Gene News and Research

Latest Gene News and Research

New gene therapy strategy reactivates dormant genes to treat blood disorders

Researchers have found a promising new method for gene therapy. They successfully restarted inactive genes by bringing them closer to genetic switches on the DNA called enhancers.

19 Jun 2025

Early infant behavior predicts cognitive ability decades later

Watching a baby babble, play and interact with others can provide useful insight into what their cognitive ability might be like decades later, according to new University of Colorado Boulder research published in the journal PNAS.

19 Jun 2025

Mass photometry recognized by U.S. Pharmacopeia in new AAV reference standards for gene therapy quality

Refeyn, the company behind pioneering mass photometry technology, announces that mass photometry has been recognized by the U.S. Pharmacopeia (USP)—and referenced in the new draft USP general chapter <1067>—as a key orthogonal method for characterizing USP's new adeno-associated virus (AAV) reference standards.

19 Jun 2025

Global guideline released by WHO on managing sickle cell disease in pregnancy

The World Health Organization (WHO) today released its first-ever global guideline on the management of sickle cell disease (SCD) during pregnancy, addressing a critical and growing health challenge that can have life-threatening consequences for both women and babies.

19 Jun 2025

Time-restricted feeding restores microbial rhythms and boosts metabolism

The gut microbiome, a vast assortment of bacteria and other microorganisms that inhabit our digestive system, plays a critical role in converting food into energy.

19 Jun 2025

Researchers explore new approach to target rare epileptic disorders

Humans have tens of thousands of genes, many of which produce proteins essential to basic bodily functions.

18 Jun 2025

Immune cell signatures explain variations in systemic sclerosis severity

Treating rare diseases can be complicated at the best of times, and it gets even more complicated when different patients with the same disease exhibit different symptoms.

18 Jun 2025

Yale researchers triple precision and efficiency in genome editing

The promise of genome editing to help understand human diseases and create new therapies is vast, but technological limitations have limited advancement of the field.

18 Jun 2025

Stanford study identifies gene signature that predicts immune health

At the start of the COVID-19 pandemic, information began to trickle in about who was at higher risk of a dangerous outcome from infection. Men. Those over 65. Smokers. Those with obesity.

18 Jun 2025

New switchable CAR-T therapy enters phase 1 trial for advanced breast cancer

The Calibr-Skaggs Institute for Innovative Medicines, the drug development division of Scripps Research, today announced a milestone in cancer treatment with the dosing of the first patient in a phase 1 trial (NCT06878248) evaluating CLBR001 + ABBV-461, a modular, switchable chimeric antigen receptor T cell (sCAR-T) therapy in patients with advanced or metastatic breast cancer who have no suitable treatment options.

18 Jun 2025

CAR T-cell therapy successfully treats rare autoimmune disease of the peripheral nervous system

Physicians from Bochum have used a cancer treatment method to treat severe autoimmune diseases.

18 Jun 2025

Interferon pathway genes linked to treatment success in juvenile arthritis

A set of genes that could be used to help doctors predict which children will respond well to treatment for juvenile idiopathic arthritis (JIA) have been identified by researchers at UCL and Great Ormond Street Hospital for Children (GOSH), who are part of the CLUSTER Consortium.

18 Jun 2025

Disrupted biological rhythms in teens can produce unexpected brain effects

A new McGill University-led study suggests that disrupting the body's internal clock during adolescence can alter how the brain responds to an in-utero risk factor linked to certain brain disorders.

17 Jun 2025

Healthy glial cell transplants slow Huntington’s disease progression

Huntington's disease has long defied attempts to rescue suffering neurons. A new study in Cell Reports shows that transplanting healthy human glial progenitor cells into the brains of adult animal models of the disease not only slowed motor and cognitive decline but also extended lifespan.

17 Jun 2025

Breakthrough therapy targets aggressive bladder cancer variant

Bladder tumors that have been excluded from clinical trials have a few things in common that could lead to new therapies.

17 Jun 2025

Black tea helps in one Parkinson’s type, but soda and pesticides worsen another

A large longitudinal study found that pesticide exposure and caffeinated soda intake were linked to more severe motor symptoms in idiopathic Parkinson’s disease (iPD). In contrast, black tea consumption was associated with milder symptoms in patients with LRRK2-related Parkinson’s disease (LRRK2-PD).

16 Jun 2025