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Study explains why some AML patients do not respond to standard treatment

An international study led by the University of Colorado Cancer Center has uncovered why a widely used treatment for acute myeloid leukemia (AML) doesn't work for everyone.

21 May 2025

Personalized gene editing corrects fatal infant metabolic disorder in world-first treatment

Researchers have used a personalised base-editing gene therapy to successfully treat an infant with carbamoyl-phosphate synthetase 1 (CPS1) deficiency, a rare and life-threatening inborn error of metabolism. This pioneering approach chemically corrects the underlying mutation without cutting DNA, showing promise for precision treatment of ultrarare genetic disorders.

20 May 2025

Combination of two drugs shows promise in treating B-cell acute lymphoblastic leukemia

A combination of two drugs could improve outcomes and reduce the need for toxic chemotherapy for B-cell acute lymphoblastic leukemia (B-ALL), the commonest cancer in childhood and one that can be particularly difficult to treat in older patients, according to Cambridge scientists.

20 May 2025

What makes H5N1 a pandemic risk and how can we stop it?

A new PEARLS article in PLoS Pathogens reviews the rapid evolution and pandemic potential of emerging influenza A viruses, with a focus on H5N1. It highlights scientific advances and future strategies to outpace the virus, from next-generation vaccines to AI-driven antiviral discovery.

19 May 2025

Study uncovers common trigger across gene mutations that cause ALS

Using the gene scissors CRISPR and stem cells, researchers at Stockholm University and the UK Dementia Research Institute (UK DRI) at King's College London have managed to identify a common denominator for different gene mutations that all cause the neurological disease ALS.

19 May 2025

Targeting PME-1 may offer new strategies for treating neurological disorders and cancer

PME-1 is a protein that modifies other proteins by removing methyl groups, but high levels of PME-1 are observed in some diseases, including Alzheimer's disease (AD) and cancer.

16 May 2025

Study reveals subtle effects of recessive mutations on health and reproduction

As a group, carriers of recessive disorders are slightly less healthy and have a reduced chance of having offspring.

16 May 2025

HIV vaccine study marks breakthrough in targeting broad immune responses

A decades-long scientific challenge in HIV vaccine development has been finding a way to train the immune system to produce antibodies that can target many variants of the virus.

15 May 2025

Cardiac MRI could help detect lamin heart disease

Magnetic resonance imaging (MRI) scans of the heart could help to detect a life-threatening heart disease and enable clinicians to better predict which patients are most at risk, according to a new study led by UCL (University College London) researchers.

14 May 2025

DiffInvex identifies key genes driving chemotherapy resistance

Just as species adapt over generations, our body's cells accumulate DNA changes throughout life. Most are harmless, yet a few "driver" mutations give a cell a competitive edge and can spark cancer.

13 May 2025

‘Cell line atlas’ can help better understand biliary tract cancer biology

Advanced biliary tract cancer (BTC) includes cholangiocarcinoma, gallbladder carcinoma, and ampullary carcinoma. BTC is a rare and aggressive group of cancers, carrying one of the worst prognoses in all of oncology.

12 May 2025

HER3 reemerges as a key driver of cancer progression and therapy resistance

A new publication shines a spotlight on HER3, a long-overlooked member of the ErbB receptor family, revealing its critical role in cancer progression and resistance to therapy.

12 May 2025

New model reveals H5N1 is spreading undetected in US dairy herds

Researchers developed a nationwide model to simulate the spread of H5N1 in US dairy cattle, revealing significant under-reporting and predicting continued outbreaks into 2025. Targeted farm-level biosecurity and surveillance are urgently needed to contain the epidemic.

11 May 2025

Newly identified gene mutation causes severe form of Fanconi anemia

Fanconi anemia is an aggressive, life-threatening disorder. Most individuals living with this rare genetic condition, characterized by bone marrow failure and cancer predisposition, survive into adulthood only with bone marrow transplantation and regular cancer screening.

11 May 2025

St. Jude Children's scientists use brain organoids to study rare encephalopathy

While extremely rare, encephalopathy (a condition affecting brain function) triggered by mutations in the UBA5 gene has devastating impacts, with affected individuals reaching few developmental milestones and experiencing frequent and early-onset seizures.

8 May 2025

Monoclonal antibody protects mice from lethal influenza A doses

Scientists have engineered a monoclonal antibody that can protect mice from a lethal dose of influenza A, a new study shows.

8 May 2025

Scientists use AI to build mutation-proof antibodies for SARS-CoV-2

Researchers used AI models to design antibodies that neutralize over 1,300 SARS-CoV-2 strains, including Delta and Omicron. The models demonstrated faster, cheaper, and more adaptable therapeutic development compared to traditional methods.

5 May 2025

Researchers unravel the genetic contributions to familial Alzheimer's disease development

A group of researchers in the lab of Prof. Lucía Chávez Gutiérrez (VIB-KU Leuven) have unraveled the genetic contributions to familial Alzheimer's Disease development and revealed how specific mutations act as a clock to predict the disease age of onset.

5 May 2025

New gene editing tool enables precise control of mitochondrial mutation load

Mitochondrial diseases affect approximately 1 in 5,000 people worldwide, causing debilitating symptoms ranging from muscle weakness to stroke-like episodes.

3 May 2025

TDP-43 dysfunction in brain blood vessels may drive Alzheimer's and ALS progression

Reduced levels of a critical protein are linked to devastating brain diseases like Alzheimer's, frontotemporal dementia, and amyotrophic lateral sclerosis (ALS). Surprisingly, the protein shortage primarily affects the brain's blood vessels.

30 Apr 2025