome-scale DNA sequences, enabling new approaches to studying and designing biological systems.

5 Mar 2026

Researchers find novel path to treat Rett syndrome

A team of researchers at Texas Children's Duncan Neurological Research Institute (NRI) and Baylor College of Medicine report in Science Translational Medicine a potential new approach to treat Rett syndrome – offering early promise for a rare neurodevelopmental disorder that currently has no cure.

5 Mar 2026

Novel gene regulation therapy passes key clinical milestone

Groundbreaking Phase 1/2a clinical trials co-led by Linda Laux, MD, from Ann & Robert H. Lurie Children's Hospital of Chicago, show that the first gene regulation treatment for epilepsy is safe and well tolerated by patients with Dravet syndrome for whom antiseizure medications are not effective.

4 Mar 2026

AI cancer tools may be using visual shortcuts rather than true biology

New research warns that popular deep learning systems trained for cancer pathology may be relying on hidden shortcuts rather than genuine biological signals.

2 Mar 2026

Targeted fluorescent probes for high-contrast cancer detection

Cancer is a universal issue that affects uncountably many people around the world. Many will turn to surgery in the hope that a surgeon will be able to completely remove a tumor, leaving healthy tissues unaffected. Various tools and techniques have been developed over the years to improve the way these surgeries are performed, and visual imaging methods such as glowing dyes have proven to be very useful.

2 Mar 2026