Gene News and Research

Latest Gene News and Research

Finnish researchers uncover how to optimize bexmarilimab use in cancer care

Researchers at the University of Turku, Finland, have taken a step towards more personalized and effective cancer treatments.

20 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

Genetic variations in Prader-Willi syndrome offer key insights into autism and psychosis

In a comprehensive Genomic Press Invited Expert Review, researchers from the University of Haifa have synthesized cutting-edge findings on Prader-Willi syndrome (PWS), revealing how this complex neurodevelopmental disorder serves as a critical model for understanding the interplay between autism spectrum disorder (ASD) and psychotic spectrum disorders (PSD).

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

MIT researchers discover new potential drug targets for Alzheimer’s disease

By combining information from many large datasets, MIT researchers have identified several new potential targets for treating or preventing Alzheimer's disease.

20 May 2025

Discovery reveals how neuronal ion channels store molecular memory

Researchers have discovered how an ion channel in the brain's neurons has a kind of 'molecular memory', which contributes to the formation and preservation of lifelong memories.

19 May 2025

Study reveals how PAX3 protein drives melanoma growth

PAX3 is a transcription factor (proteins involved in converting DNA into RNA) that drives melanoma progression by promoting cell growth, migration and survival, while inhibiting cellular terminal differentiation, which is the final stage where a cell becomes specialized and cell division ends.

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

EMA designates allopurinol as first orphan drug for the treatment of Marfan syndrome

The European Medicines Agency (EMA) has designated allopurinol as the first orphan drug for the treatment of Marfan syndrome, a rare connective tissue disease which as no cure to date.

19 May 2025

Common anti-inflammatory drugs may aid virus spread

New research shows an unintended and unfortunate side effect of common drugs. They can simply help viruses spread.

19 May 2025

Genomic study of non-Hispanic Black veterans with prostate cancer yields key insights for precision medicine

In the largest clinical genomic profiling study of non-Hispanic Black men with metastatic prostate cancer to date, researchers from Moffitt Cancer Center, University of Pennsylvania, University of California Los Angeles and the Veterans Affairs (VA) National Precision Oncology Program found key differences in tumor biology between non-Hispanic Black and non-Hispanic white veterans, but similar survival outcomes when both groups had equal access to care.

16 May 2025

Single gene found to play a big role in how the liver stores energy

A new study published in Science Advances reveals that a single gene plays a big role in how the liver stores energy, a process that's critical for overall health and for managing diseases like type 2 diabetes.

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

Malaria parasite found to evade the immune system by shutting down key genes

Researchers at Weill Cornell Medicine have discovered how a parasite that causes malaria when transmitted through a mosquito bite can hide from the body's immune system, sometimes for years.

16 May 2025

Aptamer announces new data at ASGCT

Aptamer Group plc, the leading developer of next-generation synthetic binders delivering innovation to the life science industry, presented new data on its Optimer therapeutic delivery vehicle for liver fibrosis at the American Society of Gene and Cell Therapy (ASGCT) Annual Meeting in New Orleans, LA (13-17 May 2025).

From Aptamer Group 16 May 2025

New gene editor evoCAST enables precise insertion of complete genes

Ask scientists what gene editing tool is most needed to advance gene therapy, and they'd probably describe a system that's now close to realization in the labs of Samuel Sternberg at Columbia University Vagelos College of Physicians and Surgeons and David Liu at the Broad Institute of MIT and Harvard.

15 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

Ubrogepant relieves early migraine symptoms and may help prevent full-blown attacks

A phase 3 trial found that ubrogepant, given during the premonitory phase of migraine, improved common warning symptoms such as fatigue, light and sound sensitivity, and neck pain. These benefits were observed as early as one hour post-treatment, suggesting early intervention can reduce migraine burden.

15 May 2025

Customized CRISPR gene editing therapy successfully treats infant with rare disease

In a historic medical breakthrough, a child diagnosed with a rare genetic disorder has been successfully treated with a customized CRISPR gene editing therapy by a team at Children's Hospital of Philadelphia (CHOP) and Penn Medicine.

15 May 2025

Study sheds light on why women have faster heartbeats than men

For decades, doctors and researchers have puzzled over a basic heart rhythm mystery: Why do women tend to have faster heartbeats while men are more likely to develop irregular rhythms like atrial fibrillation (AFib)?

15 May 2025

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Gene News and Research

Latest Gene News and Research

Finnish researchers uncover how to optimize bexmarilimab use in cancer care

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

Genetic variations in Prader-Willi syndrome offer key insights into autism and psychosis

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

MIT researchers discover new potential drug targets for Alzheimer’s disease

Discovery reveals how neuronal ion channels store molecular memory

Study reveals how PAX3 protein drives melanoma growth

Study uncovers common trigger across gene mutations that cause ALS

EMA designates allopurinol as first orphan drug for the treatment of Marfan syndrome

Common anti-inflammatory drugs may aid virus spread

Genomic study of non-Hispanic Black veterans with prostate cancer yields key insights for precision medicine

Single gene found to play a big role in how the liver stores energy

Study reveals subtle effects of recessive mutations on health and reproduction

Malaria parasite found to evade the immune system by shutting down key genes

Aptamer announces new data at ASGCT

New gene editor evoCAST enables precise insertion of complete genes

HIV vaccine study marks breakthrough in targeting broad immune responses

Ubrogepant relieves early migraine symptoms and may help prevent full-blown attacks

Customized CRISPR gene editing therapy successfully treats infant with rare disease

Study sheds light on why women have faster heartbeats than men

Industry Focus: Cell & Gene Therapy eBook

How the Vutara VXL simplifies single-molecule imaging

How cohesin mechanics and nanotracker tech are redefining DNA research

Todd Bauman and Chris Povich